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. (Show More)
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 (ESTIMATED)
Results Posted	Not Available

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
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

Eligible Age	>=18 Years
Standard Ages	Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Key Inclusion Criteria 1. Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD 2. Screening/baseline 17-OHP levels \> 5-10 × ULN and \< 40 × ULN (upper limit of normal) 3. Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy 4. Naïve to prior gene therapy or AAV-mediated therapy Key Exclusion Criteria 1. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies 2. History of adrenalectomy and/or significant liver disease View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	5
Locations	United States

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