Janus Kinase (JAK) Inhibitor
Ruxolitinib + Panobinostat for Myelofibrosis
Recruiting3 awardsPhase 4
Toronto, Ontario
This trial is looking at the safety of long-term treatment with ruxolitinib alone or in combination with panobinostat in patients who have previously participated in a study sponsored by Novart
Gene Therapy
EDIT-301 for Beta Thalassemia
Recruiting1 awardPhase 1 & 2
Nashville, Tennessee
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.
Protein Therapeutics
Luspatercept for Thalassemia
Recruiting1 awardPhase 2
Los Angeles, California
This trial is testing a drug called luspatercept in children with β-thalassemia to see if it is safe and how it works in their bodies. The study includes children who need regular blood transfusions and those who do not. Luspatercept helps the body make more red blood cells, which can lessen the need for transfusions. Luspatercept is a newly approved treatment for reducing the need for blood transfusions in adults.
Popular Filters
Trials for Beta Thalassemia Patients
Procedure
Biotin Labeling for Sickle Cell Anemia
Recruiting1 awardPhase < 1
Bethesda, Maryland
"This trial aims to determine the lifespan of red blood cells in patients with sickle cell disease and other blood disorders, before and after receiving treatments such as stem cell transplantation. The study will use a labeling
Corticosteroid
Immune Suppression Treatment for Sickle Cell Disease
Recruiting1 awardPhase 2
Basking Ridge, New Jersey
This trial is studying a new way of doing an HCT: using drugs to lower the activity of the body's immune system before transplanting healthy stem cells to replace diseased or damaged cells.
Gene Therapy
CRISPR Therapy (CTX001) for Thalassemia
Recruiting2 awardsPhase 3
Nashville, Tennessee
This trial will test a new way to treat a rare disease called transfusion-dependent thalassemia (TDT). The treatment involves using a person's own stem cells that have been modified using CRISPR-Cas9. The goal is to see if this is a safe and effective treatment for TDT.
Small Molecule
Etavopivat for Sickle Cell Disease
Recruiting1 awardPhase 2
Whittier, California
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.
Activator
AG-348 for Thalassemia
Recruiting1 awardPhase 2
Oakland, California
This trial is testing a new drug, AG-348, for adults with non-transfusion-dependent thalassemia, a genetic blood disorder. The trial includes a core period of 24 weeks followed by an extension period of up to 10 years for eligible participants.
Gene Therapy
CRISPR-Cas9 Gene Editing for Beta Thalassemia
Recruiting1 awardPhase 2 & 3
Stanford, California
This trial is testing a new treatment for people with a blood disorder called transfusion-dependent β-thalassemia. The treatment involves using CRISPR-Cas9 to modify the patient's own blood cells. The trial will evaluate the safety and efficacy of this treatment.
Trials for Sickle Cell Disease Patients
Procedure
Biotin Labeling for Sickle Cell Anemia
Recruiting1 awardPhase < 1
Bethesda, Maryland
"This trial aims to determine the lifespan of red blood cells in patients with sickle cell disease and other blood disorders, before and after receiving treatments such as stem cell transplantation. The study will use a labeling
Corticosteroid
Immune Suppression Treatment for Sickle Cell Disease
Recruiting1 awardPhase 2
Basking Ridge, New Jersey
This trial is studying a new way of doing an HCT: using drugs to lower the activity of the body's immune system before transplanting healthy stem cells to replace diseased or damaged cells.
Small Molecule
Etavopivat for Sickle Cell Disease
Recruiting1 awardPhase 2
Whittier, California
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.
Immunosuppressive Agent
Nonmyeloablative Stem Cell Transplant for Sickle Cell Anemia and Thalassemia
Recruiting1 awardPhase 2
Bethesda, Maryland
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.
Stem Cell Transplant
Bone Marrow/Kidney Transplant for Blood Disorders & Chronic Kidney Disease
Recruiting1 award11 criteria
Boston, Massachusetts
This trial is for a combined bone marrow and kidney transplant from a partially matched related donor to treat a blood disorder and kidney disease. The expectation is to have 10 people participate.
Phase 3 Trials
Gene Therapy
CRISPR Therapy (CTX001) for Thalassemia
Recruiting2 awardsPhase 3
Nashville, Tennessee
This trial will test a new way to treat a rare disease called transfusion-dependent thalassemia (TDT). The treatment involves using a person's own stem cells that have been modified using CRISPR-Cas9. The goal is to see if this is a safe and effective treatment for TDT.
Gene Therapy
CRISPR-Cas9 Gene Editing for Beta Thalassemia
Recruiting1 awardPhase 2 & 3
Stanford, California
This trial is testing a new treatment for people with a blood disorder called transfusion-dependent β-thalassemia. The treatment involves using CRISPR-Cas9 to modify the patient's own blood cells. The trial will evaluate the safety and efficacy of this treatment.
Activator of Pyruvate Kinase-R
Mitapivat for Thalassemia
Recruiting1 awardPhase 3
Boston, Massachusetts
This 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.
Protein Therapy
Long-Term Safety of Luspatercept for Blood Disorders
Recruiting2 awardsPhase 3
Los Angeles, California
This trial is for people who have been taking luspatercept and are tolerate the protocol-prescribed regimen. The study is to evaluate the long-term safety of luspatercept and participants will be followed for safety-related parameters and adverse event reporting.
Trials With No Placebo
Procedure
Biotin Labeling for Sickle Cell Anemia
Recruiting1 awardPhase < 1
Bethesda, Maryland
"This trial aims to determine the lifespan of red blood cells in patients with sickle cell disease and other blood disorders, before and after receiving treatments such as stem cell transplantation. The study will use a labeling
Corticosteroid
Immune Suppression Treatment for Sickle Cell Disease
Recruiting1 awardPhase 2
Basking Ridge, New Jersey
This trial is studying a new way of doing an HCT: using drugs to lower the activity of the body's immune system before transplanting healthy stem cells to replace diseased or damaged cells.
Gene Therapy
CRISPR Therapy (CTX001) for Thalassemia
Recruiting2 awardsPhase 3
Nashville, Tennessee
This trial will test a new way to treat a rare disease called transfusion-dependent thalassemia (TDT). The treatment involves using a person's own stem cells that have been modified using CRISPR-Cas9. The goal is to see if this is a safe and effective treatment for TDT.
Small Molecule
Etavopivat for Sickle Cell Disease
Recruiting1 awardPhase 2
Whittier, California
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.
Activator
AG-348 for Thalassemia
Recruiting1 awardPhase 2
Oakland, California
This trial is testing a new drug, AG-348, for adults with non-transfusion-dependent thalassemia, a genetic blood disorder. The trial includes a core period of 24 weeks followed by an extension period of up to 10 years for eligible participants.
Gene Therapy
CRISPR-Cas9 Gene Editing for Beta Thalassemia
Recruiting1 awardPhase 2 & 3
Stanford, California
This trial is testing a new treatment for people with a blood disorder called transfusion-dependent β-thalassemia. The treatment involves using CRISPR-Cas9 to modify the patient's own blood cells. The trial will evaluate the safety and efficacy of this treatment.
Frequently Asked Questions
Do I need insurance to participate in a trial?
Almost all clinical trials will cover the cost of the 'trial drug' — so no insurance is required for this. For trials where this trial drug is given alongside an already-approved medication, there may be a cost (which your insurance would normally cover).
Is there any support for travel costs?
Many of the teams running clinical trials will cover the cost of transportation to-and-from their care center.
Will I know what medication I am taking?
This depends on the specific study. If you're worried about receiving a placebo, you can actively filter out these trials using our search.
How long do clinical trials last?
Some trials will only require a single visit, while others will continue until your disease returns. It's fairly common for a trial to last somewhere between 1 and 6 months.
Do you verify all the trials on your website?
All of the trials listed on Power have been formally registered with the US Food and Drug Administration. Beyond this, some trials on Power have been formally 'verified' if the team behind the trial has completed an additional level of verification with our team.
How quickly will I hear back from a clinical trial?
Sadly, this response time can take anywhere from 6 hours to 2 weeks. We're working hard to speed up how quickly you hear back — in general, verified trials respond to patients within a few days.