Natural History Study of Oculopharyngeal Muscular Dystrophy | NCT03161847

Studies
Natural History Study of Oculopharyngeal Muscular Dystrophy

NCT03161847

Sponsor

University of New Mexico

Status

Withdrawn

Last Update Posted

May 27, 2022Actual

Enrollment

0Actual

Phase

Not provided

Conditions

Oculopharyngeal Muscular Dystrophy

Interventions

Non-interventional study

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2017

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

No subjects were enrolled

Expanded Access Info

Start Date

Dec 12, 2016Actual

Primary Completion Date

Jul 18, 2018Actual

Completion Date

Jul 18, 2018Actual

First Submitted Date

May 16, 2017

First Submission Date that Met QC Criteria

May 18, 2017

First Posted Date

May 22, 2017Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 23, 2022

Last Update Posted Date

May 27, 2022Actual

Name	Class
University of Florida	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Non-interventional study	Other	Non-interventional study	OPMD Subjects

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Muscle Strength over time	Change in Manual muscle testing over time	Baseline and every 9 months for 3 years

Secondary Outcomes

Measure	Description	Time Frame
Dysphagia severity over time	Videofluoroscopic Swallow Studies	Baseline and every 9 months for 3 years

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

99 Years

Standard Ages

AdultOlder Adult

Study Population

Genetically Confirmed Oculopharyngeal Muscular Dystrophy

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Sarah Youssof, MD, MS	New Mexico Neuromuscular Center	Principal Investigator

Locations

Not provided

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D039141	Muscular Dystrophy, Oculopharyngeal

Ancestor Terms

ID	Term
D009136	Muscular Dystrophies
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases

Browse Leaves

Not provided

Browse Branches

Not provided