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NCT04998396 · ClinicalTrials.gov registry record · Phase 1
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
A Phase 1 study of Canavan Disease, sponsored by Aspa Therapeutics.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 26
- Enrollment target
- 4
- Study locations
NCT04998396: Recruiting Phase 1 study of Canavan Disease, sponsored by Aspa Therapeutics.
NCT04998396 is a Phase 1 study of Canavan Disease that is actively recruiting participants, run by Aspa Therapeutics. The registered enrollment target is 26 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (57% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04998396, a Phase 1 study of Canavan Disease, is actively recruiting participants, sponsored by Aspa Therapeutics.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 26 participants
- Enrollment target
- 4
- Study locations
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 Studied
Interventions
- BIOLOGICAL AAV9 BBP-812
Study Locations (4)
California
- UCSF Benioff Children's Hospital Oakland - Oakland
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Massachusetts
- Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) - Boston
New York
- Weill Cornell Medicine; Division of Pediatric Neurology - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 26 participants |
| Start Date | 2021-09-08 |
| Est. Completion | 2030-10-08 |
| Phase | Phase 1 |
What NCT04998396 shows while recruiting
NCT04998396 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 26 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (57% lower).
The record links to 1 condition, with Canavan Disease appearing as the primary indexed condition, and to 1 intervention - of which AAV9 BBP-812 is the first listed.
NCT04998396 reports a single indexed study location in California, Illinois, Massachusetts.
Frequently Asked Questions
What is clinical trial NCT04998396 about?
NCT04998396 is a clinical study titled "A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)". 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.
What is the current status of trial NCT04998396?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 26 participants. The study started on 2021-09-08. Estimated completion is 2030-10-08.
What conditions does trial NCT04998396 study?
This clinical trial studies the following conditions: Canavan Disease.
What interventions are being tested in trial NCT04998396?
The interventions under investigation include: AAV9 BBP-812 (BIOLOGICAL).
Who is sponsoring clinical trial NCT04998396?
This trial is sponsored by Aspa Therapeutics, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04998396 being conducted?
This trial has 4 study locations across California, Illinois, Massachusetts, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04998396's enrollment target sits among peer trials
26 1482nd of 2000 higher than 506 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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