Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
NCT05230459 · View on ClinicalTrials.gov ↗
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)
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 |
Interested in This Trial?
Always speak with your doctor before enrolling in a clinical trial.
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05230459
The ClinicalTrials.gov registry entry for NCT05230459 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is AskBio, which has 7 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05230459 reports 6 study locations spanning 6 distinct geographic areas — top geographies include California, Iowa, Kansas. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
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). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05230459?
This trial is sponsored by AskBio, which has 7 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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
How Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.