Thalassemia Clinical Trials
Here are the 6 most popular medical studies for thalassemia
Procedure
Biotin-labeled red blood cells for Thalassemia
"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
Protein Therapeutics
Luspatercept for Thalassemia
This trial is studying luspatercept to see if it is safe and effective in treating thalassemia in children. The trial will be conducted in two parts with different age groups of children. If the treatment is working for a child, they will be offered the opportunity to keep taking luspatercept for up to five years.
Popular filter options for thalassemia trials
Sickle Cell Disease Clinical Trials
View 19 Sickle Cell Disease medical studies.
Gene Editing
CTX001 for Sickle Cell Disease and Thalassemia
This trial will test a new way to treat transfusion-dependent beta thalassemia or severe sickle cell disease, by using the patient's own modified stem cells. The goal is to see if this is safe and effective.
Small Molecule
Etavopivat for Sickle Cell Disease
This trial will evaluate the safety and clinical activity of a new drug, etavopivat, in patients with thalassemia or sickle cell disease. The goal is to see if the drug can lower the number of red blood cell transfusions required and increase hemoglobin.
Immunosuppressive Agent
Nonmyeloablative Stem Cell Transplant for Sickle Cell Anemia and Thalassemia
This trial is testing a new variation of stem cell transplant that uses low dose radiation and a combination of immunosuppressive drugs to see if it helps the body better accept donor stem cells.
Stem Cell Transplant
Bone Marrow/Kidney Transplant for Blood Disorders & Chronic Kidney Disease
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.
Beta Thalassemia Clinical Trials
View 18 Beta Thalassemia medical studies.
Gene Editing
CTX001 for Sickle Cell Disease and Thalassemia
This trial will test a new way to treat transfusion-dependent beta thalassemia or severe sickle cell disease, by using the patient's own modified stem cells. The goal is to see if this is safe and effective.
Gene Therapy
CRISPR Therapy (CTX001) for Thalassemia
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
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.
Corticosteroid
Immune Suppression Treatment for Sickle Cell Disease
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.
Phase 3 Thalassemia Clinical Trials
View 35 phase 3 thalassemia medical studies.
Gene Editing
CTX001 for Sickle Cell Disease and Thalassemia
This trial will test a new way to treat transfusion-dependent beta thalassemia or severe sickle cell disease, by using the patient's own modified stem cells. The goal is to see if this is safe and effective.
Gene Therapy
CRISPR Therapy (CTX001) for Thalassemia
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
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.
Protein Therapy
Long-Term Safety of Luspatercept for Blood Disorders
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.
Thalassemia Clinical Trials With No Placebo
View 35 thalassemia medical studies that do not have a placebo group.
Gene Editing
CTX001 for Sickle Cell Disease and Thalassemia
This trial will test a new way to treat transfusion-dependent beta thalassemia or severe sickle cell disease, by using the patient's own modified stem cells. The goal is to see if this is safe and effective.
Gene Therapy
CRISPR Therapy (CTX001) for Thalassemia
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
This trial will evaluate the safety and clinical activity of a new drug, etavopivat, in patients with thalassemia or sickle cell disease. The goal is to see if the drug can lower the number of red blood cell transfusions required and increase hemoglobin.
Activator
AG-348 for Thalassemia
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
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.
View More Thalassemia Trials
See another 13 medical studies focused on thalassemia.
Frequently Asked Questions
Introduction to thalassemia
What are the top hospitals conducting thalassemia research?
In the realm of thalassemia research, several top hospitals are spearheading clinical trials to advance our understanding and treatment of this complex genetic blood disorder. One such institution is Massachusetts General Hospital in Boston, where five active thalassemia trials are currently underway, building upon their history of six previous investigations since their inaugural study in 2004. In Philadelphia, the Children's Hospital of Philadelphia has been at the forefront as well, with three ongoing thalassemia trials and an impressive record of twenty completed studies dating back to 2000. Meanwhile, at the National Institutes of Health Clinical Center in Bethesda, Maryland researchers are actively engaged in three clinical trials for thalassemia and have conducted a total of six trials since embarking on their first study in 2014.
Furthermore adding itself to this list is Ann & Robert H Lurie Children's Hospital situated within Chicago; similar efforts being poured into it as well from having undergone studies tailored towards establishing diagnosis or developing treatments by engaging patients through around three concurrent tests alongside an additional set tallying up-to five summative experiments carried out until today starting from most recently recognized trial that dates back merely till 2018. Contributing its own valuable insights into combating thalassemia is Columbia University Medical Center located in New york City which presently hosts two active clinical assessments focusing specifically on this condition but not too far behind overall has participated hands-on conducting a countable number totaling up-to five all-time explorations ever recorded bearing significance across broader spectrum solely due to initiating earliest known investigation way back until just ten years ago commencing from year2011.
These leading medical centers serve as beacons of hope for individuals living with thalassemia and strive tirelessly towards finding novel approaches to better manage and ultimately cure this challenging disease. Through collaboration between dedicated doctors and brave participants willing to take part in these important studies, we move closer each day to unraveling the mysteries of thalassemia and offering effective treatments that can enhance the lives of those affected worldwide.
Which are the best cities for thalassemia clinical trials?
When it comes to thalassemia clinical trials, several cities are at the forefront of research and development. New york City leads with 15 active trials, focusing on promising treatments like Mitapivat and EDIT-301. Toronto follows closely with 11 ongoing studies investigating interventions such as Mitapivat and Luspatercept. Philadelphia and Oakland tie next with 8 active trials each, exploring options like Etavopivat tablets and AG-348. Finally, Boston also shows promise in thalassemia research with 7 active trials studying treatments including AG-348 and Haploidentical Bone Marrow/Kidney transplants. These cities offer individuals affected by thalassemia access to cutting-edge clinical trials that pave the way for advancements in care towards better outcomes.
Which are the top treatments for thalassemia being explored in clinical trials?
Thalassemia research is making significant strides, with several promising treatments currently being explored in clinical trials. CTX001 takes the lead, participating in two active trials and accumulating a total of four thalassemia trials since its introduction in 2018. Mitapivat follows closely behind, also involved in two ongoing trials and showing great potential since its debut in 2021. Additionally, etavopivat tablets are being investigated through one active trial as well as AG-348, both of which show promise after their initial listings in 2021 and 2019 respectively. These innovative therapies bring hope to individuals living with thalassemia worldwide as researchers continue to pave the way for better treatment options.
What are the most recent clinical trials for thalassemia?
Exciting developments are underway in the field of thalassemia research, as recent clinical trials offer promising avenues for treatment. One such trial involves participants with both sickle cell disease and β-thalassemia, aiming to assess their response to a potential therapeutic intervention. Additionally, CTX001 is being evaluated in a phase 3 trial as a potential treatment option for thalassemia patients. Another study explores the use of briquilimab in individuals who have undergone stem cell transplants for SCD-related thalassemia. Furthermore, EDIT-301 and abatacept are also being investigated in separate phase 1/2 trials for their efficacy in managing thalassemia symptoms. These groundbreaking initiatives hold immense potential to transform the lives of those affected by this condition.
What thalassemia clinical trials were recently completed?
In recent years, several clinical trials focusing on thalassemia have been successfully completed, bringing new insights and potential treatments to light. Notably, the University of Florida wrapped up a trial in June 2018 investigating the efficacy of Thiotepa--escalated dose. Additionally, Sangamo Therapeutics completed their trial on ST-400 Investigational product in March 2018. In February 2018, Celgene concluded a trial studying Luspatercept's potential benefits for patients with thalassemia. Lastly, bluebird bio's study on LentiGlobin BB305 Drug Product reached completion in June 2017. These milestones showcase continual efforts by researchers and pharmaceutical companies to enhance treatment options for individuals affected by thalassemia.