NCT03874910

Brief Summary

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 their routine healthcare and treatment.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Mar 2019

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
unknown

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

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 4, 2019

Completed
10 days until next milestone

First Posted

Study publicly available on registry

March 14, 2019

Completed
5 days until next milestone

Study Start

First participant enrolled

March 19, 2019

Completed
4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2023

Completed
Last Updated

March 21, 2019

Status Verified

March 1, 2019

Enrollment Period

4 years

First QC Date

March 4, 2019

Last Update Submit

March 19, 2019

Conditions

Oculopharyngeal Muscular Dystrophy

Outcome Measures

Primary Outcomes (2)

  • Number of genes that show changes in RNA profile, measured by sequencing.

    RNA profile analysis of OPMD muscle samples will be compared to unaffected muscle samples using sequencing to identify gene numbers.

    Data will be collected within four years.

  • Number of genes that show changes in protein profile, measured by SDS page.

    Protein profile analysis of OPMD muscle samples will be compared to unaffected muscle samples to identify gene numbers using SDS page.

    Data will be collected within four years.

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients who suffer from oculopharyngeal muscular dystrophy (OPMD).

You may qualify if:

  • OPMD patients both male and female.
  • OPMD patients between the ages 18-70 years.
  • OPMD patients who are referred to the cricopharyngeal muscle-cutting surgery as part of their usual treatment.

You may not qualify if:

  • OPMD patients not referred to the cricopharyngeal muscle-cutting surgery as part of their usual treatment.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hillel Yaffe Medical Center

Hadera, Israel

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Oculopharyngeal

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Itzhak Braverman, Doctor (MD)

    Hillel Yaffe Medical Center, Israel

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Itzhak Braverman, Doctor (MD)

CONTACT

+972-4-6304549braverman@hy.health.gov.il

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER GOV
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 4, 2019

First Posted

March 14, 2019

Study Start

March 19, 2019

Primary Completion

March 1, 2023

Study Completion

March 1, 2023

Last Updated

March 21, 2019

Record last verified: 2019-03

Locations

IL(1)