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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 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.

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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)

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

FieldValue
Enrollment Target 10 participants
Start Date 2023-05-15
Est. Completion 2032-12
Phase Phase 1

Sponsor

AskBio

8 total trials

What the Registry Record Tells You About NCT05230459

The ClinicalTrials.gov registry entry for NCT05230459 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 880-participant average among 11 other Muscular Dystrophy trials with a reported enrollment target (99% lower). The listed sponsor is AskBio, which has 8 total studies on file at ClinicalTrials.gov.

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 reports 6 study locations spanning 6 distinct geographic areas - top geographies include 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.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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