RecruitingEarly Phase 1NCT06107400

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

Study to Evaluate the Safety and Efficacy of RM-004 Cells for the Treatment of Hemoglobin H-Constant Spring Disease

Sponsor

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

Enrollment

5 participants

Start Date

Oct 8, 2023

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 12 YearsMax Age: 35 Years

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.

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.

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GENETICRM-004

Intravenous infusion of gene-edited CD34+autologous hematopoietic stem cells (RM-004) after myeloablative conditioning with busulfan.

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

Nanning, Guangxi, China

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NCT06107400