Registry for people affected by alpha thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This is a research registry collecting information about people with alpha thalassemia to support ongoing studies. It may help the team better understand how fetal stem cell treatments are working and for whom they may be most suitable.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a diagnosis of alpha thalassemia, during pregnancy (prenatal) or after birth (postnatal).
- Your genetic test results (genotype) match what they consider a specific alpha thalassemia pattern (ATM or BHFS).
- You have been referred to UCSF’s Fetal Treatment Center for fetal diagnosis, care, and/or evaluation related to an in-utero stem cell transplant study.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial looks at the genetic profiles of children with suspected alpha thalassemia, a type of inherited anemia. It aims to better understand this condition in children treated at Sohag University Hospital.
This study is for people with beta-thalassemia who have already received the gene therapy betibeglogene autotemcel (beti-cel). It collects information to learn how patients are doing after this treatment in real-world settings.
This early-phase trial studies HGI-002 to see if it’s safe and can help people with severe alpha thalassemia who need frequent blood transfusions. It also requires planning for a special treatment approach that includes busulfan pre-treatment and stem cell transplantation.
This Phase 1 study tests a new medicine (GMCN-508A) to see how safe it is and whether it can help people with transfusion-dependent alpha thalassemia. Participants are also expected to follow a treatment plan that includes stem cell transplantation preparation.
This early-phase trial tests a new way to restore working blood-making cells using your own stem cells for people with transfusion-dependent beta thalassemia major. The goal is to improve safety and blood results, and possibly reduce problems from the disease.
This trial tests whether giving you your own (autologous) gene-modified blood stem cells can improve transfusion-dependent beta-thalassemia and its safety. It may help people who do not have a fully matched donor and need an alternative approach to care.
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