Recent Updates
Recently added Catalysts

AAV BBP-631

Phase 1

Congenital Adrenal Hyperplasia | Monoclonal antibody | Endocrine |BridgeBio Pharma, Inc.|Last Updated: Dec 23, 2025

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment8

FDA Designations

No designations recorded

Clinical trial landscape

AAV BBP-631 · 1 trial · 1 indication

Phase 1 1
NCT04783181A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)Congenital Adrenal Hyperplasia
ACTIVE NOT_RECRUITING8 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Congenital Adrenal Hyperplasia
NCT047831818
Unlock trial analytics

Study Endpoints

Primary Endpoints

Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
up to 5 years
To select the optimum dose or dose range of BBP 631 for future studies
up to 5 years

Secondary Endpoints

Change from Baseline in 17-OHP (hydroxyprogesterone) levels
Baseline, Week 52 and through study completion, an average of 5 years
Change from Baseline in androstenedione (A4) levels
Baseline, Week 52 and through study completion, an average of 5 years
Change from Baseline in endogenous cortisol levels
Baseline, Week 52 and through study completion, an average of 5 years
Unlock Study Endpoints

Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Dose Level 1EXPERIMENTALBBP-631 lowest dose, administered once, intravenously (IV)
Dose Level 2EXPERIMENTALBBP-631 middle dose, administered once, IV
Dose Level 3EXPERIMENTALBBP-631, high dose, administered once, IV
Dose Level 4EXPERIMENTALBBP-631, highest dose, administered once, IV

Interventions

NameTypeDescription
AAV BBP-631BIOLOGICALintravenous
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites5

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) maint...

Countries:United States
Unlock Eligibility Criteria

Frequently asked questions about AAV BBP-631

What is AAV BBP-631 used for?

AAV BBP-631 is an investigational gene therapy being developed for the treatment of congenital adrenal hyperplasia, a genetic condition affecting the adrenal glands. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes AAV BBP-631?

AAV BBP-631 is being developed by BridgeBio Pharma, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BBIO. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational gene therapy.

What phase is AAV BBP-631 in?

AAV BBP-631 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is active but not recruiting participants.

What clinical trials is AAV BBP-631 in?

AAV BBP-631 is being studied in a Phase 1 clinical trial with the identifier NCT04783181, titled 'A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH).' This trial is enrolling 8 adult participants in the United States and is currently active but not recruiting.

How does AAV BBP-631 work?

AAV BBP-631 is a gene therapy designed to deliver a functional gene to address the underlying genetic cause of congenital adrenal hyperplasia. The specific molecular target or mechanism of action has not been disclosed in the available information.