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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
Enrollment Count 5 (ESTIMATED)
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

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
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

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