Testing APR-2020 for RPS19-deficient Diamond-Blackfan Anemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial is testing a new treatment called APR-2020 for children and young adults with a specific type of Diamond-Blackfan Anemia (caused by a change in the RPS19 gene). The goal is to see if this treatment is safe, tolerable, and can help reduce the need for blood transfusions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of Diamond-Blackfan Anemia with a specific gene change called RPS19.
- You are between 2 and 25 years old.
- Steroid treatments have not worked for you, and you need regular blood transfusions.
- You are eligible for a stem cell transplant but do not have a matching brother or sister who can donate.
- You must be willing to come back for long-term follow-up visits.
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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