Current trials in LGMD
TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trials that are currently ongoing.
Nevertheless, we cannot ourselves keep an up-to-date overview of all the trials that are currently happening across the world. The most comprehensive online listing of trials is at clinicaltrials.gov – companies are obliged to provide details of their trials there.
The table below lists all LGMD trials registered or updated on clinicaltrials.gov in the last 1000 days. Because it links directly to clinicaltrials.gov it is automatically updated whenever the information on clinicaltrials.gov changes.
Please note that TREAT-NMD can take no responsibility for the accuracy of the information below, which is pulled directly from clinicaltrials.gov.
Trials for LGMD listed on clinicaltrials.gov
- Trial Readiness and Endpoint Assessment in LGMD R1
- Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet)
- Limb-Girdle Video Assessment
- Defining Endpoints in Becker Muscular Dystrophy
- A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
- GNT0006 Gene Therapy Trial in Patients With LGMDR9
- Assessment of Safety and Acute Effects of a Knee-hip Powered Soft Exoskeleton in Patients With Neuromuscular Disorders
- Assessment of Safety and Acute Effects of a Lower-limb Powered Dermoskeleton in Patients With Neuromuscular Disorders
- A Multicenter Phenotype-Genotype Analysis of LGMD Patients in China
- Study of BBP-418 in Patients With LGMD2I
- Motor Parameters in Patients With Limb Girdle Muscular Dystrophy
- Clinical Determinants of Disease Progression in Patients With Limb Girdle Muscular Distrophy Type 2E
- A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
- Telerehabilitation in Proximal Muscle Weakness
- LARA: Use of JACO2 Controlled by Joystick or by Voice Control System, in Muscular Dystrophy