Status:
RECRUITING
Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease
Lead Sponsor:
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Collaborating Sponsors:
Guangzhou Reforgene Medicine Co., Ltd.
Conditions:
Alpha Thalassemia Hemoglobin H Constant Spring
Hemoglobinopathies
Eligibility:
All Genders
12-35 years
Phase:
EARLY_PHASE1
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of RM-004 for Hemoglobin H-Constant Spring disease.
Detailed Description
This is a non-randomized, one-arm, open label study to evaluate the safety and efficacy of RM-004 for autologous hematopoietic stem cell transplantation (HSCT) for the treatment of Hemoglobin H-Consta...
Eligibility Criteria
Inclusion
- Subjects voluntarily sign informed consent by themselves or their legal guardians and complete the study procedures, follow-up examination and treatment.
- At the time of screening, subjects should be aged from 12 to 35 years old, regardless of gender.
- History of at least 100 mL/kilograms (kg)/year of packed red blood cells (pRBC) transfusions in the prior 2 years before screening.
- Subjects diagnosed with Hemoglobin H-Constant Spring disease (--/ααCS) with HBA2 c.427T\>C mutation.
Exclusion
- Subject who has an available HLA-matched/well-matched HSCT donor for allogeneic hematopoietic stem cell transplantation (HSCT).
- Prior HSCT or gene therapy.
- History of severe hemorrhagic disease.
- Clinically significant active bacterial, viral, fungal or parasitic infections per investigator's judgement at the time of screening.
Key Trial Info
Start Date :
October 8 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 31 2026
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT06107400
Start Date
October 8 2023
End Date
October 31 2026
Last Update
June 3 2024
Active Locations (1)
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1
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Nanning, Guangxi, China