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

Recruiting now · Early Phase 1

Conditions studied: Alpha Thalassemia Hemoglobin H Constant Spring, Hemoglobinopathies, Hereditary Diseases

In brief

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

Key facts

Study ID

NCT06107400

Run by

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

People needed

5

Starts

2023-10-08

Expected to finish

2026-10-31

Last updated by the study team

2024-06-03

Who can join

Age: 12 and older, up to 35. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army — Nanning, Guangxi, China (enrolling)

Full record on ClinicalTrials.gov

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