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Gene Therapy Trial Browser
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
This study is designed to evaluate the safety, tolerability, and efficacy of AAV5 based BBP-631 in adult participants diagnosed with classic congenital adrenal hyperplasia.
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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
News and Press Releases
BridgeBio Pharma Reports Topline Results from Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH)
BridgeBio Pharma Drops Development of Congenital Adrenal Hyperplasia Gene Therapy BBP-631
SEC Form 10-K: 2024 Annual Report
Preclinical Publications
OR25-01 Durable CYP21A2 Gene Therapy in Non-Human Primates for Treatment of Congenital Adrenal Hyperplasia
(Presentation) Intravenous AAV5 Gene Therapy with Human CYP21A2 Corrects Phenotypic Deficiencies of the 21-hydroxylase Knockout Mouse Model and Demonstrates Durability and Safety in Non-Human Primates and Mice. - ASGCT 2021
Protocol
(Presentation) Clinical Trial Design - Endocrinology 2021