Clinical trials: Oculopharyngeal Muscular Dystrophy (OPMD)
Muscular Dystrophy, Oculopharyngeal (OPMD)
Continuation Protocol to Protocol BBCO-001
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized…
- Trial name
- HOPEMD (NCT02328482)
- Testing
- Tehalose 30gr
- Where
- Canada
- Volunteers
- 9 people
- Sponsor
- Bioblast Pharma Ltd.
Oculopharyngeal Muscular Dystrophy
Screening in Oculopharyngeal Muscular Dystrophy
Oculopharyngeal muscular dystrophy (OPMD) is a rare myopathic disease that results in progressive degeneration of the oral and pharyngeal muscular, resulting in severe dysphagia and dysarthria.
- Trial name
- NCT02877784
- Testing
- Videofluoroscopic swallowing study, Functional Oral Intake Scale, Eating Assessment Tool-10
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Florida
Oculopharyngeal Muscular Dystrophy
Effect of Aerobic Training in Patients With Oculopharyngeal Muscular Dystrophy
The investigators want to investigate if patients with Oculopharyngeal Muscular Dystrophy can improve fitness by home-training on a cycle ergometer 30 minute, 3 times a week for 10 weeks.
- Trial name
- NCT02158156
- Testing
- Exercise
- Where
- Denmark
- Volunteers
- 8 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Oculopharyngeal Muscular Dystrophy
Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose
BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly…
- Trial name
- NCT04226924
- Testing
- Trehalose
- Where
- 3 sites in Canada
- Sponsor
- Bioblast Pharma Ltd.
Oculopharyngeal Muscular Dystrophy, Muscular Dystrophies
Expiratory Muscle Strength Training (EMST) in Neuromuscular Disorders
The purpose of this study is to investigate the impact of expiratory muscle strength training (EMST) on the swallowing, breathing, oral intake, quality of life and cough function of people with oculopharyngeal muscular dystrophy…
- Conditions
- Oculopharyngeal Muscular Dystrophy, Muscular Dystrophies, Myopathy; Hereditary
- Trial name
- NCT04009408
- Testing
- Expiratory muscle strength therapy (EMST150, Aspire LLC)
- Where
- Canada
- Volunteers
- 20 people
- Sponsor
- University of Calgary
Oculopharyngeal Muscular Dystrophy
Natural History Study of Oculopharyngeal Muscular Dystrophy
The objective of this study is to test a set of clinical outcome measures longitudinally in a cohort of OPMD patients to identify ones that show quantifiable change over time as the disease progresses.
- Trial name
- NH-OPMD (NCT03161847)
- Testing
- Non-interventional study
- Sponsor
- University of New Mexico
Oculopharyngeal Muscular Dystrophy
Oral Health, Saliva Viscosity and Composition in Oculo-Pharyngeal Muscular Dystrophy (OPMD)
The goal of this observational study is to explore whether oral health and saliva viscosity and composition in Oculopharyngeal Muscular Dystrophy (OPMD) is different from control subjects.
- Trial name
- NCT07118280
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- Sheba Medical Center
Oculopharyngeal Muscular Dystrophy
A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
Subjects who have enrolled in the oculopharyngeal muscular dystrophy (OPMD) natural history study (Study BNTC-OPMD-NH-001) and have completed at least 6 months of follow up in Study BNTC-OPMD-NH-001 may be eligible to participate…
- Trial name
- NCT06185673
- Testing
- BB-301: Dose escalation phase 1b cohort 1, BB-301: Dose escalation phase 1b cohort 2, BB-301: Dose escalation phase 1b cohort 3
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Benitec Biopharma, Inc.
Cardiomyopathy, Hypertrophic, Muscular Dystrophy, Oculopharyngeal
Study of Muscle Abnormalities in Patients With Specific Genetic Mutations
Hypertrophic cardiomyopathy (HCM) is a genetically inherited disease affecting the heart. It causes thickening of heart muscle, especially the chamber responsible for pumping blood out of the heart, the left ventricle.
- Trial name
- NCT00001871
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Oculopharyngeal Muscular Dystrophy
Pathology Analysis of OPMD Patient Myotomies
The study aims to characterize the effect of OPMD mutation on muscle function. Muscle samples will be received from oculopharyngeal muscular dystrophy (OPMD) patients who undergo cricopharyngeal muscle-cutting surgery, as part of…
- Trial name
- NCT03874910
- Where
- Israel
- Volunteers
- 10 people
- Sponsor
- Hillel Yaffe Medical Center
Dysphagia
Dysphagia in Oculopharyngeal Muscular Dystrophy (OPMD)- Evaluation, Endoscopic Examination of Swallowing, Treatment and Long Term Follow up
The investigators aimed to review the natural history of dysphagia and dysphonia in OPMD in order to identify the best candidates and the proper timing to perform dysphagia alleviating procedures in both heterozygote and…
- Trial name
- OPMD (NCT01167439)
- Testing
- Upper esophageal sphincter myotomy
- Where
- Israel
- Volunteers
- 40 people
- Sponsor
- Hillel Yaffe Medical Center
Muscular Dystrophy, Oculopharyngeal
Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts
The OCULO-Pharyngeal Muscular Dystrophy (OPMD) is a late onset hereditary muscle disease which is characterised by the selective affection of the pharyngeal muscles resulting in swallowing disorders, and by a ptosis from the…
- Trial name
- OPMD (NCT00773227)
- Testing
- Autologous myoblasts transplantation and myotomy
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Oculopharyngeal Muscular Dystrophy
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
- Trial name
- HOPEMD (NCT02015481)
- Testing
- Cabaletta
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Bioblast Pharma Ltd.
Oculopharyngeal Muscular Dystrophy (OPMD)
Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry
The goal of this open prospective multi-disciplinary observational study of patients with OPMD at various stages of clinical manifestations is to explore the natural history of the disease in the Israeli population.
- Trial name
- Isr-OPMD (NCT07146256)
- Testing
- Non-interventional observational study
- Where
- Israel
- Volunteers
- 300 people
- Sponsor
- Sheba Medical Center