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Clinical Trial Report

Gene Therapy Trial Report

Summary

Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)


NCTID NCT06178432 (View at clinicaltrials.gov)
Description
Indication Glycogen Storage Disease Type 2 (Late-Onset Pompe Disease)
Compound Name BBM-G102
Sponsor Huashan Hospital
Funder Type Other
Status
Not yet recruiting
Enrollment Count 6

Therapy Information


Target Gene/Variant GAA
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV
Editor Type
Dose 1 Undisclosed dose 1
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Early phase1
Submit Date 2023-11-30
Completion Date 2028-12
Last Update 2023-12-21

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
Recent Updates

Resources/Links


Resources/Links

No External Links Available.