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NCT03533673 · ClinicalTrials.gov registry record · Phase 1
AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease
A Phase 1 study of Pompe Disease, sponsored by AskBio.
- Active
- Registry status
- Phase 1
- Development phase
- 7
- Enrollment target
- 1
- Study location
NCT03533673: Active Phase 1 study of Pompe Disease, sponsored by AskBio.
NCT03533673 is a Phase 1 study of Pompe Disease that is active but no longer recruiting, run by AskBio. The registered enrollment target is 7 participants, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (97% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03533673, a Phase 1 study of Pompe Disease, is active but no longer recruiting, sponsored by AskBio.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 7 participants
- Enrollment target
- 1
- Study location
Study Summary
Open-label, ascending dose trial of ACTUS-101 administered intravenously.
Primary Outcome
The incidence of patient reported TEAEs will be measured according to protocol specifications.
Conditions Studied
Interventions
- BIOLOGICAL ACTUS-101
Study Locations (1)
North Carolina
- Duke University - Durham
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 7 participants |
| Start Date | 2018-12-17 |
| Est. Completion | 2026-03 |
| Phase | Phase 1 |
What the registry record for NCT03533673 still lists
NCT03533673 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 7 participants, a relatively small participant target, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (97% lower).
The record links to 1 condition, with Pompe Disease appearing as the primary indexed condition, and to 1 intervention - of which ACTUS-101 is the first listed.
NCT03533673 reports a single indexed study location in North Carolina.
Frequently Asked Questions
What is clinical trial NCT03533673 about?
NCT03533673 is a clinical study titled "AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease". Open-label, ascending dose trial of ACTUS-101 administered intravenously.
What is the current status of trial NCT03533673?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 7 participants. The study started on 2018-12-17. Estimated completion is 2026-03.
What conditions does trial NCT03533673 study?
This clinical trial studies the following conditions: Pompe Disease.
What interventions are being tested in trial NCT03533673?
The interventions under investigation include: ACTUS-101 (BIOLOGICAL).
Who is sponsoring clinical trial NCT03533673?
This trial is sponsored by AskBio, which has 8 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03533673 being conducted?
This trial has 1 study location across North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Pompe Disease
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 NCT03533673's enrollment target sits among peer trials
7 13th of 14 higher than 2 of 14 other Pompe Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease 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
-
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
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-
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
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Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
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Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
RECRUITING · Phase 2
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Cognitive and Neurological Pathologies in Pompe Disease
RECRUITING
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Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
RECRUITING
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