Clinical trials: Limb Girdle Muscular Dystrophy
Hypertension, Pulmonary
Prospective, Non-interventional, Multi-center, Post-authorization Safety Study of Riociguat for Pulmonary Arterial Hypertension (PAH)
The primary objective in this study is to collect post-marketing information on Riociguat safety.Thus, the information on adverse events (AEs) and adverse drug reactions (ADRs) that occur within the first 12 months and in the…
- Trial name
- JPMS-PAH (NCT02428985)
- Testing
- Riociguat (ADEMPAS, BAY63-2521)
- Where
- Japan
- Volunteers
- 882 people
- Sponsor
- Bayer
LGMDR9
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
- Trial name
- NCT05224505
- Testing
- ATA-100 (AAV9 encoding FKRP gene)
- Where
- 3 sites in Denmark, France and 1 more
- Volunteers
- 6 people
- Sponsor
- Atamyo Therapeutics
Limb-girdle Muscular Dystrophy
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- EMERGENE (NCT06246513)
- Testing
- SRP-9003, Glucocorticoid
- Where
- 10 sites in United States, Belgium and 4 more
- Volunteers
- 17 people
- Sponsor
- Sarepta Therapeutics, Inc.
Limb Girdle Muscular Dystrophy, Muscular Dystrophies
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)
Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles.
- Trial name
- GRASP-01-001 (NCT03981289)
- Where
- 10 sites in United States, United Kingdom
- Volunteers
- 116 people
- Sponsor
- Virginia Commonwealth University
Limb-Girdle Muscular Dystrophy, Social Competence
Telecare Psychosocial and Cognitive Intervention for Children and Adolescents With Limb-Girdle Muscular Dystrophy
This clinical trial aims to evaluate the efficacy of a telecare-mediated psychosocial and cognitive intervention for children and adolescents aged 7 to 17 with limb-girdle muscular dystrophy.
- Conditions
- Limb-Girdle Muscular Dystrophy, Social Competence, Self Esteem, Health Related Quality of Life
- Trial name
- TPCLGMD (NCT07711730)
- Testing
- Neuro-psychosocial teleassistance program
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- University of Deusto
Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)
Limb Girdle Muscular Dystrophy (LGMD) Natural History
The purpose of this study is to understand more about limb-girdle muscular dystrophy. Therefore, the investigators would like to track the following information collected once a year from patients with GENETICALLY CONFIRMED LGMD…
- Trial name
- NCT01783509
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Wake Forest University Health Sciences
LGMD2I
Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I
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.
- Trial name
- NCT03842878
- Where
- 3 sites in Denmark, France and 1 more
- Volunteers
- 52 people
- Sponsor
- Genethon
Limb-Girdle Muscular Dystrophy
Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy
The objective of this study is to identify and maintain a registry of well-characterized limb-girdle muscular dystrophy (LGMD) patients.
- Trial name
- NCT00457912
- Where
- United States
- Volunteers
- 277 people
- Sponsor
- Nationwide Children's Hospital
Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A
Clinical and Biochemical Features for the Identification of Dominant Calpainopathies
Mutations in the CAPN3 gene cause muscular dystrophies with dysfunction in calpain-3. Calpainopathies are usually inherited in an autosomal recessive manner but in some families they can occur in a dominant inheritance.
- Trial name
- DOM-CAL (NCT05956132)
- Testing
- retrospective study
- Where
- 2 sites in Italy
- Volunteers
- 50 people
- Sponsor
- IRCCS San Camillo, Venezia, Italy
Limb-girdle Muscular Dystrophy
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy…
- Trial name
- NCT04475926
- Where
- 26 sites in United States, Belgium and 8 more
- Volunteers
- 205 people
- Sponsor
- Sarepta Therapeutics, Inc.