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Clinical Trial Report

Gene Therapy Trial Report

Summary

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

NCTID NCT06107400 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Alpha Thalassemia Hemoglobin H Constant Spring
Disease Ontology Term DOID:0110031
Compound Name RM-004
Sponsor The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 5
Results Posted Not Available

Therapy Information

Target Gene/Variant (HBA2):c.427T>C (p.Ter143Gln)
Therapy Type Gene editing
Therapy Route Ex-vivo
Mechanism of Action Mutation correction
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Lipid encapsulation
Vector Type LNP
Editor Type SpRY-CBE
Dose 1 Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Early phase1
Submit Date 2023-10-25
Completion Date 2026-10-31
Last Update 2024-06-03

Participation Criteria

Eligible Age 12 Years - 35 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 1
Locations China

Regulatory Information

Has US IND False
FDA Designations
Recent Updates First patient dosed (5/27/24)

Resources/Links

News and Press Releases