Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

Launched by THE 923RD HOSPITAL OF JOINT LOGISTICS SUPPORT FORCE OF PEOPLE'S LIBERATION ARMY · Oct 25, 2023

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ClinConnect Summary

This clinical trial is studying a new treatment called RM-004 for patients with Hemoglobin H-Constant Spring disease, a type of genetic blood disorder that affects how hemoglobin, the protein that carries oxygen in the blood, works. The main goal of the study is to find out if RM-004 is safe and effective for people with this condition. The trial is currently looking for participants aged 12 to 35 years who have received a lot of blood transfusions in the past two years and meet specific genetic criteria related to their condition.

If you or someone you know is interested in participating, you would need to sign consent forms and complete a series of study procedures and follow-up examinations. However, there are some important restrictions: if you have had certain other treatments like stem cell transplants or gene therapy, or if you have specific health issues, you may not be eligible. This trial could provide a new option for those who have struggled with the limitations of their current treatment.

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Eligibility criteria

• 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.