Oculopharyngeal Muscular DystrophyNCT03874910
Pathology Analysis of OPMD Patient Myotomies
An observational study of Oculopharyngeal Muscular Dystrophy, run by Hillel Yaffe Medical Center at 1 site in Israel.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Oculopharyngeal Muscular Dystrophy
- How many people
- 10 participants
- Where
- 1 siteIsrael
- Study dates
- 19 Mar 2019to Mar 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients who suffer from oculopharyngeal muscular dystrophy (OPMD).
Who may be able to join
- 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.
Who cannot join
- OPMD patients not referred to the cricopharyngeal muscle-cutting surgery as part of their usual treatment.
Trial locations and contacts
1 site in 1 country.
Israel1 site, 1 recruiting
- Recruiting
Hillel Yaffe Medical Center
Hadera
Ilana Alony, Mrs, +972-52-3747719 ext. +97246304549, IlanaA@hy.health.gov.il
Itzhak Braverman, Doctor (MD)
About this study
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.
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Oculopharyngeal
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Data will be collected within four years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2019
- Study start19 Mar 2019
- Primary completionMar 2023 (estimated)
- Study completionMar 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Oculopharyngeal Muscular DystrophyStudies of the same condition, open and under way.
- 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
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hillel Yaffe Medical Center
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Itzhak Braverman, Doctor (MD), Principal investigator, Hillel Yaffe Medical Center, Israel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03874910
- Sponsor's protocol ID
- 0115-18-HYMC
- Status last verified
- Mar 2019
- First submitted
- 4 Mar 2019
- Last updated
- 21 Mar 2019
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pathology Analysis of OPMD Patient Myotomies
- ClinicalTrials.gov ID
- NCT03874910
- Acronym / sponsor ID
- 0115-18-HYMC
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Hillel Yaffe Medical CenterOther government
- Study start
- 19 Mar 2019 (actual)
- Primary completion
- Mar 2023 (estimated)
- Study completion
- Mar 2023 (estimated)
- First posted
- 14 Mar 2019 (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 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.
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients who suffer from oculopharyngeal muscular dystrophy (OPMD).
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (1 listed)
- OPMD patients not referred to the cricopharyngeal muscle-cutting surgery as part of their usual treatment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of genes that show changes in RNA profile, measured by sequencing.
Time frame: Data will be collected within four years.
RNA profile analysis of OPMD muscle samples will be compared to unaffected muscle samples using sequencing to identify gene numbers.
Number of genes that show changes in protein profile, measured by SDS page.
Time frame: Data will be collected within four years.
Protein profile analysis of OPMD muscle samples will be compared to unaffected muscle samples to identify gene numbers using SDS page.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2019.
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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. This study is not recruiting at the moment.
Itzhak Braverman, Doctor (MD) principal investigator
Hillel Yaffe Medical Center, Israel
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, run by Hillel Yaffe 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 3 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: status not confirmed, as of the sponsor's last update on 21 Mar 2019. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Itzhak Braverman, Doctor (MD) (+972-4-6304549 ext. +97246304549, braverman@hy.health.gov.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of genes that show changes in RNA profile, measured by sequencing. (time frame: Data will be collected within four years.). The study lists 2 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 21 Mar 2019; AzTrials last refreshed its copy on 7 Nov 2026.