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43 Thrombocytopenia Trials Near You
Power is an online platform that helps thousands of Thrombocytopenia 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 PowerThe objective of the NeoPlaTT trial is to test whether, among extremely preterm infants born at 23 0/7 to 26 6/7 weeks' gestation, a lower platelet transfusion threshold, compared to a higher threshold, improves survival without major or severe bleeding up to 40 0/7 weeks' postmenstrual age (PMA).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:1 - 48
Key Eligibility Criteria
Disqualifiers:Comfort Care, Thrombocytopenia, No Vitamin K, Others
2433 Participants Needed
Treosulfan-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
Efgartigimod for ITP
Columbus, Ohio
This trial is testing ARGX-113, a medication for adults with primary ITP, a condition where the immune system attacks platelets. The medication aims to calm the immune system to prevent it from attacking these important blood cells.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:Pregnancy, Lactation, Hypersensitivity, Others
Must Not Be Taking:Anti-CD20, Romiplostim, Monoclonal Antibodies, Others
101 Participants Needed
RAY121 for Immune System Disorders
Columbus, Ohio
This trial is testing a new drug called RAY121, which aims to help people with certain immune system diseases by blocking a part of the immune system that causes inflammation. The trial will check if the drug is safe and effective for patients with conditions like APS, BP, BS, DM, IMNM, and ITP.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Cancer, Hepatitis, HIV, Tuberculosis, Others
Must Not Be Taking:Antivirals, Antibiotics, Antifungals, Others
144 Participants Needed
Eltrombopag vs Standard Treatment for ITP in Children
Columbus, Ohio
This trial tests eltrombopag, a drug that helps make more platelets, in children with a specific blood condition. The goal is to see if it works better than standard treatments.
Stay on current meds
No Placebo Group
Prior Safety Data
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:1 - 18
Key Eligibility Criteria
Disqualifiers:Not Listed
122 Participants Needed
Avatrombopag for Pediatric ITP
Columbus, Ohio
This trial is testing a medication called avatrombopag, which helps increase blood platelets. It targets children who have had low platelet counts for an extended period due to their immune system attacking these cells. The medication works by encouraging the body to make more platelets, which are important for stopping bleeding. Avatrombopag has been shown to increase platelet counts in patients with low platelet levels, including those with liver disease.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:1 - 17
Key Eligibility Criteria
Disqualifiers:Secondary ITP, Thrombosis, MDS, Others
75 Participants Needed
Canakinumab for Clonal Cytopenia
Columbus, Ohio
This phase II trial tests how well canakinumab works to prevent progression to cancer in patients with clonal cytopenias of unknown significance (CCUS). CCUS is a blood condition defined by a decrease in blood cells. Blood cells are composed of either red blood cells, white blood cells, or platelets. In patients with CCUS, blood counts have been low for a long period of time. Patients with CCUS also have a mutation in one of the genes that are responsible for helping blood cells develop. The combination of genetic mutations and low blood cell counts puts patients with CCUS at a higher risk to develop blood cancers in the future. This transformation from low blood cell counts to cancer may be caused by inflammation in the body. Canakinumab is a monoclonal antibody that may block inflammation in the body by targeting a specific antibody called the anti-human interleukin-1beta (IL-1beta).
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Concurrent Malignancy, MDS, Active Infection, Others
Must Not Be Taking:Immune Modulators, ESAs
110 Participants Needed
Enasidenib for Clonal Cytopenia
Columbus, Ohio
Study researchers think that a drug called enasidenib may help people with clonal cytopenia of undetermined significance (CCUS) because the drug blocks the mutated IDH2 protein, which may improve blood cell counts. The purpose of this study is to find out whether enasidenib is a safe and effective treatment for CCUS.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Active Malignancy, Hematologic Malignancy, Infection, Others
4 Participants Needed
Ianalumab + Eltrombopag for Low Platelet Count
Dayton, Ohio
The purpose of this study is to evaluate the effect of two different doses of ianalumab added to eltrombopag to prolong Time to Treatment Failure (TTF) in adults with primary ITP who failed previous first-line treatment with steroids.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis, Coagulation Disorders, Others
Must Be Taking:Eltrombopag
150 Participants Needed
The purpose of this study is to evaluate the effect of two different doses of ianalumab versus placebo in addition to first-line corticosteroids in maintaining platelet count ≥30 G/L in adult participants with primary ITP.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:Evans Syndrome, Coagulation Disorders, Others
Must Be Taking:Corticosteroids
225 Participants Needed
HMPL-523 for Low Platelet Count
Cleveland, Ohio
This is an open-label, multicenter study to evaluate the safety, tolerability, and efficacy of HMPL-523 in adult subjects with ITP.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Secondary ITP, Active Malignancy, Others
48 Participants Needed
Selinexor for Myelofibrosis
Cleveland, Ohio
The main purpose of this study with corresponding optional expansion is to evaluate the efficacy of selinexor in JAKi-naïve participants with myelofibrosis (MF) and moderate thrombocytopenia based on spleen volume reduction (SVR). Additional efficacy and safety parameters will also be assessed during the study.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Pregnancy, Heart Disease, Stroke, Others
118 Participants Needed
Avatrombopag for Thrombocytopenia in Gastrointestinal Cancer
Cleveland, Ohio
The purpose of this study is to compare the efficacy of two study drugs, Avatrobopag versus placebo, to treat persistent Chemotherapy-Induced Thrombocytopenia (CIT) in patients with gastrointestinal (GI) malignancies receiving cytotoxic chemotherapy.
The names of the study drugs involved in this study are:
* Avatrombopag (a thrombopoietin receptor agonist)
* Matching placebo
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Hematologic Malignancy, Bone Marrow Invasion, Others
Must Not Be Taking:Thrombopoietin Receptor Agonists
60 Participants Needed
Rilzabrutinib for Immune Thrombocytopenia
Cleveland, Ohio
This trial tests rilzabrutinib, a drug that may help people with chronic ITP who have very low platelet counts. The drug works by blocking a substance that causes the immune system to destroy platelets. Participants will take the drug for several months to see if it helps increase their platelet levels.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:10+
Key Eligibility Criteria
Disqualifiers:Secondary ITP, Pregnancy, Malignancy, Others
Must Not Be Taking:Immunosuppressants, Rituximab, BTK Inhibitors
232 Participants Needed
Ivosidenib for Blood Disorders
Cleveland, Ohio
This trial is testing a medication called ivosidenib in patients with a specific blood condition and a genetic mutation. The goal is to see if the medication can safely improve their blood counts. The study is also designed to be conducted remotely. Ivosidenib has shown significant improvements in patients with certain types of cancers.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Active Malignancy, Solid Tumor Therapy, Others
Must Not Be Taking:CYP3A4 Inducers
20 Participants Needed
Efgartigimod for Low Platelet Count
Fort Wayne, Indiana
This trial is testing a new injection treatment for adults whose immune systems destroy their own platelets. The treatment aims to calm the immune system to stop it from attacking these important blood cells. Romiplostim promotes platelet production and has been used in the treatment of this condition.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:Pregnancy, Hypersensitivity, Other Investigational Drugs, Others
Must Not Be Taking:Anti-CD20, Romiplostim, Monoclonal Antibodies, Others
173 Participants Needed
Anticoagulation Strategies for Blood Clots in Cancer Patients
Windsor, Ontario
Patients with cancer are prone to have blood clots, which are usually treated with blood thinners. The main complication of blood thinners is bleeding. This is especially a concern when the number of platelets in the blood is lower than 50,000 per microliter. The role of platelets is to stop bleeding, so when the number of platelets is low, patients are at a higher risk of bleeding. Cancer patients are prone to have lower platelet numbers due to cancer therapies and/or cancer itself. It is not clear what the best treatment is for cancer patients who need blood thinners for a blood clot but have low platelet counts.
The investigators plan to do a small study called a pilot study to help plan for a larger study in such patients. In the pilot study, investigators will include 50 patients with cancer, low platelet counts, and a blood clot diagnosed within 4 weeks. Patients will be randomly assigned to one of the two treatment strategies: the full dose of blood thinners along with platelet transfusion or a reduced dose of blood thinners without platelet transfusion. The investigators will follow all patients for 90 days. If this pilot study is successful, it will help lead to a much larger trial, which will provide important information on the best treatment strategy in these patients.
No Placebo Group
Trial Details
Trial Status:Recruiting
Key Eligibility Criteria
Disqualifiers:Short Life Expectancy, Low Creatinine, Others
Must Be Taking:Blood Thinners
50 Participants Needed
VLX-1005 for Heparin Induced Thrombocytopenia
Ann Arbor, Michigan
The purpose of this study is to evaluate the efficacy and safety of VLX-1005, a 12-lipoxygenase (12-LOX) enzyme inhibitor in treating heparin induced thrombocytopenia (HIT). Participants with suspected HIT will receive the usual standard of care, and will be assigned randomly to either VLX-1005 or placebo treatment. The study will measure important outcomes including platelet count, stroke, pulmonary embolus (clot to the lungs) and bleeding.
Trial Details
Trial Status:Recruiting
Key Eligibility Criteria
Disqualifiers:Renal Dialysis, Pregnant, Others
Must Be Taking:Heparin
60 Participants Needed
Oral Azacitidine for Myelodysplastic Syndrome
Pittsburgh, Pennsylvania
The purpose of this study is to evaluate the safety and efficacy of oral azacitidine in participants with low to intermediate International Prognostic Scoring System Revised (IPSS-R) myelodysplastic syndrome (MDS).
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Key Eligibility Criteria
Disqualifiers:Hypoplastic MDS, MDS-EB2, Others
Must Not Be Taking:Azacitidine, Decitabine
230 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
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
"I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."
ZS
Depression PatientAge: 51
"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'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
Romiplostim for Chemotherapy-Induced Low Platelet Count
Lansing, Michigan
This trial tests romiplostim, a drug that boosts platelet production, in adults with certain cancers undergoing chemotherapy. It aims to help these patients maintain their chemotherapy schedule by increasing their platelet counts. Romiplostim has shown effectiveness in increasing platelet counts in patients with low platelet levels due to chemotherapy.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:Leukemia, Myeloma, Heart Failure, Others
Must Be Taking:Carboplatin-based Chemotherapy
162 Participants Needed
Rilzabrutinib for Immune Thrombocytopenia
Midland, Michigan
This is a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with a platelet count \<30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The dose-finding portion of the study has been completed. Part B treatment dose is 400 mg twice daily.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Pregnancy, Drug Abuse, Organ Transplant, Others
81 Participants Needed
Romiplostim for Low Platelet Count in Cancer
Kitchener, Ontario
Study of Romiplostim for Chemotherapy-induced Thrombocytopenia in Adult Subjects with Gastrointestinal, Pancreatic, or Colorectal Cancer
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Key Eligibility Criteria
Disqualifiers:Not Listed
162 Participants Needed
Povetacicept for Autoimmune Hemolytic Anemia
Hamilton, Ontario
This trial is testing a medication called povetacicept in adults with certain blood disorders where the immune system attacks their own blood cells. The goal is to see if povetacicept is safe and can help improve these conditions. Participants will receive the medication regularly for several months, with an option to extend the treatment for additional months.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Secondary AIHA, CAD, ITP, Others
Must Not Be Taking:Rituximab, IVIg, Sutimlimab, Others
126 Participants Needed
PF-06835375 for Low Platelet Count
Germantown, Maryland
This trial uses a new injectable medicine called PF-06835375. It targets adults with long-lasting or chronic low platelet counts due to primary immune thrombocytopenia (ITP). The medicine works by reducing specific immune cells to help increase platelet counts.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Recent Bleeding, Splenectomy, Others
61 Participants Needed
Slow vs Standard Platelet Transfusion for Low Platelet Count
Bethesda, Maryland
Background:
Platelets are cell fragments in the blood that help it clot. Some people get very low platelet counts during a disease or treatment. Low platelet counts can cause severe bleeding. Some people are not helped by platelet transfusions at the standard transfusion rate. This is called platelet transfusion refractoriness (PTR). Researchers want to learn more about transfusing platelets so they can make transfusions more effective.
Objectives:
To study the effects of transfusing platelets more slowly than the standard rate. To obtain data to improve the effectiveness of platelet transfusions in people with PTR and decrease the risk of bleeding in some people.
Eligibility:
Adults ages 18-100 who have very low platelet counts requiring platelet transfusion, and have evidence of PTR
Design:
Participants will be screened with a review their recent NIH medical records. They will have blood drawn.
Participants will have up to three 12-hour treatment blocks. They can have only one block per day. During each block, they will have 2 platelet transfusions in those 12 hours.
One transfusion will take place over 1 hour (SHORT infusion). The other will take place over 4 hours (LONG infusion).
Participants will be randomly put in 1 of 2 treatment groups. This will dictate whether they get the SHORT or LONG infusion first.
Participants will have blood drawn:
* When they enroll
* Right before each transfusion
* 2, 4, and 6 hours after each transfusion
Each blood draw will consist of a complete blood count. Smaller tubes that require only small amounts of blood will be used to minimize the amount of blood drawn.
No Placebo Group
Trial Details
Trial Status:Recruiting
Key Eligibility Criteria
Disqualifiers:Not Listed
30 Participants Needed
Fostamatinib for Immune Thrombocytopenia (ITP)
Bethesda, Maryland
Background:
People who have a blood stem cell transplant can sometimes develop cytopenia. This means that their levels of one or more types of blood cell, such as the red cells or platelets, are lower than they should be. This can occur because a person s immune system might attack these cells after a stem cell transplant. Up to 20% of people who have blood stem cell transplants develop cytopenias, which can lead to anemia, severe bleeding, infections, and other problems. Treatments are needed to help keep blood cell levels stable after blood stem cell transplant.
Objective:
To evaluate the long-term effects of a study drug (fostamatinib) in people with cytopenia after a blood stem cell transplant.
Eligibility:
People who responded well to fostamatinib in an earlier study.
Design:
Participants will be screened. They will have a physical exam and blood tests.
Fostamatinib is an oral tablet taken by mouth. Participants will take the pills at the same dose and frequency as they did during the previous study. They will take the pills for up to 21 months. The dosage of the drug may be reduced over time if their blood cell levels are stable.
Participants will have a medical assessment every month. This can be with their local doctor or at the NIH clinic.
Participants will have blood tests every 3 months.
Participants will have a follow-up visit after they stop taking the drug. Their vital signs will be taken, and they will have blood drawn. They will answer questions about their health.
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Severe Psychiatric Illness, Uncontrolled Hypertension, Others
Must Be Taking:Fostamatinib
20 Participants Needed
Fostamatinib for Post-Transplant Cytopenias
Bethesda, Maryland
Background:
People who have a blood stem cell transplant can sometimes develop cytopenia. This means that their levels of one or more types of blood cell, such as the red cells or platelets, are lower than they should be. This can occur because a person s immune system might attack these cells after a stem cell transplant. Cytopenia can lead to anemia, severe bleeding, infections, and other problems. Treatments are needed to help keep blood cell levels stable after blood stem cell transplant.
Objective:
To test a study drug (fostamatinib) in people who have cytopenia after a blood stem cell transplant.
Eligibility:
People aged 18 to 75 years who have cytopenia after a blood stem cell transplant.
Design:
Participants will be screened. They will have a physical exam. They will have blood, urine, and stool tests.
Fostamatinib is an oral tablet taken by mouth. Participants will take the pills 2 times a day for 12 weeks.
Participants will have a medical assessment every 2 weeks; their vital signs will be checked, and they will have blood and stool tests. Participants must come to the NIH clinic for these visits in weeks 4 and 12. Other visits may be done by telephone or telehealth; the blood and stool tests can be sent to the researchers from a local lab.
After 4 weeks, some participants may begin taking a higher dose of the drug.
Participants will return for a final medical assessment 2 weeks after they finish taking the drug.
Participants who complete this study and show evidence that fostamatinib has increased their blood cell counts may enroll in an extension study to continue taking fostamatinib.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Severe Psychiatric Illness, Uncontrolled Hypertension, Others
Must Not Be Taking:Fostamatinib, Cytokine-targeting Biologics
20 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
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
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Frequently Asked Questions
How much do Thrombocytopenia 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 Thrombocytopenia 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 Thrombocytopenia 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 Thrombocytopenia 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 Thrombocytopenia 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 Thrombocytopenia clinical trials?
Most recently, we added Pirtobrutinib for Immune Thrombocytopenic Purpura, Platelet Transfusion Thresholds for Low Platelet Count in Premature Infants and Fostamatinib for Immune Thrombocytopenia (ITP) to the Power online platform.
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