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BBP-631

Phase 1

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

Target and mechanism

ModalityMonoclonal antibody

Also known as AAV BBP-631

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment8

FDA Designations

No designations recorded

Clinical trial landscape

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
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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
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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
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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
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