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LGMDR9 clinical trials

View clinical trials related to LGMDR9.

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NCT ID: NCT05224505 Recruiting - LGMDR9 Clinical Trials

GNT0006 Gene Therapy Trial in Patients With LGMDR9

Start date: August 10, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

Phase 1-2 study including a dose escalation safety and proof of concept phase (Stage 1, open label), followed by a double-blind, randomized, placebo-controlled confirmatory phase (Stage 2)

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.