Oculopharyngeal Muscular Dystrophy (OPMD)Isr-OPMDNCT07146256
Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry
An observational study of Oculopharyngeal Muscular Dystrophy (OPMD), run by Sheba Medical Center at 1 site in Israel.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Oculopharyngeal Muscular Dystrophy (OPMD)
- What is being tested?
- Non-interventional observational study
- How many people
- 300 participants
- Where
- 1 siteIsrael
- Study dates
- 1 Feb 2022to Jan 2030
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Genetically Confirmed Oculopharyngeal Muscular Dystrophy
Who may be able to join
- Patients with clinical diagnosis of OPMD or patients with signs /symptoms suggestive of OPMD
Who cannot join
- Age <18 years and Pregnancy
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Israel1 site, 1 recruiting
- Recruiting
Sheba Medical Center
Ramat Gan
Naama Study coordinator, +972552929006, OPMD@sheba.health.gov.il
Amir Dori, MD, PhD
About this study
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.
Read the 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
- Being tested
- Non-interventional observational study
- Keywords
- Oculopharyngeal muscular dystrophy (OPMD), Natural history, Dysphagia, Eyelid ptosis, Muscle weakness, Oral health
- Medical subject headings
- Muscular Dystrophy, Oculopharyngeal, Deglutition Disorders, Blepharoptosis, Muscle Weakness
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Non-interventional observational studyOther
No intervention will be studied
| Study group | Role | What they receive |
|---|---|---|
| Subjects with genetically diagnosed OPMD | Not described Receives: Other: Non-interventional observational study |
What is being measured
The primary outcome is the main question the study is designed to answer.
Severity of muscle strength decline over time
10-meter walk test
Measured: 1 year
Penetration/Aspiration Scale
Penetration/Aspiration Scale (PAS)
Measured: 1 year
Upper Margin Reflex Distance
Upper Margin Reflex Distance
Measured: 1 year
Lower Margin Reflex Distance
Lower Margin Reflex Distance
Measured: 1 year
Secondary outcomes (6)
Maximum Expiratory Pressure
Maximum Expiratory Pressure (MEP)
Measured: 2 years
Cough Peak Flow
Cough Peak Flow (CPF)
Measured: 2 years
Oral health
Decayed, Missing, and Filled Teeth (DMFT) index
Measured: 2 years
Dysphagia severity
Yale Pharyngeal Residue Severity Rating Scale
Measured: 1 year
Functional Oral Intake Scale
Functional Oral Intake Scale (FOIS)
Measured: 1 year
Swallowing Disturbance
Swallowing Disturbance Questionnaire (SDQ)
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2025
- Study start1 Feb 2022
- Primary completionJan 2030 (estimated)
- Study completionJan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Oculopharyngeal Muscular Dystrophy (OPMD)Studies of the same condition, open and under way.
- More trials run by Sheba Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Sheba Medical Center
- Sponsor type
- Other government
- Collaborators
- The non-profit organization for promotion of health and cure of OPMD
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07146256
- Sponsor's protocol ID
- SHEBA-21-8801-AD-CTIL
- Acronym
- Isr-OPMD
- Status last verified
- Aug 2025
- First submitted
- 5 Aug 2025
- Last updated
- 28 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry
- ClinicalTrials.gov ID
- NCT07146256
- Acronym / sponsor ID
- Isr-OPMD / SHEBA-21-8801-AD-CTIL
- Keywords
- Oculopharyngeal muscular dystrophy (OPMD), Natural history, Dysphagia, Eyelid ptosis, Muscle weakness, Oral health
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Sheba Medical CenterOther government
- Collaborators
- The non-profit organization for promotion of health and cure of OPMD
- Study start
- 1 Feb 2022 (actual)
- Primary completion
- Jan 2030 (estimated)
- Study completion
- Jan 2030 (estimated)
- First posted
- 28 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Subjects with genetically diagnosed OPMD | – | Other: Non-interventional observational study |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Genetically Confirmed Oculopharyngeal Muscular Dystrophy
Key inclusion criteria (1 listed)
- Patients with clinical diagnosis of OPMD or patients with signs /symptoms suggestive of OPMD
Key exclusion criteria (1 listed)
- Age <18 years and Pregnancy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Non-interventional observational studyNo intervention will be studied | Other | Subjects with genetically diagnosed OPMD |
Primary outcome
Severity of muscle strength decline over time
Time frame: 1 year
10-meter walk test
Penetration/Aspiration Scale
Time frame: 1 year
Penetration/Aspiration Scale (PAS)
Upper Margin Reflex Distance
Time frame: 1 year
Upper Margin Reflex Distance
Lower Margin Reflex Distance
Time frame: 1 year
Lower Margin Reflex Distance
6 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2025.
- The National Israeli Registry for Oculo-Pharyngeal Muscular Dystrophy (IsrO-PMD): rationale and designLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Oculopharyngeal Muscular Dystrophy (OPMD), run by Sheba Medical Center at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 28 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Severity of muscle strength decline over time (time frame: 1 year). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Aug 2025.