Oculopharyngeal Muscular DystrophyNH-OPMDNCT03161847
Natural History Study of Oculopharyngeal Muscular Dystrophy
An observational study of Oculopharyngeal Muscular Dystrophy, run by University of New Mexico.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Oculopharyngeal Muscular Dystrophy
- What is being tested?
- Non-interventional study
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 12 Dec 2016to 18 Jul 2018
- 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 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Genetically Confirmed Oculopharyngeal Muscular Dystrophy
Who may be able to join
- OPMD by genetic criteria
- ≥ 18 years old
- English-speaking
Who cannot join
- 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
About this study
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.
Read the 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.
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Being tested
- Non-interventional study
- Keywords
- OPMD
- Medical subject headings
- Muscular Dystrophy, Oculopharyngeal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Non-interventional studyOther
Non-interventional study
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: Non-interventional study |
What is being measured
The primary outcome is the main question the study is designed to answer.
Muscle Strength over time
Change in Manual muscle testing over time
Measured: Baseline and every 9 months for 3 years
Secondary outcomes (1)
Dysphagia severity over time
Videofluoroscopic Swallow Studies
Measured: Baseline and every 9 months for 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2017
- Study start12 Dec 2016
- Primary completion18 Jul 2018
- Study completion18 Jul 2018
Why it stopped: No subjects were enrolled
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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of New Mexico
- Sponsor type
- University, hospital or other
- Collaborators
- University of Florida
- Responsible party
- Sponsor
- Investigators
- Sarah Youssof, MD, MS, Principal investigator, New Mexico Neuromuscular Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03161847
- Sponsor's protocol ID
- 16-326
- Acronym
- NH-OPMD
- Status last verified
- May 2017
- First submitted
- 16 May 2017
- Last updated
- 27 May 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History Study of Oculopharyngeal Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03161847
- Acronym / sponsor ID
- NH-OPMD / 16-326
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Keywords
- OPMD
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: No subjects were enrolled
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of New MexicoUniversity, hospital or other
- Collaborators
- University of Florida
- Study start
- 12 Dec 2016 (actual)
- Primary completion
- 18 Jul 2018 (actual)
- Study completion
- 18 Jul 2018 (actual)
- First posted
- 22 May 2017 (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 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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| OPMD Subjects | – | Other: Non-interventional studyThe 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. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Genetically Confirmed Oculopharyngeal Muscular Dystrophy
Key inclusion criteria (3 listed)
- OPMD by genetic criteria
- ≥ 18 years old
- English-speaking
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Non-interventional studyNon-interventional study | Other | OPMD Subjects |
Primary outcome
Muscle Strength over time
Time frame: Baseline and every 9 months for 3 years
Change in Manual muscle testing over time
1 secondary outcome
Dysphagia severity over time
Time frame: Baseline and every 9 months for 3 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2022.
- Individual participant dataSharing planned: No
Related trials for Oculopharyngeal Muscular Dystrophy
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingObservational study
Oral Health, Saliva Viscosity and Composition in Oculo-Pharyngeal Muscular Dystrophy (OPMD)
Oculopharyngeal Muscular Dystrophy
RecruitingPhase 1/2
A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
Oculopharyngeal Muscular Dystrophy
RecruitingObservational study
Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry
Oculopharyngeal Muscular Dystrophy (OPMD)
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.
Sarah Youssof, MD, MS principal investigator
New Mexico Neuromuscular Center
Questions about this trial
What is this clinical trial studying?
An observational study of Oculopharyngeal Muscular Dystrophy, run by University of New Mexico.
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 4 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: withdrawn, as of the sponsor's last update on 27 May 2022. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Muscle Strength over time (time frame: Baseline and every 9 months for 3 years). 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 27 May 2022.