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Study identifier: NCT05230459 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Condition: Limb Girdle Muscular Dystrophy · Limb-Girdle Muscular Dystrophy Type 2 · LGMD2I  ·  Sponsor: AskBio Inc

PhasePhase 1/Phase 2
Planned participants10
Who can joinAll sexes, 18 Years to 65 Years
Healthy volunteersNo

About this study

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)

This description comes directly from the study's public registry record.

Talk to the study team

AskFirst Patient Engagement  ·  919-561-6210  ·  AskFirst@askbio.com

myTomorrows (see link below in reference section)

Always discuss trial participation with your own doctor first.

Locations (6)

University of California - IrvineIrvine, California, United StatesRecruiting
University of IowaIowa City, Iowa, United StatesRecruiting
University of Kansas Medical CenterKansas City, Kansas, United StatesRecruiting
Kennedy Krieger InstituteBaltimore, Maryland, United StatesRecruiting
VCURichmond, Virginia, United StatesRecruiting
University of Washington Medical CenterSeattle, Washington, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT05230459