Study to Evaluate the Safety and Efficacy of RM-004 Cells for the Treatment of Hemoglobin H-Constant Spring Disease
Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Early Phase 1
SUMMARY
The purpose of this study is to evaluate the safety and efficacy of RM-004 for Hemoglobin H-Constant Spring disease.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 35
Healthy Volunteers: f
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• 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.
Locations
Other Locations
China
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
RECRUITING
Nanning
Contact Information
Primary
Xinhua Zhang, MD
zxh303@163.com
+8613321717386
Time Frame
Start Date: 2023-10-08
Estimated Completion Date: 2026-10-31
Participants
Target number of participants: 5
Treatments
Experimental: RM-004
RM-004 (ex vivo gene-edited CD34+ autologous hematopoietic stem cells) will be administered as a single intravenous infusion
Related Therapeutic Areas
Sponsors
Leads: The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Collaborators: Guangzhou Reforgene Medicine Co., Ltd.