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Scientists watch LGMD progress in 205 patients over years

NCT ID NCT04475926

Knowledge-focused Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study follows 205 people with four types of limb-girdle muscular dystrophy (LGMD) to understand how the disease changes over time. Participants will have their muscle strength, movement, and breathing tested regularly for up to 5 years. No treatment is given; the goal is to learn more about the disease's natural history.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

205 people

The number who actually took part.

Started

Apr 2021

Expected to finish

Sep 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

The population for this study is ambulatory or non-ambulatory participants ≥ 4 years of age with confirmed genetic diagnosis of LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, or LGMD2A/R1.

Ages

4 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Male or female participant ≥ 4 years of age who demonstrate symptoms of LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, or LGMD2A/R1 in the opinion of the investigator (eg, muscle weakness, loss of function, delayed milestones). * Confirmed clinical and genetic diagnosis of LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, or LGMD2A/R1. Exclusion Criteria: * Demonstrates cognitive delay or impairment that could confound motor development, in the opinion of the Investigator. * Has a medical condition, in the opinion of the Investigator, that might compromise participants ability to comply with study requirements. * Is participating in other interventional study(ies) at the time of enrollment in this study.

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Conditions

The condition(s) this trial relates to.

autosomal recessive limb-girdle muscular dystrophy type 2A autosomal recessive limb-girdle muscular dystrophy type 2C autosomal recessive limb-girdle muscular dystrophy type 2D autosomal recessive limb-girdle muscular dystrophy type 2E limb-girdle muscular dystrophy muscular dystrophy sarcoglycanopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Limb girdle muscular dystrophy

Contacts and locations

Locations

  • Anne & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Arkansas Children's

    Little Rock, Arkansas, 72202, United States

  • Azienda Ospedale Università Padova

    Padova, Veneto, 35129, Italy

  • Barrow Neurological Institute

    Phoenix, Arizona, 85013, United States

  • Children's Hospital - London Health Science Centre

    London, Ontario, N6C 2R5, Canada

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, 23507, United States

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Hospital Sant Joan de Déu Universidad de Barcelona

    Barcelona, 08950, Spain

  • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

  • Hôpital Universitaire des Enfants Reine Fabiola

    Brussels, 1020, Belgium

  • Institute of Genetic Medicine, International Centre for Life

    Newcastle upon Tyne, NE1 3BZ, United Kingdom

  • Istituto Giannina Gaslini

    Genoa, Liguria, 16147, Italy

  • Karolinska Universitetssjukhuset Solna

    Stockholm, Stockholm County, 17176, Sweden

  • Lokman Hekim Etlik Hastanesi

    Ankara, 06100, Turkey (Türkiye)

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Oregon Health and Science University

    Portland, Oregon, 97068, United States

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • UCL Institute of Child Health & Great Ormond Street Hospital for Children

    London, WC1N 1EH, United Kingdom

  • UZ Leuven

    Leuven, Vlaams Brabant, 3000, Belgium

  • University Hospital Gent

    Ghent, Oost-Vlaanderen, 9000, Belgium

  • University of California San Diego

    La Jolla, California, 92037, United States

  • University of California, Davis Health Dept of PM&R

    Sacramento, California, 95817, United States

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75201, United States

  • University of Utah Hospital

    Salt Lake City, Utah, 84112, United States

  • Universitätsklinikum Essen

    Essen, North Rhine-Westphalia, D-45147, Germany

  • Zentrum für Kinderheilkunde und Jugendmedizin Uniklinikum Giessen Marburg (UKGM), Standort Giessen

    Giessen, Hesse, 35392, Germany

More trials for these conditions

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