Clin2
NCT05776173Possibly a fitRecruiting

Gene-modified stem cell treatment for transfusion-dependent thalassemia

β-thalassemia Major

Part of Blood & lymphatic, Genetic & congenital clinical trials.

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.

Summary written for real people, not researchers, by Clin2.

Phase
N/A
Enrollment
10 people
Ages
6 years to 35 years
Study type
Interventional

Who can take part

  • You are age 6 to 35, and you can sign informed consent.
  • You have confirmed transfusion-dependent beta-thalassemia (you need regular red blood cell transfusions).
  • You must be able to keep taking your current iron chelation medicine on schedule.
  • Your blood/marrow genetic and leukemia screening (gene panel) must not show certain cancer-related findings.
  • You cannot have a fully HLA-matched donor available for transplant (unless the monitoring team says it’s okay).
  • You must not have major infections, serious organ problems, low blood counts, or certain past treatments.

View the official record on ClinicalTrials.gov

Quick eligibility check

Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.

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