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45 Thalassemia Trials
Power is an online platform that helps thousands of Thalassemia 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.
Etavopivat for Sickle Cell Disease
Cincinnati, OhioThis trial is testing a new oral medicine called etavopivat in patients with sickle cell disease or thalassemia. The medicine helps red blood cells produce energy more efficiently. The goal is to reduce the need for blood transfusions and increase hemoglobin levels.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:12 - 65
Sex:All
Key Eligibility Criteria
Disqualifiers:Hepatic Dysfunction, HIV, Hepatitis B/C, Others
60 Participants Needed
Etavopivat for Sickle Cell Anemia
Cincinnati, OhioEtavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:All
Sex:All
325 Participants Needed
EDIT-301 for Beta Thalassemia
Cleveland, OhioThis trial tests a new treatment called EDIT-301, which modifies a patient's own stem cells to treat severe beta Thalassemia. It targets adults who need regular blood transfusions. The goal is to fix their cells so they can produce healthy blood cells and reduce the need for transfusions.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18 - 35
Sex:All
Key Eligibility Criteria
Disqualifiers:Alpha Thalassemia, Hemoglobinopathy, Malignancy, Others
9 Participants Needed
Long-Term Safety of Luspatercept for Blood Disorders
Cleveland, OhioA Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants:
* Participants receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the investigator, may derive clinical benefit from continuing treatment with luspatercept
* Participants in the follow-up phase previously treated with luspatercept or placebo in the parent protocol will continue into long-term post-treatment follow-up in the rollover study until the follow-up commitments are met
* The study design is divided into the Transition Phase, Treatment Phase and Follow-up Phase. Participants will enter transition phase and depending on their background will enter either the treatment phase or the Long-term Post-treatment Follow-up (LTPTFU) phase
* Transition Phase is defined as one Enrollment visit
* Treatment Phase: For participants in luspatercept treatment the dose and schedule of luspatercept in this study will be the same as the last dose and schedule in the parent luspatercept study. This does not apply to participants that are in long-term follow-up from the parent protocol
* Follow-up Phase includes:
- 42 Day Safety Follow-up Visit
* During the Safety Follow up, the participants will be followed for 42 days after the last dose of luspatercept, for the assessment of safety-related parameters and adverse event (AE) reporting
- Long-term Post-treatment Follow-up (LTPTFU) Phase
* Participants will be followed for overall survival every 6 months for at least 5 years from first dose of luspatercept in the parent protocol, or 3 years of post-treatment from last dose, whichever occurs later, or until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. Participants will also be monitored for progression to AML or any malignancies/pre-malignancies. New anticancer or disease related therapies should be collected at the same time schedule
Participants transitioning from a parent luspatercept study in post-treatment follow-up (safety or LTPTFU) will continue from the same equivalent point in this rollover study.
The ACE-536-LTFU-001 rollover study will be terminated, and relevant participants will discontinue from the study when all participants fulfill 5 years on the study, including treatment and follow-up.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Breastfeeding, Significant Medical Condition, Others
Must Be Taking:Luspatercept
665 Participants Needed
Mitapivat for Thalassemia
Detroit, MichiganThe primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-dependent alpha- or beta-thalassemia (TDT).
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
258 Participants Needed
Mitapivat for Thalassemia
Detroit, MichiganThis trial is testing mitapivat, a drug that may help improve anemia by making red blood cells healthier and last longer. It targets patients with a specific type of thalassemia who don't need regular blood transfusions but still suffer from anemia.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
194 Participants Needed
Hydroxyurea + EPO for Sickle Cell Disease
Pittsburgh, PennsylvaniaThe proposed study is a Phase 1/2 multi-center study evaluating the safety and efficacy of erythropoietin (EPO) in combination with hydroxyurea in the treatment of chronic anemia in patients with sickle cell disease (SCD).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
17 Participants Needed
Bone Marrow Transplant for Sickle Cell Disease
Pittsburgh, PennsylvaniaThis trial uses stem cells from mismatched donors with certain immune cells removed to treat patients with severe blood disorders who lack a perfect donor match. The approach aims to replace damaged cells, reduce complications, and support recovery with additional immune cells. A new technique has been developed to improve the treatment process.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:5 - 40
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, CNS Event, Infections, Others
Must Be Taking:Hydroxyurea
5 Participants Needed
Hemanext ONE System for Sickle Cell Anemia
Pittsburgh, PennsylvaniaThe overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia. The Hemanext ONE device was cleared through the De Novo process in September 2023.
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Unphased
Age:7+
Sex:All
48 Participants Needed
Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders
Pittsburgh, PennsylvaniaThe 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
Sex:All
Key Eligibility Criteria
Disqualifiers:Active Malignancy, Severe Aplastic Anemia, Others
100 Participants Needed
CRISPR-Cas9 Gene Editing for Beta Thalassemia
Chicago, IllinoisThis is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:12 - 35
Sex:All
59 Participants Needed
MED-Go App for Sickle Cell Disease
Chicago, IllinoisThe primary objective of this study is to evaluate a potential behavioral intervention (MED-Go app). To meet this objective, the researchers will conduct a pilot randomized controlled trial to test the feasibility and acceptability of MED-Go app in adolescents and young adults (AYA) with sickle cell disease (SCD). The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:12 - 21
Sex:All
40 Participants Needed
HU-Go App for Sickle Cell Disease Adherence
Chicago, IllinoisThe primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD). To meet this objective, the researchers will conduct a prospective cohort study to determine the longitudinal relationship between HU adherence and health-related quality of life (HRQOL) overtime among AYA with SCD. The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:12 - 21
Sex:All
68 Participants Needed
Abatacept for Graft-versus-Host Disease Prevention
Chicago, IllinoisThis 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
Sex:All
30 Participants Needed
Taurine Supplementation for Cardiomyopathy in Thalassemia
Toronto, OntarioHypothesis: Taurine, in combination with standard iron chelation therapy, is more effective than chelation therapy alone in reducing cardiac iron overload, oxidative stress and cardiac damage in β-Thalassemia.
Protocol: Sixty subjects with transfusion dependent β-Thalassemia receiving deferasirox iron chelation therapy will be recruited and randomized in a 1:1 ratio to either (1) placebo and continuation of their iron chelation or (2) a combination of iron chelation plus taurine. Transfusion and safety visits will be scheduled monthly with clinical/biochemical assessment visits every three months. The efficacy of taurine combined with standard chelation therapy will be assessed at baseline and 12 months posttreatment by both cardiac T2\*MRI, and cardiac function. The recruitment period is projected to be 12 months from initiation.
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Heart Failure, Pregnancy, Others
Must Be Taking:Iron Chelation
60 Participants Needed
Benserazide for Beta Thalassemia
Toronto, OntarioBeta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers oxygen throughout the body.The diseases are characterized by hemolytic anemia, organ damage, and early mortality without treatment. Increases in another type of (normal) hemoglobin, fetal globin (HbF), which is normally silenced in infancy, reduces anemia and morbidity. Even incremental augmentation of fetal globin is established to reduce red blood cell pathology, anemia, certain complications, and to improve survival.
This trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein (HbF and red blood cells expressing HbF)and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice. The study drug acts by suppressing 4 repressors of the fetal globin gene promoter in progenitor cells from patients. The drug has been used for 50 years in a combination product for different actions - to enhance half-life and reduce side effects of a different active drug- and is considered safe for long-term use.
This trial will first evaluate 3 dose levels in small cohorts of nontransfused patients with beta thalassemia intermedia. The most active dose will then be evaluated in larger subject groups with beta thalassemia and other hemoglobinopathies, such as sickle cell disease.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
36 Participants Needed
This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:2 - 11
Sex:All
Key Eligibility Criteria
Disqualifiers:Prior HSCT, Sickle Cell Variant, Others
15 Participants Needed
This trial tests if using low dose radiation and certain drugs can help patients with beta-thalassemia or sickle cell disease better accept donor stem cells. The treatment aims to suppress the immune system to reduce rejection of the new cells.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:4 - 80
Sex:All
56 Participants Needed
AG-348 for Thalassemia
Toronto, OntarioStudy AG348-C-010 is a multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of treatment with AG-348 in adult participants with non-transfusion-dependent thalassemia (NTDT). This study includes a core period (up to 24 weeks) followed by an extension period (up to 10 years) for eligible participants. 20 participants with NTDT were enrolled. The initial dose of AG-348 was 50 milligrams (mg) twice daily (BID) with one potential dose-level increase to 100 mg BID at the Week 6 visit based on the participant's safety and hemoglobin (Hb) concentrations.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
20 Participants Needed
Biotin Labeling for Sickle Cell Anemia
Bethesda, MarylandBackground:
Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of healthy cells. SCD and other blood disorders can be managed with drugs or cured with a bone marrow transplant. Researchers want to know how long RBCs survive in people with SCD and other blood disorders before and after treatment compared to those who had a bone marrow transplant.
Objective:
To learn how long RBCs survive in the body in people with SCD and other blood disorders compared to those whose disease was cured with a bone marrow transplant.
Eligibility:
People aged 18 years or older with SCD or another inherited blood disorder. People whose SCD or blood disorder was cured with a bone marrow transplant are also needed.
Design:
Participants will be screened. They will have a physical exam with blood and urine tests.
Participants will have about 7 tablespoons of blood drawn. In the lab, this blood will be mixed with a vitamin called biotin. Biotin sticks to the outside of RBCs. This process is called "biotin labeling of RBCs." The next day, the participant s own biotin-labeled RBCs will be returned to their bloodstream.
Participants will return regularly to have smaller blood samples (about 2 teaspoons) drawn. These samples will be tested to detect the percentage of cells that have biotin labels. These visits may be every 2 weeks, 4 weeks, or some other interval. Participants will continue this schedule for up to 20 weeks or until biotin can no longer be detected....
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Early Phase 1
Age:18 - 100
Sex:All
Key Eligibility Criteria
Disqualifiers:Biotin Supplements, Chronic Transfusion Therapy, Hemodialysis, Malignancy, Others
100 Participants Needed
JSP191 + Stem Cell Transplant for Sickle Cell Anemia
Bethesda, MarylandBackground:
Sickle cell disease (SCD) is an inherited disorder of the blood. It can damage a person s organs and cause serious illness and death. A blood stem cell transplant is the only potential cure for SCD. Treatments that improve survival rates are needed.
Objective:
To find out if a new antibody drug (briquilimab, JSP191) improves the success of a blood stem cell transplant
Eligibility:
People aged 13 or older who are eligible for a blood stem cell transplant to treat SCD. Healthy family members over age 13 who are matched to transplant recipients are also needed to donate blood.
Design:
Participants receiving transplants will undergo screening. They will have blood drawn. They will have tests of their breathing and heart function. They may have chest x-rays. A sample of marrow will be collected from a pelvic bone.
Participants will remain in the hospital about 30 days for the transplant and recovery. They will have a large intravenous line inserted into the upper arm or chest. The line will remain in place for the entire transplant and recovery period. The line will be used to draw blood as needed. It will also be used to administer the transplant stem cells as well as various drugs and blood transfusions. Participants will also receive some drugs by mouth.
Participants must remain within 1 hour of the NIH for 3 months after transplant. During that time, they will visit the clinic up to 2 times a week.
Follow-up visits will include tests to evaluate participants mental functions. They will have MRI scans of their brain and heart.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:4 - 100
Sex:All
40 Participants Needed
Ruxolitinib + Panobinostat for Myelofibrosis
Toronto, OntarioThis is a long term safety study for patients that have been treated with either ruxolitinib or a combination of ruxolitinib with panobinostat, on a Novartis or Incyte sponsored study, who have been judged by the study Investigator to benefit from ongoing treatment.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 4
Age:1 - 100
Sex:All
279 Participants Needed
Fostamatinib for Sickle Cell Disease
Bethesda, MarylandThis trial tests the safety and tolerability of fostamatinib, a drug taken by mouth, in people aged 18 to 65 with stable sickle cell disease. Fostamatinib aims to reduce inflammation and abnormal blood cell behavior by inhibiting a specific protein. Participants will take the drug for several weeks and have frequent check-ups to monitor side effects and effectiveness.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 65
Sex:All
Key Eligibility Criteria
Disqualifiers:Pain Crisis, Hypertension, Hepatitis, Others
Must Be Taking:Hydroxyurea
25 Participants Needed
Stem Cell Transplant for Sickle Cell Anemia
Bethesda, MarylandThis trial tests a new bone marrow transplant method for adults with sickle cell disease and Beta-thalassemia. It uses stem cells from a half-matched family donor, low-dose radiation, and immunosuppressant drugs, but no chemotherapy. The goal is to reduce complications and improve outcomes for patients who lack fully matched donors.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:2 - 80
Sex:All
23 Participants Needed
Low-Dose Radiation + Immunosuppressants for Sickle Cell Disease
Bethesda, MarylandPeople with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have been cured with bone marrow transplantation (BMT). The procedure, however, is limited to children younger than the age of 16 because the risks are lower for children than for adults.
The purpose of this study is to explore the use of a BMT regimen that, instead of chemotherapy, uses a low dose of radiation, combined with two immunosuppressive drugs. This type BMT procedure is described as nonmyeloablative, meaning that it does not destroy the patient s bone marrow. It is hoped that this type of BMT will be safe for patients normally excluded from the procedure because of their age and other reasons.
To participate in this study, patients must be between the ages of 18 and 65 and have a sibling who is a well-matched stem-cell donor. Beyond the standard BMT protocol, study participants will undergo additional procedures. The donor will receive G-CSF by injection for five days; then his or her stem cells will be collected and frozen one month prior to BMT. Approximately one month later, the patient will be given two immune-suppressing drugs, Campath 1-H and Sirolimus, as well as a single low dose of total body irradiation and then the cells from the donor will be infused.
Prior to their participation in this study, patients will undergo the following evaluations: a physical exam, blood work, breathing tests, heart-function tests, chest and sinus x-rays, and bone-marrow sampling.
...
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:2 - 80
Sex:All
130 Participants Needed
Non-Myeloablative Bone Marrow Transplant for Sickle Cell Disease
Nashville, TennesseeAllogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia. Even when it is clear that alloBMT can give to these patients an improvement in their disease, myeloablative transplants have important toxicities and mortalities associated. The lack of suitable donors continues to be a limit to access to transplantation. Substantial progress has been made recently in the development of pre-treatment regimens that facilitate the sustained engraftment of donor marrow with reduced toxicity. Most of these regimens incorporate highly immunosuppressive drugs, which allow the reduction or elimination of myeloablative agents or total body irradiation without endangering the sustained engraftment of HLA-identical allogeneic stem cells. Preliminary results of non-myeloablative allogeneic stem cell transplantation suggest that the procedure can be performed in patients who are ineligible for myeloablative alloBMT, and that sustained remissions of several hematologic malignancies can be obtained.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:1 - 70
Sex:All
26 Participants Needed
CTX001 for Sickle Cell Disease and Thalassemia
Nashville, TennesseeThis trial tests a new treatment where a patient's own blood stem cells are modified to fix faulty genes. It targets patients with severe blood disorders who need frequent transfusions. The goal is to help their bodies produce healthy blood cells. Recent advances in treatment methods expand the potentially curative options for patients.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12 - 35
Sex:All
26 Participants Needed
Stem Cell Transplant with TCRab Depletion for Sickle Cell Disease and Beta Thalassemia
Philadelphia, PennsylvaniaThis is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:2 - 25
Sex:All
9 Participants Needed
Gene Therapy for Beta Thalassemia
Philadelphia, PennsylvaniaThe main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells. This is done by collecting blood stem cells from the subject, modifying those cells, adding a healthy beta globin gene, and then giving them back to the subject. It is hoped that these modified cells will decrease the need for blood transfusions. The gene modified blood stem cells are called CHOP-ALS20 ("study drug"). This experimental gene therapy has not been tried on human beings before and is not FDA approved.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 35
Sex:All
Key Eligibility Criteria
Disqualifiers:Malignancy, HIV, Hepatitis, Cardiac Dysfunction, Others
12 Participants Needed
Immune Suppression Treatment for Sickle Cell Disease
Basking Ridge, New JerseyHematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:2 - 50
Sex:All
Key Eligibility Criteria
Disqualifiers:HIV, Active Hepatitis B Or C, Others
24 Participants Needed
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Frequently Asked Questions
How much do Thalassemia 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 Thalassemia 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 Thalassemia 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 Thalassemia 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 Thalassemia 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 Thalassemia clinical trials ?
Most recently, we added Hemanext ONE System for Sickle Cell Anemia, Gene Therapy for Beta Thalassemia and Gene Therapy for Sickle Cell Anemia to the Power online platform.