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NCT05230459 · ClinicalTrials.gov registry record · Phase 1
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
A Phase 1 study of Muscular Dystrophy and Limb Girdle Muscular Dystrophy, sponsored by AskBio.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
- 6
- Study locations
NCT05230459: Recruiting Phase 1 study of Muscular Dystrophy and Limb Girdle Muscular Dystrophy, sponsored by AskBio.
NCT05230459 is a Phase 1 study of Muscular Dystrophy and Limb Girdle Muscular Dystrophy that is actively recruiting participants, run by AskBio. The registered enrollment target is 10 participants, below the 880-participant average among 11 other Muscular Dystrophy trials with a reported enrollment target (99% lower). The trial reports 6 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05230459, a Phase 1 study of Muscular Dystrophy and Limb Girdle Muscular Dystrophy, is actively recruiting participants, sponsored by AskBio.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
- 6
- Study locations
Study Summary
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)
Primary Outcome
Treatment Emergent Adverse Events, Serious Adverse Events, Dose Limiting Toxicity
Conditions Studied
Interventions
- OTHER Placebo
- GENETIC AB-1003 dose level 1
- GENETIC AB-1003 dose level 2
Study Locations (6)
California
- University of California - Irvine - Irvine
Iowa
- University of Iowa - Iowa City
Kansas
- University of Kansas Medical Center - Kansas City
Maryland
- Kennedy Krieger Institute - Baltimore
Virginia
- VCU - Richmond
Washington
- University of Washington Medical Center - Seattle
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2023-05-15 |
| Est. Completion | 2032-12 |
| Phase | Phase 1 |
What NCT05230459 shows while recruiting
NCT05230459 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 880-participant average among 11 other Muscular Dystrophy trials with a reported enrollment target (99% lower).
The record links to 9 conditions, with Muscular Dystrophy appearing as the primary indexed condition, and to 3 interventions - of which Placebo is the first listed.
NCT05230459 lists 6 locations in 6 states (California, Iowa, Kansas).
Frequently Asked Questions
What is clinical trial NCT05230459 about?
NCT05230459 is a clinical study titled "A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)". The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)
What is the current status of trial NCT05230459?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2023-05-15. Estimated completion is 2032-12.
What conditions does trial NCT05230459 study?
This clinical trial studies the following conditions: Muscular Dystrophy, Limb Girdle Muscular Dystrophy, LGMD2I, Limb-Girdle Muscular Dystrophy Type 2, LGMD2.
What interventions are being tested in trial NCT05230459?
The interventions under investigation include: Placebo (OTHER), AB-1003 dose level 1 (GENETIC), AB-1003 dose level 2 (GENETIC).
Who is sponsoring clinical trial NCT05230459?
This trial is sponsored by AskBio, which has 8 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05230459 being conducted?
This trial has 6 study locations across California, Iowa, Kansas, Maryland, Virginia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT05230459's enrollment target sits among peer trials
10 12th of 11 the lowest of 11 other Muscular Dystrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Muscular Dystrophy 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.
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