Facioscapulohumeral Muscular DystrophyROC-FSHDNCT02766985
Rasch-analysis of Clinical Severity in FSHD
An observational study of Facioscapulohumeral Muscular Dystrophy, run by University of Kansas Medical Center at 2 sites in United States.
Trial summary
The purpose of this study is to make a standardized and scalable Rasch-built clinical severity scale to help in finding genetic and environmental modifiers of disease in Facioscapulohumeral muscular dystrophy (FSHD). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy
- How many people
- 44 participants
- Where
- 2 sitesUnited States
- Study dates
- Mar 2016to 18 Apr 2017
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Particpants with genetically verified FSHD types 1 and 2.
Who may be able to join
- Genetic confirmation of FSHD (either FSHD-1 or FSHD-2)
Who cannot join
- Do not understand English
- Unable to provide informed consent
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Utah
Salt Lake City, Utah, 84112
About this study
The purpose of this study is to make a standardized and scalable Rasch-built clinical severity scale to help in finding genetic and environmental modifiers of disease in Facioscapulohumeral muscular dystrophy (FSHD).
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Keywords
- FSHD, muscular dystrophy
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| FSHD | Participants with FSHD-1 or FSHD-2. No intervention is given to participants. Participants will undergo series of tests and procedures in order to make a standardized and scalable Rasch-built clinical severity scale. |
What is being measured
The primary outcome is the main question the study is designed to answer.
FSHD Clinical Severity Scale
Measure severity of FSHD.
Measured: 1 day
Secondary outcomes (8)
Brooks scale
Measures upper extremity function.Scores on the scale range from 1-6. A score of 1 means the person can elevate their arms above their head while arms remain straight. 6 means the person cannot elevate their arms to their mouth and do not have useful function of their hands.
Measured: 1 day
Vignos scale
Measures lower extremity function. Scores on the scale range from 1-10. A score of 1 means the person can walk and climb stairs without assistance. A score of 10 means the person is bedridden.
Measured: 1 day
Iowa Oral Performance Instrument (IOPI)
IOPI measures lip and tongue strength by measuring the amount of pressure a person can exert on the tool.
Measured: 1 day
Manual muscle testing
Measure person's range of motion. Scored on a scale with range of 0-5. Score of 0 means no movement. Score of 5 means the person has a full range of movement.
Measured: 1 day
Quantitative strength testing
Measured: 1 day
Functional testing
Measured: 1 day
Reparatory function testing
Measured: 1 day
Patient-reported outcome
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2016 (estimated)
- Study startMar 2016
- Primary completion18 Apr 2017
- Study completion18 Apr 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Facioscapulohumeral Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University of Kansas Medical CenterThe sponsor's other registered studies.
- 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
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH)
- Responsible party
- Sponsor
- Investigators
- Jeffrey Statland, MD, Principal investigator, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02766985
- Sponsor's protocol ID
- STUDY00003640
- Other IDs
- UL1TR000001
- Acronym
- ROC-FSHD
- Status last verified
- Apr 2018
- First submitted
- 22 Mar 2016
- Last updated
- 12 Apr 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rasch-analysis of Clinical Severity in FSHD
- ClinicalTrials.gov ID
- NCT02766985
- Acronym / sponsor ID
- ROC-FSHD / STUDY00003640
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Keywords
- FSHD, muscular dystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH)
- Study start
- Mar 2016
- Primary completion
- 18 Apr 2017 (actual)
- Study completion
- 18 Apr 2017 (actual)
- First posted
- 10 May 2016 (estimated)
- 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 purpose of this study is to make a standardized and scalable Rasch-built clinical severity scale to help in finding genetic and environmental modifiers of disease in Facioscapulohumeral muscular dystrophy (FSHD).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FSHD | – | –Participants with FSHD-1 or FSHD-2. No intervention is given to participants. Participants will undergo series of tests and procedures in order to make a standardized and scalable Rasch-built clinical severity scale. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Particpants with genetically verified FSHD types 1 and 2.
Key inclusion criteria (1 listed)
- Genetic confirmation of FSHD (either FSHD-1 or FSHD-2)
Key exclusion criteria (2 listed)
- Do not understand English
- Unable to provide informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
FSHD Clinical Severity Scale
Time frame: 1 day
Measure severity of FSHD.
8 secondary outcomes
Brooks scale
Time frame: 1 day
Vignos scale
Time frame: 1 day
Iowa Oral Performance Instrument (IOPI)
Time frame: 1 day
Manual muscle testing
Time frame: 1 day
Quantitative strength testing
Time frame: 1 day
Functional testing
Time frame: 1 day
Reparatory function testing
Time frame: 1 day
Patient-reported outcome
Time frame: 1 day
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2018.
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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.
Jeffrey Statland, MD principal investigator
University of Kansas Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Facioscapulohumeral Muscular Dystrophy, run by University of Kansas Medical Center at 2 sites in United States.
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 1 inclusion and 2 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: finished, as of the sponsor's last update on 12 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
FSHD Clinical Severity Scale (time frame: 1 day). 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 12 Apr 2018.