Facioscapulohumeral Muscular DystrophyFSHDNCT02413190
Bone Health in Facioscapulohumeral Muscular Dystrophy
An observational study of Facioscapulohumeral Muscular Dystrophy, run by Hugo W. Moser Research Institute at Kennedy Krieger, Inc. at 2 sites in 2 countries.
Trial summary
This is a cross-sectional single visit study to determine bone health in individuals with FSHD. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy
- How many people
- 94 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Jul 2014to Aug 2016
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The population consists of individuals with Facioscapulohumeral Muscular Dystrophy.
Who may be able to join
- Genetic diagnosis of FSHD including chromosome 4 deletion and haplotype
- Age > 18 years
- Ability to provide written informed consent for participation in the study
- Ability to participate in the DEXA scan
Who cannot join
- Unwillingness or inability to comply with the requirements of this protocol (in the opinion of the PI) including, but not limited to, the presence of any condition (physical, mental or social) that precludes the participant from comfortably and safely obtaining a DEXA scan, phlebotomy, or neurological examination
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Kennedy Krieger Institute, Johns Hopkins School of Medicine
Baltimore, Maryland, 21205
Australia1 site
Concord Hospital Neurology Department, Hospital Road, Concord NSW 2139
Sydney
About this study
Official title: Bone Health in Facioscapulohumeral Muscular Dystrophy: A Cross-sectional Study
This is a cross-sectional single visit study to determine bone health in individuals with FSHD.
Read the detailed description
Bone health will be assessed using 1) a DEXA scan to measure bone mineral density and lean body mass, and 2) blood tests of biomarkers of bone resorption and formation. These procedures will be correlated to measures of muscle strength testing and timed function tests to determine fracture risk in FSHD individuals considering age, gender, muscle strength and FSHD allele size.
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral
What is being measured
The primary outcome is the main question the study is designed to answer.
Bone Mineral Density in FSHD
To determine if bone mineral density is reduced in individuals with FSHD compared to normative data of individuals of the same age and gender without FSHD.The Bone Mineral Density Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). Negative numbers indicate values lower than the reference population and positive numbers indicate values higher than the reference population
Measured: Single visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2015 (estimated)
- Study startJul 2014
- Primary completionJul 2015
- Study completionAug 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Facioscapulohumeral 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
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 94 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Sponsor type
- University, hospital or other
- Collaborators
- Concord Hospital, FSHD Global Research Foundation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02413190
- Sponsor's protocol ID
- IRB00031738
- Acronym
- FSHD
- Status last verified
- Feb 2019
- First submitted
- 6 Apr 2015
- Last updated
- 5 Nov 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Bone Health in Facioscapulohumeral Muscular Dystrophy: A Cross-sectional Study
- ClinicalTrials.gov ID
- NCT02413190
- Acronym / sponsor ID
- FSHD / IRB00031738
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 94 participants (actual)
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.University, hospital or other
- Collaborators
- Concord Hospital, FSHD Global Research Foundation
- Study start
- Jul 2014
- Primary completion
- Jul 2015 (actual)
- Study completion
- Aug 2016 (actual)
- First posted
- 9 Apr 2015 (estimated)
- Results
- Posted 5 Nov 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a cross-sectional single visit study to determine bone health in individuals with FSHD.
Detailed description
Bone health will be assessed using 1) a DEXA scan to measure bone mineral density and lean body mass, and 2) blood tests of biomarkers of bone resorption and formation. These procedures will be correlated to measures of muscle strength testing and timed function tests to determine fracture risk in FSHD individuals considering age, gender, muscle strength and FSHD allele size.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The population consists of individuals with Facioscapulohumeral Muscular Dystrophy.
Key inclusion criteria (4 listed)
- Genetic diagnosis of FSHD including chromosome 4 deletion and haplotype
- Age > 18 years
- Ability to provide written informed consent for participation in the study
- Ability to participate in the DEXA scan
Key exclusion criteria (1 listed)
- Unwillingness or inability to comply with the requirements of this protocol (in the opinion of the PI) including, but not limited to, the presence of any condition (physical, mental or social) that precludes the participant from comfortably and safely obtaining a DEXA scan, phlebotomy, or neurological examination
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
Bone Mineral Density in FSHD
Time frame: Single visit
To determine if bone mineral density is reduced in individuals with FSHD compared to normative data of individuals of the same age and gender without FSHD.The Bone Mineral Density Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). Negative numbers indicate values lower than the reference population and positive numbers indicate values higher than the reference population
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: 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 Hugo W. Moser Research Institute at Kennedy Krieger, Inc. at 2 sites in 2 countries.
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 4 inclusion and 1 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 5 Nov 2019. 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, Australia. 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?
Yes. Results are posted in the registry (first posted 5 Nov 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Bone Mineral Density in FSHD (time frame: Single visit). 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 5 Nov 2019.