NCT07146256

Brief 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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
300

participants targeted

Target at P75+ for all trials

Timeline
41mo left

Started Feb 2022

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress57%
Feb 2022Jan 2030

Study Start

First participant enrolled

February 1, 2022

Completed
3.5 years until next milestone

First Submitted

Initial submission to the registry

August 5, 2025

Completed
23 days until next milestone

First Posted

Study publicly available on registry

August 28, 2025

Completed
4.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2030

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2030

Last Updated

August 28, 2025

Status Verified

August 1, 2025

Enrollment Period

7.9 years

First QC Date

August 5, 2025

Last Update Submit

August 21, 2025

Conditions

Oculopharyngeal Muscular Dystrophy (OPMD)

Keywords

Oculopharyngeal muscular dystrophy (OPMD)Natural historyDysphagiaEyelid ptosisMuscle weaknessOral health

Outcome Measures

Primary Outcomes (4)

  • Severity of muscle strength decline over time

    10-meter walk test

    1 year

  • Penetration/Aspiration Scale

    Penetration/Aspiration Scale (PAS)

    1 year

  • Upper Margin Reflex Distance

    Upper Margin Reflex Distance

    1 year

  • Lower Margin Reflex Distance

    Lower Margin Reflex Distance

    1 year

Secondary Outcomes (6)

  • Maximum Expiratory Pressure

    2 years

  • Cough Peak Flow

    2 years

  • Oral health

    2 years

  • Dysphagia severity

    1 year

  • Functional Oral Intake Scale

    1 year

  • +1 more secondary outcomes

Study Arms (1)

Subjects with genetically diagnosed OPMD

Other: Non-interventional observational study

Interventions

Non-interventional observational studyOTHER

No intervention will be studied

Subjects with genetically diagnosed OPMD

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Genetically Confirmed Oculopharyngeal Muscular Dystrophy

You may qualify if:

  • Patients with clinical diagnosis of OPMD or patients with signs /symptoms suggestive of OPMD

You may not qualify if:

  • Age \<18 years and Pregnancy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Sheba Medical Center

Ramat Gan, Israel

RECRUITING

Related Links

MeSH Terms

Conditions

Muscular Dystrophy, OculopharyngealDeglutition DisordersBlepharoptosisMuscle Weakness

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesEsophageal DiseasesGastrointestinal DiseasesDigestive System DiseasesPharyngeal DiseasesOtorhinolaryngologic DiseasesEyelid DiseasesEye DiseasesNeuromuscular ManifestationsNeurologic ManifestationsPathologic ProcessesPathological Conditions, Signs and SymptomsSigns and Symptoms

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
5 Years
Sponsor Type
OTHER GOV
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 5, 2025

First Posted

August 28, 2025

Study Start

February 1, 2022

Primary Completion (Estimated)

January 1, 2030

Study Completion (Estimated)

January 1, 2030

Last Updated

August 28, 2025

Record last verified: 2025-08

Locations

IL(1)