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28 Hemoglobinopathies Trials Near You
Power is an online platform that helps thousands of Hemoglobinopathies 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 PowerEDIT-301 for Sickle Cell Disease
Columbus, Ohio
This trial is testing a new treatment called EDIT-301 for people aged 12 to 50 with severe sickle cell disease. The treatment uses the patient's own stem cells, which are changed in a lab and then put back into their body to help fight the disease.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:12 - 50
Key Eligibility Criteria
Disqualifiers:HLA-matched Donor, Prior HSCT, Vasculopathy, Malignancy, Others
Must Not Be Taking:Hydroxyurea, Voxelotor, Crizanlizumab, L-glutamine
45 Participants Needed
Etavopivat for Sickle Cell Disease
Columbus, Ohio
This trial is testing a drug called etavopivat to see if it can help people with sickle cell disease. The drug aims to improve blood health and reduce pain by making red blood cells work better. The study includes patients aged 12 to 65.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12 - 65
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis B/C, Renal Dysfunction, Others
Must Not Be Taking:Voxelotor, Erythropoietin, CYP3A4/5 Inducers
450 Participants Needed
Etavopivat for Sickle Cell Disease
Cincinnati, Ohio
This 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
Key Eligibility Criteria
Disqualifiers:Hepatic Dysfunction, HIV, Hepatitis B/C, Others
60 Participants Needed
EDIT-301 for Beta Thalassemia
Cleveland, Ohio
This 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
Key Eligibility Criteria
Disqualifiers:Alpha Thalassemia, Hemoglobinopathy, Malignancy, Others
9 Participants Needed
Transplant Conditioning Regimen for Blood Disorders
Indianapolis, Indiana
The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells. It is also to determine major toxicities from these conditioning regimens, within the first 100 days after transplantation.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:< 20
Key Eligibility Criteria
Disqualifiers:HIV, Invasive Infection, Pregnancy, Others
220 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
CTX001 for Sickle Cell Disease
Chicago, Illinois
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). 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 (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:12 - 35
Key Eligibility Criteria
Disqualifiers:Prior HSCT, Active Infection, Others
63 Participants Needed
CRISPR-Cas9 Gene Editing for Beta Thalassemia
Chicago, Illinois
This 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 (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:12 - 35
Key Eligibility Criteria
Disqualifiers:Prior Allo-HSCT, Sickle Cell, Others
59 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 (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:2 - 11
Key Eligibility Criteria
Disqualifiers:Prior HSCT, Sickle Cell Variant, Others
15 Participants Needed
Biotin Labeling for Sickle Cell Anemia
Bethesda, Maryland
Background:
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
Key Eligibility Criteria
Disqualifiers:Biotin Supplements, Chronic Transfusion Therapy, Hemodialysis, Malignancy, Others
100 Participants Needed
Gene Modified T-cells for Blood Disorders
Washington, District of Columbia
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (graft versus host disease).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:1 - 26
Key Eligibility Criteria
Disqualifiers:Severe Cardiovascular, Active Infections, Others
Must Not Be Taking:Immunosuppressants
120 Participants Needed
CRISPR-Cas9 Modified Stem Cells for Sickle Cell Disease
Nashville, Tennessee
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. 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 (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:2 - 11
Key Eligibility Criteria
Disqualifiers:HLA-matched Donor, Prior HSCT, Infections, Others
Must Be Taking:Hydroxyurea
15 Participants Needed
CTX001 for Sickle Cell Disease and Thalassemia
Nashville, Tennessee
This 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 (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12 - 35
Key Eligibility Criteria
Disqualifiers:Prior HSCT, Active Infection, Others
26 Participants Needed
Non-Myeloablative Bone Marrow Transplant for Sickle Cell Disease
Nashville, Tennessee
Allogeneic 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
Age:1 - 70
Key Eligibility Criteria
Disqualifiers:Not Listed
26 Participants Needed
RIC Regimen for Nonmalignant Diseases
Philadelphia, Pennsylvania
This is a Phase II pilot study to evaluate engraftment and toxicity of patients with non-malignant diseases using a reduced intensity conditioning regimen in the setting of allogeneic transplant for non malignant diseases. Bone Marrow or cord blood will be acceptable as a stem cell source.
Recently, reduced intensity conditioning (RIC) regimens have been used for both adult patients with leukemias and pediatric patients with non-malignant diseases. These regimens are better tolerated, resulting in less transplant related morbidity and mortality. Stable mixed chimerism, while insufficient for eradication of leukemias, may be sufficient to cure patients with non-malignant diseases.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:6 - 25
Key Eligibility Criteria
Disqualifiers:Uncontrolled Infections, Others
75 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
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
Stem Cell Transplants for Sickle Cell Disease and Thalassemia
Minneapolis, Minnesota
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 55
Key Eligibility Criteria
Disqualifiers:Pregnancy, HIV, Uncontrolled Infection, Others
62 Participants Needed
Why Other Patients Applied
"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
"As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."
IZ
Healthy Volunteer PatientAge: 38
"I 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 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
Reduced Intensity Stem Cell Transplant for Blood Disorders
Minneapolis, Minnesota
This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) in patients with non-malignant or malignant diseases. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HCT.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:< 55
Key Eligibility Criteria
Disqualifiers:Previous Irradiation, Pregnant, HIV Positive, Others
30 Participants Needed
Stem Cell Transplant for Blood Disorders
Minneapolis, Minnesota
This trial is studying how well stem cell transplants work for patients with blood diseases that are not cancer. The treatment involves giving patients healthy stem cells from a donor to replace their damaged or diseased cells. This method is the oldest and most widely used technique of stem cell transplant, primarily used to treat blood disorders.
No Placebo Group
Trial Details
Trial Status:Recruiting
Age:< 55
Key Eligibility Criteria
Disqualifiers:Not Listed
25 Participants Needed
Gene Therapy for Sickle Cell Anemia
Boston, Massachusetts
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace a diseased gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of Graft-Versus-Host Disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to fix or replace a diseased gene is called gene editing. A person's own cells are edited using a specialized biological medicine that has been formulated for use in human beings.
Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. Investigators have recently discovered a gene called BCL11A that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition.
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 1
Age:13 - 40
Key Eligibility Criteria
Disqualifiers:Uncontrolled Infection, Active Malignancy, Major Surgery, Others
10 Participants Needed
Haploidentical Stem Cell Transplant for Blood Diseases
Saint Petersburg, Florida
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:0 - 21
Key Eligibility Criteria
Disqualifiers:Pregnancy, HIV, Active Infections, Others
17 Participants Needed
Stem Cell Transplant for Leukemia
Houston, Texas
This trial tests a special stem cell transplant for patients with serious conditions. It removes harmful immune cells from the donor's blood to lower the risk of complications, potentially reducing the need for strong medications afterward.
No Placebo Group
Trial Details
Trial Status:Recruiting
Age:< 55
Key Eligibility Criteria
Disqualifiers:Not Listed
47 Participants Needed
Etavopivat for Sickle Cell Disease
Salt Lake City, Utah
The study aims to test if a new medicine called etavopivat potentially affects other medicines in healthy participants. The purpose of the study is to investigate whether the use of etavopivat affects the breakdown and metabolism of commonly used medicines in the body. During the study, participants will receive etavopivat and five different medicines that are already approved and available on the market, and which can be prescribed by doctors. These marketed medicines are called substrate drugs and they are: digoxin, pitavastatin, metformin, midazolam, and rosuvastatin. During a period of the study, participants will take 2 tablets of etavopivat daily for 10 consecutive days. The study will last for about 34 to 64 days.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 55
Key Eligibility Criteria
Disqualifiers:Pregnancy, Hypersensitivity, Recent Investigational Drug, Others
Must Not Be Taking:Moderate/strong Enzyme Inhibitors/inducers
29 Participants Needed
Stem Cell Transplant for Sickle Cell Disease
Duarte, California
Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot).
Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism.
Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor).
Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications.
This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because:
1. Half-matched related donors will be used, and
2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and
3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes.
It is hoped that the research transplant:
1. Will reverse sickle cell disease and improve patient quality of life,
2. Will reduce side effects and help the patient recover faster from the transplant,
3. Help the patient keep the transplant longer and
4. Reduce serious transplant-related complications.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:18 - 45
Key Eligibility Criteria
Disqualifiers:Prior Transplants, Active Infection, Malignancy, Others
Must Not Be Taking:CYP3A4 Inhibitors
3 Participants Needed
Oral Carbon Monoxide Therapy for Sickle Cell Anemia
San Diego, California
This is a multi-center, open label Phase 2a clinical trial in subjects with sickle cell disease to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with doses daily for 14 days.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 55
Key Eligibility Criteria
Disqualifiers:Cardiac Disease, Renal Dysfunction, Cancer, Others
Must Be Taking:Hydroxyurea
9 Participants Needed
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We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.
Bask GillCEO at Power
Frequently Asked Questions
How much do Hemoglobinopathies 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 Hemoglobinopathies 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 Hemoglobinopathies 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 Hemoglobinopathies 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 Hemoglobinopathies 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 Hemoglobinopathies clinical trials?
Most recently, we added Gene Therapy for Sickle Cell Anemia, CD45RA Depleted Stem Cell Addback for Leukemia and Oral Carbon Monoxide Therapy for Sickle Cell Anemia to the Power online platform.
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