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

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

Status and phase: Enrolling; Early Phase 1

Conditions: Hereditary Diseases; Hemoglobinopathies; Alpha Thalassemia Hemoglobin H Constant Spring

Treatments: Genetic: RM-004

Study type: Interventional

Funder types: Other

Identifiers

NCT06107400

IIT-RM-004-01

Details and patient eligibility

About

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

Full 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-Constant Spring disease. Five subjects aged from 12 to 35 years will be recruited in this study.

Enrollment: 5 estimated patients

Sex: All

Ages: 12 to 35 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

RM-004

Experimental group

Description:

RM-004 (ex vivo gene-edited CD34+ autologous hematopoietic stem cells) will be administered as a single intravenous infusion

Treatment:

Genetic: RM-004