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NH-OPMD: Natural History Study of Oculopharyngeal Muscular Dystrophy

Sponsor
University of New Mexico (Other)
Overall Status
Withdrawn
CT.gov ID
NCT03161847
Collaborator
University of Florida (Other)
0
Enrollment
19.2
Actual Duration (Months)

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase
  • Other: Non-interventional study

Detailed Description

OPMD patients will undergo a screening evaluation and testing to confirm the participants carry the OPMD mutation. Subjects fulfilling the inclusion/exclusion criteria will be enrolled and followed prospectively at regular intervals to determine the natural history of this disease. Measures of muscle function and swallowing will be made at baseline and at follow-up visits to measure natural clinical progression.

Study Design

Study Type:
Observational
Actual Enrollment :
0 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Natural History Study of Oculopharyngeal Muscular Dystrophy
Actual Study Start Date :
Dec 12, 2016
Actual Primary Completion Date :
Jul 18, 2018
Actual Study Completion Date :
Jul 18, 2018

Arms and Interventions

Arm Intervention/Treatment
OPMD Subjects

The study cohort consists of individuals with genetically confirmed OPMD who will be followed longitudinally using periodic standardized assessments of clinical status in an observational, non-interventional study.

Other: Non-interventional study
Non-interventional study

Outcome Measures

Primary Outcome Measures

  1. Muscle Strength over time [Baseline and every 9 months for 3 years]

    Change in Manual muscle testing over time

Secondary Outcome Measures

  1. Dysphagia severity over time [Baseline and every 9 months for 3 years]

    Videofluoroscopic Swallow Studies

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 99 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • University of New Mexico
  • University of Florida

Investigators

  • Principal Investigator: Sarah Youssof, MD, MS, New Mexico Neuromuscular Center

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
University of New Mexico
ClinicalTrials.gov Identifier:
NCT03161847
Other Study ID Numbers:
  • 16-326
First Posted:
May 22, 2017
Last Update Posted:
May 27, 2022
Last Verified:
May 1, 2017
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by University of New Mexico
OPMD
Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Oculopharyngeal

Study Results

No Results Posted as of May 27, 2022