WithdrawnNot Applicable

Natural History Study of Oculopharyngeal Muscular Dystrophy

Stopped: No subjects were enrolled

0Started 2016-12-12

Plain-language summary

The objective of this study is to test a set of clinical outcome measures longitudinally in a cohort of OPMD patients to identify ones that show quantifiable change over time as the disease progresses. The investigators' goal is to delineate the natural history of OPMD.

Who can participate

Age range

18 Years – 99 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * OPMD by genetic criteria * ≥ 18 years old * English-speaking Exclusion Criteria: * Another medical condition that precludes safe completion of study tasks (such as severe cardiac or respiratory disease) * Another medical condition that causes symptoms similar to OPMD (i.e., ptosis, dysphagia \[trouble swallowing\] or limb weakness). * History of head or neck cancer, or history of radiation to the head or neck * A videofluoroscopic swallow study within the 12 months prior to study enrollment

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Muscle Strength over time

Timeframe: Baseline and every 9 months for 3 years

Trial details

NCT IDNCT03161847

SponsorUniversity of New Mexico

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2018-07-18

View on ClinicalTrials.gov More Oculopharyngeal Muscular Dystrophy trials

Who can participate

Age range

18 Years – 99 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.