RM-004 cell therapy for Hemoglobin H-Constant Spring disease
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This early-phase trial tests a new cell therapy called RM-004 for people with Hemoglobin H-Constant Spring disease, a form of alpha thalassemia that requires regular blood transfusions. The goal is to see if the treatment is safe and can reduce or eliminate the need for transfusions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 12 and 35 years old.
- In the last 2 years, you have needed at least 100 mL of packed red blood cells per kilogram of your body weight each year (this is a measure of how much transfusion support you need).
- You have Hemoglobin H-Constant Spring disease (a specific type of alpha thalassemia), confirmed by genetic testing showing the HBA2 c.427T>C mutation.
- You do not have a fully matched stem cell donor available for a transplant.
- You have not had a stem cell transplant or gene therapy before.
- You do not have a history of serious bleeding problems.
- You do not have a serious active infection (like a bacterial, viral, fungal, or parasitic infection) that needs treatment.
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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