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BBP-812

Phase 1

Canavan Disease | Monoclonal antibody | Rare Disease |BridgeBio Pharma, Inc.|Last Updated: Apr 17, 2026

Target and mechanism

ModalityMonoclonal antibody

Also known as AAV9 BBP-812

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment26

FDA Designations

RMATORPHAN_DRUGRARE_PEDIATRIC_DISEASEFAST_TRACK

Clinical trial landscape

BBP-812 · 1 trial · 1 indication

Phase 1 1
NCT04998396A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)Canavan Disease
RECRUITING26 Analytics
PHASE1RECRUITING
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
Canavan Disease
NCT0499839626
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Study Endpoints

Primary Endpoints

Number of Participants with Adverse Events (AEs)
Baseline up to Week 52
Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels
Baseline, Month 12
Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS)
Baseline, Month 12

Secondary Endpoints

Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88
Baseline, Week 52
Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4
Baseline, Week 52
Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4
Baseline, Week 52
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1)EXPERIMENTALParticipants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study.
Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2)EXPERIMENTALParticipants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study.
Enrollment Expansion Phase: BBP-812EXPERIMENTALParticipants will receive a single IV infusion of BBP-812 at the selected dose from the dose-finding phase on Day 0 in expansion phase of the study.

Interventions

NameTypeDescription
AAV9 BBP-812BIOLOGICALSterile solution for injection for 1-time use via volumetric infusion pump
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Eligibility Criteria

Age RangeN/A to 30 Months
SexALL
Healthy VolunteersNo
Study Sites4

Key Inclusion Criteria: * Maximum age for inclusion is 30 months. * Participant has stable health in the opinion of the investigator and as confirmed by medical history and laboratory studies with no acute or chronic hematologic, renal, liver, immunologic, or neurologic disease (other than Canavan ...

Countries:United States
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