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We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

Bask
Bask GillCEO at Power
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      Why We Started Power

      We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

      Bask
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

      50 Aplastic Anemia Trials Near You

      Power is an online platform that helps thousands of Aplastic Anemia patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

      Learn More About Power
      No Placebo
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      Breakthrough Medication

      Cyclosporine + Eltrombopag for Aplastic Anemia

      Bethesda, Maryland
      Background: Severe aplastic anemia (SAA) is a rare and serious blood disorder. It causes the immune system to turn against bone marrow cells. Standard treatment for SSA is a combination of 3 drugs (Cyclosporine \[CsA\], Eltrombopag \[EPAG\], and horse anti-thymocyte globulin \[h-ATG\]). Researchers want to see if starting people at a lower dose of CsA with EPAG before giving them h-ATG is helpful. Objective: To learn if early initiation of oral therapy with CsA and EPAG is safe and effective in people who have SAA and have not been treated with a course of immunosuppressive therapy and EPAG. Eligibility: People ages 3 and older with SAA Design: Participants will be screened with: medical history physical exam electrocardiogram blood tests family history bone marrow biopsy current medicines. Participants may be screened remotely via telephone conference. Participants will take a lower oral dose of CsA and EPAG. They will take CsA twice a day for 6 months. They will take EPAG for 6 months. Those who cannot visit the NIH Clinical Center within 72 hours will start taking the drugs at home. They will have weekly telephone calls with NIH staff until they visit the Clinical Center. Participants may get h-ATG at the Clinical Center for 4 days. For this, they will have a central line placed. It is a plastic tube inserted into a neck, chest, or arm vein. Participants will repeat most screening tests throughout the study. Participants will have follow-up visits at the Clinical Center at 3 months, 6 months, and annually for 5 years after the start of the study....
      No Placebo Group
      Prior Safety Data

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:3 - 99

      Key Eligibility Criteria

      Disqualifiers:Fanconi Anemia, Clonal Disorder, Others
      Must Be Taking:Cyclosporine, Eltrombopag

      80 Participants Needed

      Stem Cell Transplant for Severe Aplastic Anemia

      Bethesda, Maryland
      Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help transplants. Objectives: To see how safely and effectively SAA, MDS and PNH are treated using peripheral blood hematopoietic stem cells from a family member plus chemotherapy. Eligibility: Recipients ages 4-60 with SAA, MDS or PNH and their relative donors ages 4-75 Design: Recipients will have: * Blood, urine, heart, and lung tests * Scans * Bone marrow sample Recipients will need a caregiver for several months. They may make fertility plans and a power of attorney. Donors will have blood and tissue tests, then injections to boost stem cells for 5-7 days. Donors will have blood collected from a tube in an arm or leg vein. A machine will separate stem cells and maybe white blood cells. The rest of the blood will be returned into the other arm or leg. In the hospital for about 1 month, recipients will have: * Central line inserted in the neck or chest * Medicines for side effects * Chemotherapy over 8 days and radiation 1 time * Stem cell transplant over 4 hours Up to 6 months after transplant, recipients will stay near NIH for weekly physical exams and blood tests. At day 180, recipients will go home. They will have tests at their doctor s office and NIH several times over 5 years.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:4 - 75

      Key Eligibility Criteria

      Disqualifiers:HIV, Pregnancy, Fanconi Anemia, Others

      56 Participants Needed

      Umbilical Cord Blood Transplant for Aplastic Anemia and Myelodysplastic Syndrome

      Bethesda, Maryland
      Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant. Researchers are testing ways to make stem cell transplant safer and more effective. Objective: To test if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective. Eligibility: Recipients ages 4-60 with SAA or MDS Donors ages 4-75 Design: Recipients will be screened with: * Blood, lung, and heart tests * Bone marrow biopsy * CT scan Recipients will have an IV line placed into a vein in the neck. Starting 11 days before the transplant they will have several chemotherapy infusions and 1 30-minute radiation dose. Recipients will get the donor cells through the IV line. They will stay in the hospital 3-4 weeks. After discharge, they will have visits: * First 3-4 months: 1-2 times weekly * Then every 6 months for 5 years Donors will be screened with: * Physical exam * Medical history * Blood tests Donors veins will be checked for suitability for stem cell collection. They may need an IV line to be placed in a thigh vein. Donors will get Filgrastim or biosimilar (G-CSF) injections daily for 5-7 days. On the last day, they will have apheresis: Blood drawn from one arm or leg runs through a machine and into the other arm or leg. This may be repeated 2 days or 2-4 weeks later.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:4 - 75

      Key Eligibility Criteria

      Disqualifiers:HIV, Pregnancy, Fanconi Anemia, Others

      37 Participants Needed

      Sirolimus for Aplastic Anemia

      Bethesda, Maryland
      Background: People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body's immune system stops the bone marrow from making these cells. The treatment cyclosporine leads to better blood counts. But when this treatment is stopped, the disease may return in 1 in 3 people. The drug sirolimus may help by suppressing the immune system. Objective: To evaluate and compare the usefulness of sirolimus in preventing aplastic anemia from returning after cyclosporine is stopped, compared with stopping cyclosporine alone. Eligibility: People ages 2 and older with SAA who: Have responded to immunosuppressive therapy that includes cyclosporine, and continue to take cyclosporine Are not taking drugs with hematologic effects Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Bone marrow biopsy: The area above the hipbone will be numbed. A thin needle will remove some bone marrow. Participants will be randomly assigned to a group. All will stop cyclosporine. Group 1 will take sirolimus by mouth at the same time each day for 3 months with close monitoring. Group 2 will not receive the study drug but will be monitored closely. Participants will have clinical tests for the first 3 months: Weekly blood test Monthly fasting blood test For group 1, measurements of sirolimus in the blood every 1 2 weeks Participants will have clinic visits at 3 months, 12 months, and annually for 5 years after the study starts. They may have another visit if their SAA returns. These will include: Blood and urine tests Bone marrow biopsy...
      No Placebo Group
      Prior Safety Data

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2
      Age:2 - 99

      Key Eligibility Criteria

      Disqualifiers:Myelodysplastic Syndrome, Pregnancy, Cancer, Others
      Must Be Taking:Cyclosporine

      83 Participants Needed

      Eltrombopag + Immunosuppression for Severe Aplastic Anemia

      Bethesda, Maryland
      Background: * Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells. This causes the bone marrow to stop making red blood cells, platelets, and white blood cells. Standard treatment for this disease is horse-ATG and cyclosporine, which suppress the immune system and stop it from attacking the bone marrow. However, this treatment does not work in all people. Some people still have poor blood cell counts even after treatment. * Eltrombopag is a drug designed to mimic a protein in the body called thrombopoietin. It helps the body to make more platelets. It may also cause the body to make more red and white blood cells. Studies have shown that eltrombopag may be useful when added to standard treatment for severe aplastic anemia. It may help improve poor blood cell counts. Objectives: - To test the safety and effectiveness of adding eltrombopag to standard immunosuppressive therapy for severe aplastic anemia. Eligibility: - Individuals at least 2 years of age who have severe aplastic anemia that has not yet been treated. Design: * Participants will be screened with a physical exam, medical history, and blood tests. Blood and urine samples will be collected. * Participants will start treatment with horse-ATG and cyclosporine. Treatment will be given according to the standard of care for the disease. * Cohort 1: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 6 months. * Cohort 2: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 3 months. * Cohort 3 and Extension Cohort: Participants will start taking eltrombopag on Day 1. They will take eltrombopag for up to 6 months. * Participants may receive other medications to prevent infections during treatment. * Treatment will be monitored with frequent blood tests. Participants will also fill out questionnaires about their symptoms and their quality of life.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:2 - 95

      Key Eligibility Criteria

      Disqualifiers:Fanconi Anemia, Clonal Disorder, Others
      Must Be Taking:Horse-ATG, Cyclosporine

      207 Participants Needed

      Stem Cell Transplant for Severe Aplastic Anemia

      Bethesda, Maryland
      Background: * Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia. Allogenic stem cell transplants encourage the growth of new bone marrow to replace that of the recipient. Because stem cell transplants can have serious complications, researchers are interested in developing new approaches to stem cell transplants that will reduce the likelihood of these complications. * By reducing the number of white blood cells included in the blood taken during the stem cell collection process, and replacing them with a smaller amount of white blood cells collected prior to stem cell donation, the stem cell transplant may be less likely to cause severe complications for the recipient. Researchers are investigating whether altering the stem cell transplant donation procedure in this manner will improve the likelihood of a successful stem cell transplant with fewer complications. Objectives: - To evaluate a new method of stem cell transplantation that may reduce the possibly of severe side effects or transplant rejection in the recipient. Eligibility: * Recipient: Individuals between 4 and 80 years of age who have been diagnosed with a blood disease that can be treated with allogenic stem cell transplants. * Donor: Individuals between 4 and 80 years of age who are related to the recipient and are eligible to donate blood. OR unrelated donors found through the National Marrow Donor Program. Design: * All participants will be screened with a physical examination and medical history. * DONORS: * Donors will undergo an initial apheresis procedure to donate white blood cells. * After the initial donation, donors will receive injections of filgrastim to release bone marrow cells into the blood. * After 5 days of filgrastim injections, donors will have apheresis again to donate stem cells that are present in the blood. * RECIPIENTS: * Recipients will provide an initial donation of white blood cells to be used for research purposes only. * From 7 days before the stem cell transplant, participants will be admitted to the inpatient unit of the National Institutes of Health Clinical Center and will receive regular doses of cyclophosphamide, fludarabine, and anti-thymocyte globulin to suppress their immune system and prepare for the transplant. * After the initial chemotherapy, participants will receive the donated white blood cells and stem cells as a single infusion. * After the stem cell and white blood cell transplant, participants will have regular doses of cyclosporine and methotrexate to prevent rejection of the donor cells. Participants will have three doses of methotrexate within the week after the transplant, but will continue to take cyclosporine for up to 4 months after the transplant. * Participants will remain in inpatient care for up to 1 month after the transplant, and will be followed with regular visits for up to 3 years with periodic visits thereafter to evaluate the success of the transplant and any side effects.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:4 - 80

      Key Eligibility Criteria

      Disqualifiers:Organ Failure, Pregnancy, Active Infection, Others

      120 Participants Needed

      Eltrombopag for Aplastic Anemia

      Bethesda, Maryland
      Background: * Moderate aplastic anemia is a blood disease which may require frequent blood and platelet transfusions. Sometimes patients with this disease can be treated with immunosuppressive drugs. Not all patients respond and not all patients are suitable for this treatment. * Thrombopoietin (TPO) is a protein made by the body. The bone marrow needs TPO to produce platelets. TPO may also be able to stimulate bone marrow stem cells to produce red cells and white cells. However, TPO cannot be given by mouth. This has led researchers to develop the drug eltrombopag, which acts in the same way and can be given by mouth. Eltrombopag has been shown to safely increase platelet numbers in healthy volunteers and in patients with other chronic blood diseases, including severe aplastic anemia. Researchers are interested in looking at whether eltrombopag can be given to people with moderate aplastic anemia and significantly low blood cell counts. Objectives: - To evaluate the safety and effectiveness of eltrombopag in people with moderate aplastic anemia or patients with bone marrow failure and unilineage cytopenia who need treatment for significantly low blood cell counts. Eligibility: - People at least 2 years of age who have moderate aplastic anemia or bone marrow failure and unilineage cytopenia,and significantly low blood cell counts. Design: * Patients will be screened with a physical examination, medical history, blood tests, a bone marrow biopsy, and an eye exam. * Patients will receive eltrombopag by mouth once a day. * Patients will have weekly blood tests to monitor the effectiveness of the treatment and adjust the dose in response to possible side effects. * Patients may continue to take eltrombopag if their platelet count or hemoglobin increases, their requirement for platelet or blood transfusion decreases after 16 to 20 weeks of treatment, and there have been no serious side effects. Access to the drug will continue until the study is closed. Patients will be asked to return for a follow-up visit 6 months after the last dose of medication.
      No Placebo Group
      Prior Safety Data

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2
      Age:2 - 100

      Key Eligibility Criteria

      Disqualifiers:Fanconi Anemia, HIV, Cancer, Others
      Must Not Be Taking:ATG, Campath, Cytokines, Others

      34 Participants Needed

      Ruxolitinib for Bone Marrow Failure

      Bethesda, Maryland
      Background: Immune bone marrow failure is a condition that occurs when a person s immune system attacks the cells of the bone marrow. This can lead to diseases including different types of anemias and blood cancers. Some of these diseases can be deadly. Better treatments are needed. Objective: To test a drug (ruxolitinib) in people with different types of immune bone marrow failure. Eligibility: Adults aged 18 and older with an immune bone marrow failure. Design: Participants will be screened. They will have a physical exam. They will give samples of blood and saliva. They will have a bone marrow biopsy: A large needle will be inserted into a small cut to remove a sample of the soft tissue inside the bone. Some participants may have a skin biopsy: A small piece of skin will be removed. Some may have a computed tomography (CT) scan: They will lie on a table that slides into a donut-shaped machine that uses X-rays to make pictures of the inside of the body. Ruxolitinib is a tablet taken by mouth. Participants will take the drug twice a day for up to 6 months. Participants will have blood tests every week while they are taking the drug. These tests can be done by the participant s own physician and the results sent to the researchers. Participants will have clinic visits after taking the drug for 3 months and 6 months and then after 1, 2, and 3 years. The blood tests and bone marrow biopsy will be repeated. Participants who improve while taking the drugs may go on to an extension phase of the study.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2

      Key Eligibility Criteria

      Disqualifiers:Fanconi Anemia, MDS/MPN, Others
      Must Not Be Taking:Chemotherapy, Immunomodulatory, Others

      145 Participants Needed

      Cord Blood Transplant for Blood Disorders

      Bethesda, Maryland
      Background: - Cord blood is blood that is taken from the umbilical cord and placenta of healthy newborns after childbirth. The cord blood collected from a baby is called a cord blood unit. Cord blood units are stored frozen in public cord blood banks. About 10,000 cord blood transplants have been performed in children and adults for blood cancers and other diseases in the world. These transplants have helped save lives and improve treatments. However, not all available units of cord blood have been collected, stored, and licensed according to specific government requirements. These unlicensed units can still be used in transplant, but they can only be given as part of specific research studies. This study will evaluate the safety of giving these unlicensed units by recording any problems that may occur during and after giving the cord blood. Objectives: - To test the safety and effectiveness of unlicensed cord blood units in people who need stem cell transplants. Eligibility: - Individuals who are scheduled to have a stem cell transplant. Design: Participants will be screened with a medical history and physical exam. Participants will receive the cord blood unit as part of their stem cell transplant procedure. The transplant will be performed according to the current standard of care for the procedure. After the transplant, participants will be monitored for up to 1 year. Any problems or side effects from the transplant will be treated as necessary. All outcomes will be reported to the National Cord Blood Program and to the Center for International Blood and Marrow Transplant.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:4+

      Key Eligibility Criteria

      Disqualifiers:Licensed CB Products, Unlicensed CB From NMDP

      500 Participants Needed

      Cord Blood Transplant for Blood Cancers and Diseases

      Bethesda, Maryland
      Background: - Cord blood transplants can treat cancers and other diseases in children and adults. The U.S. Food and Drug Administration (FDA) requires cord blood to be collected and stored under certain safety standards. However, most available cord blood units were collected before the FDA set these standards. These units may not meet FDA standards, but they do meet similar standards set and followed by the National Marrow Donor Program (NMDP). Cord blood units that do not meet the new FDA standards may be used for transplants only as part of a research study. Doctors want to allow people who need transplants to receive cord blood that meets NMDP standards but may not meet FDA standards. Objectives: - To allow selected cord blood units that do not meet current FDA standards to be used for transplant. Eligibility: - Individuals who need cord blood units for transplant, and who best match cord blood units that are not FDA-licensed. Design: Participants will provide consent to receive cord blood that meets NMDP standards but may not meet FDA standards. Participants will remain on the study for observation for up to 1 year after transplant, or until they withdraw from the study for personal or medical reasons....
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:4 - 99

      Key Eligibility Criteria

      Disqualifiers:Licensed CBUs, International Centers, Other IND Protocol, Others

      500 Participants Needed

      Low-Dose Danazol for Telomere Disease

      Bethesda, Maryland
      Background: DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA. Some people with short telomeres or other gene changes can develop diseases of the bone marrow, lung, and liver. Researchers want to see if low doses of the hormone drug danazol can help. Objective: To study the safety and effect of low dose danazol. Eligibility: People ages 3 and older with a telomere disease who have either very short telomeres and a specific gene change. They must also show signs of aplastic anemia, lung, or liver disease. Design: Participants will be screened in another protocol. Participants will have: * Medical history * Physical exam * Blood tests * Lung exam. They will breathe into an instrument that records the amount and rate of air breathed in and out over a period of time. 6-minute walking test. * Abdominal ultrasound and liver scan. These tests use sound waves to measure the fibrosis in the liver. Some participants will have: * Pregnancy test * Small sample of the liver removed * Bone marrow biopsy. The bone will be numbed and a small needle will take a sample of the marrow. All participants will have hormone levels checked. All child participants will see a pediatric endocrinologist. Children may need to have a hand x-ray. We will monitor patients for 6 months before starting danazol. Participants will take danazol by mouth twice a day for 1 year. Participants must return to the clinic at 6 months and 12 months while on danazol and 6 months after stopping it. They will have blood and urine tests, a lung exam, abdominal ultrasound, and liver scan.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:3 - 99

      Key Eligibility Criteria

      Disqualifiers:Thrombosis, Hepatitis B/C, Cancer, Others
      Must Not Be Taking:Androgens, Anti-fibrotics, Chemotherapy, Others

      40 Participants Needed

      Pacritinib for Myelodysplastic Syndrome

      Bethesda, Maryland
      Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS/MPN can also progress to acute myeloid leukemia. Treatments for these disorders are risky and not always effective. Better treatments are needed. Objective: To test a study drug (pacritinib) in adults and children with MDS or MDS/MPN. Eligibility: Children (aged 12 to 17 years) and adults (aged 18 years and older) with MDS or MDS/MPN. Design: Participants will be screened. They will have a physical exam with blood tests. They will have tests of their heart function. They may have a bone marrow biopsy: An area over the hip will be numbed; a needle will be inserted to remove a sample of soft tissue from inside the hipbone. Pacritinib is a capsule taken by mouth. All participants will take the study drug 2 times a day, every day, in 28-day cycles. They will write down the date and time they take each capsule. Doctors will assign varying dosages of the drug to different participants. Participants will have clinic visits each week during cycle 1; every 2 weeks during cycle 2; and gradually increasing to every 3 months after cycle 13. Treatment will continue for up to 8 years. Bone marrow biopsies, heart tests, and other tests will be repeated at intervals throughout the study. Participants will also fill out questionnaires about their quality of life, the symptoms of their disease, and other topics.
      No Placebo Group

      Trial Details

      Trial Status:Not Yet Recruiting
      Trial Phase:Phase 1, 2
      Age:12+

      Key Eligibility Criteria

      Disqualifiers:Bleeding Disorders, Cardiac Conditions, Infections, Others
      Must Not Be Taking:Anticoagulants, Antiplatelets, CYP3A4 Inhibitors

      160 Participants Needed

      IST + BMT for Aplastic Anemia

      Washington, District of Columbia
      Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular treatment for patients with aplastic anemia who have a matched sibling (brother or sister), or family donor is a bone marrow transplant. Patients without a matched family donor normally are treated with immune suppressive therapy (IST). Match unrelated donor (URD) bone marrow transplant (BMT) is used as a secondary treatment in patients who did not get better with IST, had their disease come back, or a new worse disease replaced it (like leukemia). This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat SAA. The trial will also assess whether health-related quality of life and early markers of fertility differ between those randomized to URD BMT or IST, as well as assess the presence of marrow failure-related genes and presence of gene mutations associated with MDS or leukemia and the change in gene signatures after treatment in both study arms. This study treatment does not include any investigational drugs. The medicines and procedures in this study are standard for treatment of SAA.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:0 - 25

      Key Eligibility Criteria

      Disqualifiers:Inherited Bone Marrow Syndromes, MDS, HIV, Others
      Must Not Be Taking:Androgens, Eltrombopag, Romiplostim, Others

      234 Participants Needed

      Donor Matching Strategies for Blood Cancer Transplants

      Washington, District of Columbia
      The purpose of this study is to determine if a search strategy of searching for an HLA-matched unrelated donor for allogeneic transplantation if possible then an alternative donor if an HLA-matched unrelated donor is not available versus proceeding directly to an alternative donor transplant will result in better survival for allogeneic transplant recipients within 2 years after study enrollment.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      1753 Participants Needed

      Gene Modified T-cells for Blood Disorders

      Washington, District of Columbia
      This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (graft versus host disease).
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:1 - 26

      Key Eligibility Criteria

      Disqualifiers:Severe Cardiovascular, Active Infections, Others
      Must Not Be Taking:Immunosuppressants

      120 Participants Needed

      Bone Marrow Transplant for Aplastic Anemia

      Baltimore, Maryland
      BMT CTN 2207 will investigate the use of marrow transplantation for treatment of severe aplastic anemia that has not previously been treated.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:3 - 75

      Key Eligibility Criteria

      Disqualifiers:Inherited Bone Marrow Failure, MDS, Others
      Must Not Be Taking:Immunosuppressive Therapy

      60 Participants Needed

      Darzalex Faspro for Blood Cancers

      Baltimore, Maryland
      This research is being done to investigate the safety and effectiveness of Darzalex Faspro (daratumumab and hyaluronidase-fihj) (a monoclonal antibody that targets plasma cells that make antibodies) and whether it can lower donor specific antibodies (DSA) levels to low enough levels to permit patients to proceed with allogeneic peripheral blood transplant (alloBMT). Those being asked to participate have high DSA levels that puts those being asked to participate at high risk of rejecting the available donor's blood stem cells and making those being asked to participate ineligible to receive a stem cell transplant.
      No Placebo Group
      Prior Safety Data

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Early Phase 1

      Key Eligibility Criteria

      Disqualifiers:COPD, Asthma, HIV, Hepatitis, Others
      Must Not Be Taking:Anti-CD38 Therapies

      8 Participants Needed

      Emapalumab for Aplastic Anemia

      Richmond, Virginia
      The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options. Funding Source- FDA OOPD
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:0 - 25

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Infection, Previous SAA Treatment, Inherited Bone Marrow Failure, Others

      55 Participants Needed

      Abatacept for Graft-versus-Host Disease Prevention

      Wilmington, Delaware
      This trial will see if extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will prevent acute and chronic graft-versus-host disease (GVHD) in children and adolescents receiving unrelated donor (URD) hematopoietic stem cell transplantation (HSCT), without compromising their engraftment or reconstitution of protective immunity to infection. The study will enroll 30 pediatric patients with serious non-malignant hematologic diseases (NMHD) undergoing URD HSCT. The trial will include patients with 7/8 donors and those with 8/8 (matched) donors. All participants will receive 8 doses of abatacept. Recruitment is expected to last for about 2 years and participants will be followed for up to 3 years.
      No Placebo Group
      Prior Safety Data

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2
      Age:< 20

      Key Eligibility Criteria

      Disqualifiers:HLA Matched Donor, Pulmonary Dysfunction, Renal Dysfunction, Severe Cardiac Dysfunction, Others

      30 Participants Needed

      Stem Cell Transplant for Bone Marrow Failure Syndrome

      Philadelphia, Pennsylvania
      This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased
      Age:< 25

      Key Eligibility Criteria

      Disqualifiers:Myelodysplastic Syndrome, No Suitable Donor, Pregnancy, Others

      50 Participants Needed

      Why Other Patients Applied

      "My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."

      HZ
      Arthritis PatientAge: 78

      "As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."

      IZ
      Healthy Volunteer PatientAge: 38

      "I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."

      WR
      Obesity PatientAge: 58

      "I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."

      AG
      Paralysis PatientAge: 50

      "I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."

      ID
      Pancreatic Cancer PatientAge: 40
      Match to a Aplastic Anemia Trial

      CD45RA Depleted Stem Cell Addback for Leukemia

      Philadelphia, Pennsylvania
      The major morbidities of allogeneic hematopoietic stem cell transplant (HSCT) using donors that are not human leukocyte antigen (HLA) matched siblings are graft vs host disease (GVHD) and life- threatening infections. T cell receptor alpha beta (TCRαβ) T lymphocyte depletion and CD19+ B lymphocyte depletion of alternative donor hematopoietic stem cell (HSC) grafts is effective in preventing GVHD, but immune reconstitution may be delayed, increasing the risk of infections. The central hypothesis of this study is that an addback of CD45RO memory T lymphocytes, derived from a fraction of the original donor peripheral stem cell product depleted of CD45RA naïve T lymphocytes, will accelerate immune reconstitution and help decrease the risk of infections in TCRab/CD19 depleted PSCT.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:1 - 25

      Key Eligibility Criteria

      Disqualifiers:Hodgkin Lymphoma, Non-Burkitt Lymphoma, Others
      Must Not Be Taking:Alemtuzumab

      100 Participants Needed

      Bone Marrow Transplant for Dyskeratosis Congenita

      Philadelphia, Pennsylvania
      This trial tests a new bone marrow transplant method for patients with Dyskeratosis congenita. It uses fludarabine and antibodies to avoid harmful side effects, aiming to improve survival and reduce complications. Fludarabine-based regimens have been shown to be effective and feasible in reducing transplant-related morbidity in patients with Dyskeratosis congenita.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2
      Age:30 - 65

      Key Eligibility Criteria

      Disqualifiers:MDS, AML, HIV, Pregnancy, Others
      Must Not Be Taking:Fludarabine, Alemtuzumab, Mycophenolate, Others

      40 Participants Needed

      NR Supplementation + Exercise for Cancer Survivors

      Philadelphia, Pennsylvania
      This will be a randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on skeletal muscle quality and VO2max in AYA HCT survivors. The primary outcome is the change in muscle strength (isometric knee extension) from baseline to 16 weeks. Key secondary outcomes are the change in muscle strength (ankle plantarflexion) from baseline to 16 weeks, the change in grip strength from baseline to 16 weeks, the change in lower extremity muscle mass from baseline to 16 weeks, the change in muscle OXPHOS capacity from baseline to 16 weeks, and the change in aerobic capacity (VO2 max) from baseline to 16 weeks.

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:10 - 30

      Key Eligibility Criteria

      Disqualifiers:Diabetes, Kidney Disease, Liver Disease, Others
      Must Not Be Taking:Statins, Glucocorticoids, NAD+ Precursors

      80 Participants Needed

      Reduced-Intensity Conditioning for Blood Cancers

      Philadelphia, Pennsylvania
      This phase II clinical trial evaluates whether a modified modality of conditioning reduces treatment-related mortality (TRM) in patients who undergo a hematopoietic stem cell transplant (HSCT) for a hematological malignancy. HSCT is a curative therapy for many hematopoietic malignancies, however this regimen results in higher rates of TRM than other forms of treatment. In recent years, less intense conditioning regimens with radiation and chemotherapy prior to HSCT have been developed. Radiation therapy uses high energy sources to kill cancer cells and shrink tumors while chemotherapy drugs like fludarabine and cyclophosphamide work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This study evaluates whether a two-step approach with lower-intensity regimens of these treatments prior to HSCT reduces the rate of TRM.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2

      Key Eligibility Criteria

      Disqualifiers:HIV, CNS Malignancy, Pregnancy, Others

      63 Participants Needed

      Fludarabine RIC for Bone Marrow Failure Syndrome

      Philadelphia, Pennsylvania
      This is a pilot study to determine whether fludarabine-based reduced intensity conditioning (RIC) regimens facilitate successful donor engraftment of patients with acquired aplastic anemia (AA) and Inherited bone marrow failure (iBMF) syndromes undergoing Matched related donor bone marrow transplant (MRD-BMT).
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Early Phase 1
      Age:< 22

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Infections, Myelodysplastic Syndrome, Others

      25 Participants Needed

      T Cell-Depleted Stem Cell Transplant for Leukemia

      Pittsburgh, Pennsylvania
      This phase II trial studies how well naive T-cell depletion works in preventing chronic graft-versus-host disease in children and young adults with blood cancers undergoing donor stem cell transplant. Sometimes the transplanted white blood cells from a donor attack the body's normal tissues (called graft versus host disease). Removing a particular type of T cell (naive T cells) from the donor cells before the transplant may stop this from happening.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:6 - 26

      Key Eligibility Criteria

      Disqualifiers:Active CNS Disease, HIV-positive, Uncontrolled Infections, Pregnancy, Others
      Must Not Be Taking:Tacrolimus, Fludarabine, Methotrexate

      68 Participants Needed

      Stem Cell Transplant for Blood Cancer

      Middletown, New Jersey
      The main purpose of this study is to learn if a new combination of chemotherapy, in combination with low-dose radiation, will be safe for the patient, and at the same time provide the best opportunity to cure the bone marrow cancer. The combination of chemotherapy and radiation described in the study is considered 'low intensity.' Although the chemotherapy agents used in this study and for transplant are FDA approved, the chemotherapy treatment and conditioning regimens or combinations listed in this consent are not yet FDA approved. The CliniMACS device is FDA approved for one type of T cell depletion (positive selection of the stem cells) but not approved yet for other type of T cell depletion, which is being studied on this protocol. This pilot study, along with other studies will serve as the basis for FDA approval, if outcomes are favorable.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      17 Participants Needed

      CD34+ Stem Cell Selection for Bone Marrow Failure Syndromes

      New York, New York
      This trial uses a special machine to filter out harmful cells from donor blood to make stem cell transplants safer for young patients with non-cancerous diseases. By removing specific cells, it aims to prevent a serious immune reaction.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:< 40

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Infection, Pregnancy, HIV, Others

      37 Participants Needed

      Eltrombopag for Aplastic Anemia

      Hackensack, New Jersey
      This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Age:1 - 18

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      51 Participants Needed

      Stem Cell Transplant for Leukemia

      New York, New York
      The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults. CD34+ stem cells are the cells that make all the types of blood cells in the body. GVHD is a condition that results from a reaction of transplanted donor T-lymphocytes (a kind of white blood cell) against the recipient's body and organs. Study subjects will be offered treatment involving the use of the CliniMACS® Reagent System (Miltenyi Biotec), a CD34+ selection device to remove T-cells from a peripheral blood stem cell transplant in order to decrease the risk of acute and chronic GVHD. This study involves subjects who are diagnosed with a malignant disease, that has either failed standard therapy or is unlikely to be cured with standard non-transplant therapy, who will receive a peripheral blood stem cell transplant. A malignant disease includes the following: Chronic Myeloid Leukemia (CML) in chronic phase, accelerated phase or blast crisis; Acute Myelogenous Leukemia (AML); Myelodysplastic Syndrome (MDS); Juvenile Myelomonocytic Leukemia (JMML); Acute Lymphoblastic Leukemia (ALL); or Lymphoma (Hodgkin's and Non-Hodgkin's).
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:< 22

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Infection, Pregnancy, Breastfeeding

      14 Participants Needed

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      Learn More About Trials
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      Frequently Asked Questions

      How much do Aplastic Anemia clinical trials pay?

      Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

      How do Aplastic Anemia clinical trials work?

      After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Aplastic Anemia trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Aplastic Anemia is 12 months.

      How do I participate in a study as a "healthy volunteer"?

      Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

      What does the "phase" of a clinical trial mean?

      The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

      Do I need to be insured to participate in a Aplastic Anemia medical study?

      Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

      What are the newest Aplastic Anemia clinical trials?

      Most recently, we added Methadone for Pain Management in Stem Cell Transplants, Ruxolitinib for Aplastic Anemia and Pacritinib for Myelodysplastic Syndrome to the Power online platform.