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A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

NCT04998396 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 26

Last updated 2026-04-17

No results posted yet for this study

Summary

The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.

Conditions

  • Canavan Disease

Interventions

BIOLOGICAL

AAV9 BBP-812

Sterile solution for injection for 1-time use via volumetric infusion pump

Sponsors & Collaborators

  • Aspa Therapeutics

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Max Age
30 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-09-08
Primary Completion
2026-10-13
Completion
2032-10-08
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04998396 on ClinicalTrials.gov