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Clinical Trial Report

Gene Therapy Trial Report

Summary

Safety and Efficacy Study of PUMCH-E101 Injection in Subjects with RDH12 Retinopathy

NCTID NCT06749639 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Inherited Retinal Diseases, RDH12 Retinopathy
Disease Ontology Term DOID:0110330
Compound Name PUMCH-E101
Sponsor Peking Union Medical College Hospital
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 10
Results Posted Not Available

Therapy Information

Target Gene/Variant RDH12
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravitreal
Drug Product Type Undisclosed
Target Tissue/Cell
Delivery System Undisclosed
Vector Type undisclosed
Editor Type undisclosed
Dose 1 Undisclosed dose escalation, 2 levels
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Early phase1
Submit Date 2024-12-19
Completion Date 2030-01-04
Last Update 2024-12-31

Participation Criteria

Eligible Age 8 Years - 45 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

Resources/Links

Preclinical Publications