Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Non-interventional observational study.
- Who it may be relevant to
- Registry conditions: Oculopharyngeal Muscular Dystrophy (OPMD). Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Israel
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Detailed description
The the following procedures will be performed by a multidisciplinary team:
Neurological and neurocognitive examination; Clinical assessment of of swallowing and severity of dysphagia; Clinical assessment of eyelid ptosis severity; Clinical assessment of respiratory muscles function; Clinical assessment of oral health
Interventions
- Other Non-interventional observational study
No intervention will be studied
Primary outcome measures
- Severity of muscle strength decline over time [Time frame: 1 year]
- Penetration/Aspiration Scale [Time frame: 1 year]
- Upper Margin Reflex Distance [Time frame: 1 year]
- Lower Margin Reflex Distance [Time frame: 1 year]
Secondary outcome measures (6)
- Maximum Expiratory Pressure [Time frame: 2 years]
- Cough Peak Flow [Time frame: 2 years]
- Oral health [Time frame: 2 years]
- Dysphagia severity [Time frame: 1 year]
- Functional Oral Intake Scale [Time frame: 1 year]
- Swallowing Disturbance [Time frame: 1 year]
Eligibility criteria
Inclusion criteria
- Patients with clinical diagnosis of OPMD or patients with signs /symptoms suggestive of OPMD
Exclusion criteria
- Age <18 years and Pregnancy
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Israel · 1 center
- Sheba Medical Center — Ramat Gan
Identifiers
NCT: NCT07146256 · SHEBA-21-8801-AD-CTIL