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Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease | Clinical Trials | Clareo Health

RECRUITINGEARLY_PHASE1

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

NCT06107400•The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

View on ClinicalTrials.gov

Summary

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.

Eligibility

Age

12 - 35 years

Sex

ALL

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.

Locations (1)

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

Nanning, Guangxi, China

Key Dates

Start Date

October 8, 2023

Primary Completion Date

October 31, 2025

Completion Date

October 31, 2026

Last Updated

June 3, 2024

Enrollment

ESTIMATED participants

Conditions

Alpha Thalassemia Hemoglobin H Constant SpringHemoglobinopathiesHereditary Diseases

Interventions

RM-004

GENETIC

Contacts

Xinhua Zhang, MD

CONTACT

+8613321717386 zxh303@163.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 3, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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