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โ† Trials
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Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

RecruitingEARLY Phase 1Ages 12 Years - 35 YearsAllThe 923rd Hospital of Joint Logistics Support Forโ€ฆ
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In plain words

What the trial is, and whether it could suit you

The purpose of this study is to evaluate the safety and efficacy of RM-004 for Hemoglobin H-Constant Spring disease.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: 1. Subjects voluntarily sign informed consent by themselves or their legal guardians and complete the study procedures, follow-up examination and treatment. 2. At the time of screening, subjects should be aged from 12 to 35 years old, regardless of gender. 3. History of at least 100 mL/kilograms (kg)/year of packed red blood cells (pRBC) transfusions in the prior 2 years before screening. 4. Subjects diagnosed with Hemoglobin H-Constant Spring disease (--/ฮฑฮฑCS) with HBA2 c.427T\>C mutation. Exclusion Criteria: 1. Subject who has an available HLA-matched/well-matched HSCT donor for allogeneic hematopoietic stem cell transplantation (HSCT). 2. Prior HSCT or gene therapy. 3. History of severe hemorrhagic disease. 4. Clinically significant active bacterial, viral, fungal or parasitic infections per investigator's judgement at the time of screening.
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What it tests

  • RM-004
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Where

1 site
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Nanning, China
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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