This Early Phase 1 study evaluates the safety and efficacy of the RM-004 cell product in people with Hemoglobin H-Constant Spring disease. Participants are aged 12 to 35 at screening, of any gender, and must give voluntary informed consent themselves or through a legal guardian and be able to complete the study procedures, follow-up examinations and treatment. The trial is currently recruiting.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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 (--/ααCS) 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.