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Natural History Study of Oculopharyngeal Muscular Dystrophy (NH-OPMD)

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ClinicalTrials.gov Identifier: NCT03161847
Recruitment Status : Not yet recruiting
First Posted : May 22, 2017
Last Update Posted : May 22, 2017
Sponsor:
Collaborator:
University of Florida
Information provided by (Responsible Party):
University of New Mexico

Tracking Information
First Submitted Date May 16, 2017
First Posted Date May 22, 2017
Last Update Posted Date May 22, 2017
Estimated Study Start Date July 2017
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 18, 2017)
Muscle Strength over time [ Time Frame: Baseline and every 9 months for 3 years ]
Change in Manual muscle testing over time
Original Primary Outcome Measures Same as current
Change History No Changes Posted
Current Secondary Outcome Measures
 (submitted: May 18, 2017)
Dysphagia severity over time [ Time Frame: Baseline and every 9 months for 3 years ]
Videofluoroscopic Swallow Studies
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Natural History Study of Oculopharyngeal Muscular Dystrophy
Official Title Natural History Study of Oculopharyngeal Muscular Dystrophy
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.
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 Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Genetically Confirmed Oculopharyngeal Muscular Dystrophy
Condition Oculopharyngeal Muscular Dystrophy
Intervention Other: Non-interventional study
Non-interventional study
Study Groups/Cohorts 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.
Intervention: Other: Non-interventional study
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Not yet recruiting
Estimated Enrollment
 (submitted: May 18, 2017)
60
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 2024
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria

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
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 99 Years   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Sarah Youssof, MD, MS 505-272-6354 syoussof@salud.unm.edu
Listed Location Countries Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number NCT03161847
Other Study ID Numbers 16-326
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party University of New Mexico
Study Sponsor University of New Mexico
Collaborators University of Florida
Investigators
Principal Investigator: Sarah Youssof, MD, MS University of New Mexico School of Medicine
PRS Account University of New Mexico
Verification Date May 2017