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LGMD2I clinical trials

View clinical trials related to LGMD2I.

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NCT ID: NCT05989620 Recruiting - LGMD2I Clinical Trials

Long-Term Development of Muscular Dystrophy Outcome Assessments

GRASP-01-005
Start date: October 18, 2023
Phase:
Study type: Observational

This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).

NCT ID: NCT05230459 Recruiting - Muscular Dystrophy Clinical Trials

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

Start date: March 12, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

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)

NCT ID: NCT04800874 Active, not recruiting - LGMD2I Clinical Trials

Study of BBP-418 in Patients With LGMD2I

Start date: February 18, 2021
Phase: Phase 2
Study type: Interventional

BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I). This is an open label study to determine the safety and tolerability of ascending dose levels of BBP-418 in the treatment of ambulatory and non-ambulatory patients with LGMD2I for which no approved therapy currently exists.

NCT ID: NCT04001595 Recruiting - Clinical trials for Limb Girdle Muscular Dystrophy

Global FKRP Registry

Start date: November 2013
Phase:
Study type: Observational [Patient Registry]

Mutations in the Fukutin Related Protein (FKRP) gene cause the condition Limb Girdle Muscular Dystrophy type R9 (LGMDR9) also known as LGMD2I, and the rarer conditions Congenital Muscular Dystrophy (MDC1C), Muscle Eye Brain Disease (MEB) and Walker-Warburg Syndrome (WWS). LGMDR9 is the most common FKRP-related condition, and is especially prevalent in Northern Europe. The aim is to facilitate a questionnaire based research study in order to better characterise and understand the disease globally. By maintaining a global registry this will help identify potential participants eligible for clinical trials in the future.

NCT ID: NCT03842878 Active, not recruiting - LGMD2I Clinical Trials

Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I

Start date: February 19, 2020
Phase:
Study type: Observational

Prospective, longitudinal, interventional, single-group, multicenter natural history study to better know the LGMD2I disease physiopathology. The duration of participation for each patient will be up to 24 months.

NCT ID: NCT02841267 Completed - LGMD2I Clinical Trials

A Trial of PF-06252616 in Ambulatory Participants With LGMD2I

Start date: July 2016
Phase: Phase 1/Phase 2
Study type: Interventional

The investigational product PF 06252616, a humanized anti myostatin monoclonal antibody that neutralizes myostatin (GDF8) is in development for the treatment of Limb Girdle Muscular Dystrophy 2I (LGMD2I) to preserve and/or improve muscle function. This study will provide the clinical assessment of the safety, tolerability, Pharmacokinetics and Pharmacodynamics of PF 06252616 following repeat IV doses in ambulatory adults with LGMD2I.