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

Last updated: May 30, 2024
Sponsor: The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Overall Status: Active - Recruiting

Phase

1

Condition

Thalassemia

Birth Defects

Red Blood Cell Disorders

Treatment

RM-004

Clinical Study ID

NCT06107400
IIT-RM-004-01
  • Ages 12-35
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Subjects voluntarily sign informed consent by themselves or their legal guardiansand 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 HBA2c.427T>C mutation.

Exclusion

Exclusion Criteria:

  1. Subject who has an available HLA-matched/well-matched HSCT donor for allogeneichematopoietic 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 perinvestigator's judgement at the time of screening.

Study Design

Total Participants: 5
Treatment Group(s): 1
Primary Treatment: RM-004
Phase: 1
Study Start date:
October 08, 2023
Estimated Completion Date:
October 31, 2026

Study 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.

Connect with a study center

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

    Nanning, Guangxi
    China

    Active - Recruiting

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