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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)


NCTID NCT04783181 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Congenital Adrenal Hyperplasia
Disease Ontology Term DOID:0050811
Compound Name BBP-631
Compound Description AAV5-CYP21A2
Sponsor Adrenas Therapeutics Inc
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 8
Results Posted Not Available

Therapy Information


Target Gene/Variant CYP21A2
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 AAV5
Editor Type none
Dose 1 1.5E13 vg/kg
Dose 2 3.0E13 vg/kg
Dose 3 6.0E13 vg/kg
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2021-03-01
Completion Date 2029-02
Last Update 2024-09-26

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 5
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates Bridge Bio is cutting this program, lack of sufficient efficacy

Resources/Links