Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorSheba Medical Center
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Patients with clinical diagnosis of OPMD or patients with signs /symptoms suggestive of OPMD
Disqualifiers
Age <18 years and Pregnancy
Trial population
Genetically Confirmed Oculopharyngeal Muscular Dystrophy
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Non-interventional observational study
Other interventionNo intervention will be studied
Treatment groups
300 Participants
are divided into 1 treatment groupGroup A: Subjects with genetically diagnosed OPMD1 intervention
Trial outcomes
Primary outcomes
1
Severity of muscle strength decline over time
10-meter walk test
Time frame
1 year2
Penetration/Aspiration Scale
Penetration/Aspiration Scale (PAS)
Time frame
1 year3
Upper Margin Reflex Distance
Upper Margin Reflex Distance
Time frame
1 year4
Lower Margin Reflex Distance
Lower Margin Reflex Distance
Time frame
1 yearSecondary outcomes
1
Maximum Expiratory Pressure
Maximum Expiratory Pressure (MEP)
Time frame
2 years2
Cough Peak Flow
Cough Peak Flow (CPF)
Time frame
2 years3
Oral health
Decayed, Missing, and Filled Teeth (DMFT) index
Time frame
2 years4
Dysphagia severity
Yale Pharyngeal Residue Severity Rating Scale
Time frame
1 yearSponsors and contacts
Click on the lead sponsor to view all of their trials.
Sheba Medical Center
Lead sponsor
The non-profit organization for promotion of health and cure of OPMD
Collaborator
Source ClinicalTrials.gov