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Recruiting NCT06107400

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

Early Phase I Interventional Alpha Thalassemia Hemoglobin H Constant Spring Hemoglobinopathies Hereditary Diseases

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: RM-004.
Who it may be relevant to
Registry conditions: Alpha Thalassemia Hemoglobin H Constant Spring, Hemoglobinopathies, Hereditary Diseases. Basic parameters: 12 years — 35 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

Detailed 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.

Interventions

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

Primary outcome measures

  • Proportion of subjects with engraftment [Time frame: Within 42 days after RM-004 infusion]
  • Safety of RM-004 infusion [Time frame: From signing of informed consent up to 24 months after RM-004 infusion]
  • Proportion of subjects who achieve transfusion independence [Time frame: Up to 24 months after RM-004 infusion]
Secondary outcome measures (3)
  • Incidence of all-cause mortality [Time frame: From signing of informed consent up to 24 months after RM-004 infusion]
  • Proportion of subjects who stop receiveing transfusion ≥ 6 months [Time frame: Up to 24 months after RM-004 infusion]
  • Duration of transfusion independence [Time frame: Up to 24 months after RM-004 infusion]

Eligibility criteria

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army — Nanning

Identifiers

NCT: NCT06107400 · IIT-RM-004-01

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗