NCT06107400

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

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army · Academic / Other
• Sex: All
• Age: 12 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Alpha Thalassemia Hemoglobin H Constant Spring
• Hemoglobinopathies
• Hereditary Diseases

Interventions

Type	Name	Description
GENETIC	RM-004	Intravenous infusion of gene-edited CD34+autologous hematopoietic stem cells (RM-004) after myeloablative conditioning with busulfan.

Timeline

• Start date: 2023-10-08
• Primary completion: 2025-10-31
• Completion: 2026-10-31
• First posted: 2023-10-30
• Last updated: 2024-06-03

Locations

1 site across 1 country: China