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

NCT05230459 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-02-20

No results posted yet for this 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

  • Limb Girdle Muscular Dystrophy
  • Limb-Girdle Muscular Dystrophy Type 2
  • LGMD2I
  • Muscular Dystrophy
  • LGMD2
  • LGMD
  • FKRP
  • FKRP Mutation
  • Fukutin Related Protein

Interventions

GENETIC

AB-1003 dose level 1

Single intravenous infusion of AB-1003 gene therapy at dose level 1

GENETIC

AB-1003 dose level 2

Single intravenous infusion of AB-1003 gene therapy at dose level 2

OTHER

Placebo

Single intravenous infusion of Placebo

Sponsors & Collaborators

  • AskBio Inc

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-05-15
Primary Completion
2028-12-31
Completion
2032-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05230459 on ClinicalTrials.gov