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38 Bone Marrow Failure Syndrome Trials Near You
Power is an online platform that helps thousands of Bone Marrow Failure Syndrome 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 PowerTreosulfan-Based Conditioning for Bone Marrow Failure
Columbus, Ohio
This trial tests if a combination of three drugs can reduce complications for patients with bone marrow failure diseases. The drugs work by killing harmful cells, stopping their growth, and reducing immune reactions.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:1 - 49
Key Eligibility Criteria
Disqualifiers:Aplastic Anemia, Fanconi Anemia, MDS, Others
40 Participants Needed
Geriatric Care Optimization for Blood Cancers
Columbus, Ohio
This clinical trial tests whether a geriatric optimization plan (GO!) works to improve survival in patients over 60 with a hematologic malignancy or bone marrow failure syndrome eligible for allogeneic hematopoietic cell transplant. GO! focuses on creating a tailored and specific plan for each patient to make changes in their daily lives. These may include changes to their diet, sleep, activity, medicines, or even referrals to other providers depending on the patient's needs. Studying survival and quality of life in patients over 60 receiving an allogeneic hematopoietic cell transplant may help identify the effects of treatment.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:60+
Key Eligibility Criteria
Disqualifiers:Dementia, No Transplant, Others
30 Participants Needed
Bone Marrow Transplant for Dyskeratosis Congenita
Cincinnati, Ohio
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
Chemotherapy + Stem Cell Transplant for Fanconi Anemia
Cincinnati, Ohio
The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:3+
Key Eligibility Criteria
Disqualifiers:Active CNS Leukemia, HIV, Others
70 Participants Needed
Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders
Pittsburgh, Pennsylvania
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:2 - 55
Key Eligibility Criteria
Disqualifiers:Active Malignancy, Severe Aplastic Anemia, Others
100 Participants Needed
RIC + BMT for Non-Malignant Disorders
Grand Rapids, Michigan
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:1 - 21
Key Eligibility Criteria
Disqualifiers:HLA-identical Sibling Donor, Cirrhosis, Uncontrolled Infection, HIV, Others
29 Participants Needed
Imatinib for Bone Marrow Failure Syndrome
Bethesda, Maryland
Background:
Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood, bone marrow, and lymph nodes.
Objective:
To test a drug (imatinib) in people with RUNX1 mutations that cause symptoms.
Eligibility:
Adults aged 18 and older with RUNX1 mutations. Healthy people without this mutation, including family members of affected participants, are also needed.
Design:
Participants with the RUNX1 mutation will be screened. They will have a physical exam with blood and urine tests. They will have a test of their heart function. They may need a new bone marrow biopsy: A sample of soft tissue will be removed from inside a bone.
Imatinib is a tablet taken by mouth once a day, every day, at home. Affected participants in different parts of the study will take imatinib for either 28 days or up to 84 days.
Participants will visit the clinic once a week for the first 28 days that they are taking the imatinib. Then they will come once every 2 weeks if they are taking the drug for 84 days. Blood, urine, and tests of heart function will be repeated. They may opt to have the bone marrow biopsy repeated after they finish their course of imatinib.
Participants will have a follow-up visit 30 days after they stop taking imatinib.
Participants who do not have the RUNX1 mutation will have 1 clinic visit. They will have blood tests. They will fill out questionnaires. They may opt to have a bone marrow biopsy....
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Pregnancy, Hematologic Therapy, Cardiac Conditions, Others
Must Not Be Taking:Aspirin, Anti-platelet, CYP3A4 Inhibitors, Others
78 Participants Needed
Bitopertin for Diamond-Blackfan Anemia
Bethesda, Maryland
This trial tests bitopertin, a daily pill, in adults with Diamond-Blackfan anemia. The drug aims to reduce harmful levels of heme in the blood, which can help manage the disease. Participants will take the drug for several months, with periodic check-ups to monitor their response. Bitopertin has shown promise in improving anemia in a mouse model.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Cardiac Disease, Active Infections, Pregnancy, Others
Must Not Be Taking:Androgens, Corticosteroids, Antivirals, Others
30 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
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
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
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
This is a Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), and inherited bone marrow failure syndromes (IBMFS).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:4 - 40
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis B/C, Pregnancy, Others
27 Participants Needed
Cord Blood Transplant for Blood Diseases
Rochester, New York
This trial uses stem cells from a baby's umbilical cord to treat patients who need new healthy stem cells. Patients first get strong medicine to clear out unhealthy cells, then receive the new stem cells, and take medications to prevent complications. Umbilical cord blood has been used in the treatment of various diseases, including leukemias, lymphomas, and immune system disorders.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:2 - 75
Key Eligibility Criteria
Disqualifiers:Pregnancy, HIV, Uncontrolled Infection, Others
30 Participants Needed
Let's Get REAL Tool for Pediatric Blood Cancers
Saint Louis, Missouri
The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular therapy decisions (SCTCT). The investigators will pilot the intervention among 24 youth and their parents, stratified by youth age (stratum 1, 8-12 years of age and stratum 2, 13-17 years of age).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:8+
Key Eligibility Criteria
Disqualifiers:Severe Medical Problems, Cognitive Incapacity, Others
60 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
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
Stem Cell Transplant for Bone Marrow Failure Syndrome
Philadelphia, Pennsylvania
The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Early Phase 1
Age:< 30
Key Eligibility Criteria
Disqualifiers:Infections, HLA Matched Sibling, Pregnancy
100 Participants Needed
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
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
Why Other Patients Applied
"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
"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
"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
"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."
FF
ADHD PatientAge: 31
"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
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
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
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
Cord Blood Transplant for Blood Cancers
New York, New York
This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 21
Key Eligibility Criteria
Disqualifiers:Advanced Metabolic Disease, CNS Leukemic, Others
31 Participants Needed
Haploidentical Transplant for Severe Aplastic Anemia
Memphis, Tennessee
This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched to the patient. This includes studying the effects of the chemotherapy, radiation, the transplanted cell product and additional white blood cell (lymphocyte) infusions on the patient's body, disease and overall survival. The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year post-hematopoietic cell transplantation (HCT).
Primary Objectives
* To estimate the rate of engraftment at 30 days after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide.
* To estimate the overall survival and event free survival at 1-year post transplantation.
Secondary Objectives
* To calculate the incidence of acute and chronic GVHD after HCT.
* To calculate the rate of secondary graft rejection at 1-year post transplantation
* To calculate the cumulative incidence of viral reactivation (CMV, EBV and adenovirus).
* To describe the immune reconstitution after TCR αβ+ T-cell-depleted graft infusion at 1 month, 3 months, 6 months, 9 months, and 1 year.
Exploratory Objectives
* To longitudinally assess the phenotype and epigenetic profile of T-cells in SAA patients receiving HCT for SAA.
* To assess the phenotype and epigenetic profile of T-cells in DLI administered to SAA patients post HCT.
* To longitudinally assess CD8 T cell differentiation status in SAA patients using an epigenetic atlas of human CD8 T cell differentiation.
* To examine the effector functions and proliferative capacity of CD8 T cells isolated from SAA patients before and after DLI.
* Quantify donor derived Treg cells at different time points in patients received HCT.
* Determine Treg activation status at different stages after HCT.
* Are specific features of the DLI product associated with particular immune repertoire profiles post-transplant?
* How does the diversity and functional profile of the DLI product alter the response to pathogens in the recipient?
* Do baseline features of the recipient's innate and adaptive immune cells correlate with post-transplant immune repertoires and response profiles?
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 21
Key Eligibility Criteria
Disqualifiers:Fanconi Anemia, Dyskeratosis Congenita, MDS, Others
Must Not Be Taking:Alemtuzumab, ATG
21 Participants Needed
Stem Cell Mobilization with Plerixafor for Abnormal Endometrium
Orange, Connecticut
This trial tests if a new treatment can help women with uterine lining issues by moving their own stem cells into their blood to repair the uterus. It aims to improve pregnancy chances for women with specific reproductive challenges.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Early Phase 1
Age:18 - 40
Sex:Female
Key Eligibility Criteria
Disqualifiers:Hydrosalpinx, Endometriosis, Diminished Ovarian Reserve, Others
30 Participants Needed
This clinical trial tests next generation sequencing (NGS) for the detection of precursor features of pre-myeloid cancers and bone marrow failure syndromes. NGS is a procedure that looks at relevant cancer associated genes and what they do. Finding genetic markers for pre-malignant conditions may help identify patients who are at risk of pre-myeloid cancers and bone marrow failure syndromes and lead to earlier intervention.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Key Eligibility Criteria
Disqualifiers:Under 18 Years
2000 Participants Needed
HSCT for Leukemia and Lymphoma
Minneapolis, Minnesota
This is a Phase II study of allogeneic hematopoietic stem cell transplant (HCT) using a myeloablative preparative regimen (of either total body irradiation (TBI); or, fludarabine/busulfan for patients unable to receive further radiation). followed by a post-transplant graft-versus-host disease (GVHD) prophylaxis regimen of post-transplant cyclophosphamide (PTCy), tacrolimus (Tac), and mycophenolate mofetil (MMF).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 60
Key Eligibility Criteria
Disqualifiers:Active CNS Malignancy, Uncontrolled Infection, Others
300 Participants Needed
UCB Transplant After Non-Myeloablative Prep for Blood Cancers
Minneapolis, Minnesota
This is a phase II trial using a non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen with modifications based on factors including diagnosis, disease status, and prior treatment. Single or double unit selected according to current University of Minnesota umbilical cord blood graft selection algorithm.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:< 75
Key Eligibility Criteria
Disqualifiers:Pregnancy, Active Infection, HIV, Others
16 Participants Needed
TCD HCT for Fanconi Anemia
Minneapolis, Minnesota
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 65
Key Eligibility Criteria
Disqualifiers:Pregnancy, Uncontrolled Infection, Malignant Cancer, Others
48 Participants Needed
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Frequently Asked Questions
How much do Bone Marrow Failure Syndrome 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 Bone Marrow Failure Syndrome 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 Bone Marrow Failure Syndrome 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 Bone Marrow Failure Syndrome 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 Bone Marrow Failure Syndrome 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 Bone Marrow Failure Syndrome clinical trials?
Most recently, we added Methadone for Pain Management in Stem Cell Transplants, Pacritinib for Myelodysplastic Syndrome and CD45RA Depleted Stem Cell Addback for Leukemia to the Power online platform.
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