BETA-THALASSEMIA
Clinical trials for BETA-THALASSEMIA explained in plain language.
Never miss a new study
Get alerted when new BETA-THALASSEMIA trials appear
Sign up with your email to follow new studies for BETA-THALASSEMIA, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
Gene editing aims to cure blood disorders in new trial
⭐️ CURE ⭐️ Recruiting nowThis study tests a gene therapy that edits a patient's own blood stem cells to produce more fetal hemoglobin, a healthy type that can replace the defective hemoglobin causing sickle cell disease or beta-thalassemia. The treatment involves a stem cell transplant using the patient'…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1 • Sponsor: Daniel Bauer • Aim: ⭐️ CURE ⭐️
Last updated May 14, 2026 12:02 UTC
-
CRISPR gene editing offers hope for blood disorder patients
Disease control Recruiting nowThis study tests a single dose of a CRISPR-modified stem cell therapy (CTX001) in 26 people with transfusion-dependent β-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, edited to produce fetal hemoglobin, aiming to reduce or elimi…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 17, 2026 02:58 UTC
-
Gene therapy for blood disorder: will patients stay Transfusion-Free?
Disease control Recruiting nowThis study follows 5 people with beta-thalassemia who already received a one-time gene therapy called CS-101. Researchers want to see if the treatment remains safe and effective over many years, including whether patients can stay free from blood transfusions. The study does not …
Matched conditions: BETA-THALASSEMIA
Phase: NA • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated May 17, 2026 02:56 UTC
-
Blood disorder drug luspatercept under Long-Term safety review
Disease control Recruiting nowThis study looks at the long-term safety of luspatercept in people with certain blood disorders (MDS, beta-thalassemia, or myelofibrosis) who were in earlier luspatercept trials. Participants either continue treatment or enter long-term follow-up to monitor side effects, disease …
Matched conditions: BETA-THALASSEMIA
Phase: PHASE3 • Sponsor: Celgene • Aim: Disease control
Last updated May 15, 2026 11:56 UTC
-
Gene therapy could free Beta-Thalassemia patients from lifelong transfusions
Disease control Recruiting nowThis study tests a new gene therapy for people with a severe form of beta-thalassemia who need frequent blood transfusions. The treatment involves taking the patient's own blood stem cells, adding a working gene to help them produce healthy hemoglobin, and returning them to the b…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Lantu Biopharma • Aim: Disease control
Last updated May 13, 2026 16:02 UTC
-
Could a One-Time gene fix free thalassemia patients from lifelong transfusions?
Disease control Recruiting nowThis study tests a new gene therapy for adults with transfusion-dependent beta-thalassemia, a severe blood disorder. Doctors take the patient's own blood stem cells, add a healthy gene to make normal hemoglobin, and return the cells. The goal is to reduce or stop the need for reg…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated May 04, 2026 16:20 UTC
-
New drug trial aims to help kids with blood disorder need fewer transfusions
Disease control Recruiting nowThis study is testing a drug called luspatercept in children aged 6 to 18 with beta-thalassemia, a genetic blood disorder. Researchers want to find the safest and most effective dose and see if it can help children who need regular blood transfusions require fewer of them, or hel…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE2 • Sponsor: Celgene • Aim: Disease control
Last updated Apr 06, 2026 14:14 UTC