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AAV BBP-631

Phase 1

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMC
Total Trials1
Total Enrollment8
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04783181A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)PHASE1 ACTIVE NOT_RECRUITING 8 — — Jul 1, 2021Feb 1, 2029Dec 23, 20255 United States
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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 — 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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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT04783181studyFirstPostDate: changed