Facioscapulohumeral Muscular DystrophyReSolveNCT03458832
Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
An observational study of Facioscapulohumeral Muscular Dystrophy, run by University of Kansas Medical Center at 14 sites in 6 countries.
Trial summary
The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy. As multiple drug companies pursue treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy
- What is being tested?
- FSHD-specific functional rating scale and Electrical Impedance Myography
- Type of treatment
- Medical device
- How many people
- 324 participants
- Where
- 14 sitesin 6 countries
- Study dates
- 5 Mar 2018to Dec 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Participants with FSHD that are seen in the researchers clinic.
Who may be able to join
- Patients with genetically confirmed FSHD1 or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring
- Patients with symptomatic limb weakness
- Patients must be able to walk 30 feet without the support of another person or assistance (canes, walking sticks, and braces allowed; no walker).
- If taking over the counter supplements, willing to remain consistent with supplement regimen throughout the course of the study
Who cannot join
- Patients with cardiac or respiratory dysfunction (deemed clinically unstable, or would interfere with safe testing, in the opinion of the Investigator)
- Patients with orthopedic conditions that preclude safe testing of muscle function
- Patients that regularly use available muscle anabolic/catabolic agents such as corticosteroids, oral testosterone or derivatives, or oral beta agonists
- Patients that have used an experimental drug in an FSHD clinical trial within the past 30 days
- Patients that are pregnant
Trial locations and contacts
14 sites in 6 countries.
14 sites in 6 countries
United States8 sites
Kennedy Krieger Institute
Baltimore, Maryland, 21205
The Ohio State University
Columbus, Ohio, 43210
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of California Los Angeles
Los Angeles, California, 90095
Virginia Commonwealth University
Richmond, Virginia, 23298
University of Rochester Medical Center
Rochester, New York, 14642
University of Utah
Salt Lake City, Utah, 84132
University of Washington
Seattle, Washington, 98195
United Kingdom1 site
University of College London - Queens Square
London, WC1N 3BG
About this study
The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy. As multiple drug companies pursue treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development, including creating disease-relevant outcome measures and optimizing inclusion criteria. This proposal will develop two new outcome measures (FSHD-COM and EIM) and optimize eligibility criteria by testing 320 patients across 14 international sites over a period of 24 months.
Read the detailed description
The overall aim of this study is to hasten drug development for facioscapulohumeral muscular dystrophy (FSHD). Recent breakthroughs in FSHD research have identified the primary disease mechanism as the aberrant expression of a normally silenced gene, DUX4, resulting in a toxic gain-of-function. This disease mechanism is particularly amenable to knock-down of DUX4 using epigenetic strategies or RNA therapies, as well as to other interventions targeting the downstream effects of DUX4 expression. There are many drug companies actively working towards disease-targeted therapies, and two clinical trials either under way now, or planned to start in early Fall 2016. However, meetings with industry, advocacy groups, and FSHD researchers have identified several gaps in the clinical trial arsenal, and clinical trial planning as a major goal for the community. Consequently, there is an urgent need to establish the tools necessary for the conduct of currently planned and expected therapeutic trials in FSHD.
To this end, the researchers propose to develop two novel clinical outcome assessments (COA), a composite functional outcome measure (FSHD-COM) and skeletal muscle biomarker, electrical impedance myography (EIM). In addition, there is broad consensus a better understanding of the relationship of genetic and demographic features to disease progression will be necessary for enumerating eligibility criteria.
The specific aims are to: 1. Determine the multi-site validity of the COAs, 2. Compare the responsiveness of new COAs to other FSHD outcomes and determine the minimal clinically meaningful changes, and 3. establish FSHD cohort characteristics useful for determining clinical trial eligibility criteria. To achieve these aims, the researchers are conducting a multicenter, prospective, 24 months study of 320 subjects.
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Being tested
- FSHD-specific functional rating scale, Electrical Impedance Myography
- Keywords
- muscular dystrophy., FSHD
- 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.
FSHD-specific functional rating scaleDiagnostic test
Also called FSHD-COM
The FSHD-COM is composed of disease-relevant functional tasks such as leg function; shoulder and arm function; trunk function, hand function, and balance.
Electrical Impedance MyographyDevice
Also called EIM
EIM is a non-invasive, painless, and fast technique for obtaining information on how a patient's muscle structure is changing. EIM uses a small electrical current to measure the health of the underlying muscle. The patient will be asked to lie down and a trained clinical evaluator will perform testing on 16 total muscles (8 on each side) on your arms and legs.
| Study group | Role | What they receive |
|---|---|---|
| FSHD-COM | All participants will be asked to undergo FSHD-specific functional rating scale tests and procedures and Electrical Impedance Myography. Receives: Diagnostic Test: FSHD-specific functional rating scale; Device: Electrical Impedance Myography |
What is being measured
The primary outcome is the main question the study is designed to answer.
FSHD composite (FSHD-COM)
The FSHD composite (FSHD-COM) is an 18-item evaluator-administered instrument comprised of individually validated functional motor tasks. The body regions represented match areas of importance identified by patients and include: leg function; shoulder and arm function; trunk function, hand function; and balance. Each item is scored on a 0-4 scale, with 0 representing unaffected/normal performance, and the divisions based on healthy population normative values, or the relative degree of ability to perform the functional task. The total scale has 72 points, with larger weight given to the two most frequently patient-cited areas of functional motor concern - leg function and shoulder and arm function.
Measured: 24 Months
Electrical Impedance Myography (EIM)
EIM is administered using an investigational device manufactured by Skulpt, Inc (Boston, MA) that non-invasively measures the impedance of skeletal muscle over a frequency range between 1 kHz and 10 MHz (Figure 2). The impedance is measured at each frequency by applying low-intensity electrical current (<1 mA) via surface electrodes and measuring the resulting voltage signals using a second set of surface electrodes, converting them into 2 impedance parameters, the resistance and the reactance.
Measured: 24 Months
Secondary outcomes (17)
Motor Function Measure (MFM) Domain 1
The MFM domain 1 is a validated evaluator administered functional measure for neuromuscular disorders, with 13 items related to standing and transfers.
Measured: 24 Months
Facial Function
The Iowa Oral Performance Instrument (IOPI) is a means to quantify lip, tongue, and buccal strength using a validated tool with published ranges for normative data for lingual measurements.
Measured: 24 Months
Reachable Workspace (RWS)
Subjects are seated in front of a 3D camera and asked to perform a standardized upper extremity movement protocol under the supervision of a study clinical evaluator.
Measured: 24 Months
Manual Muscle Testing (MMT)
Strength testing will be performed using manual muscle testing (MMT) using a hand held force dynamometer.
Measured: 24 Months
Force Vital Capacity (FVC)
The researchers will obtain bedside spirometry including forced vital capacity and forced expiratory volume in 1 second.
Measured: 24 Months
Maximum Inspiratory Pressure (MIP) and Maximum Expiratory Pressure (MEP)
The researchers will obtain maximum inspiratory pressure (MIP) and maximum expiratory pressure (MEP) using bedside spirometry.
Measured: 24 Months
Whole body and regional lean muscle mass (LMM)
Whole body and regional lean muscle mass (LMM) will be measured via Dual Energy X-Ray Absorptiometry (DEXA).
Measured: 24 Months
Severity Scores
A limited physical exam and strength testing will be used to derive two FSHD clinical severity scores. These severity scores both rank weakness in the face, shoulders, arms, distal, and proximal lower extremities on either a 10 or 15 point scale.
Measured: 24 Months
FSHD-Health Index (FSHD-HI)
The HI is a 15 domain questionnaire designed and based on patient interviews to measure total FSHD health-related quality-of-life, including both motor impairment and the social and emotional impact of FSHD. 116 questions are combined into a total score, the score is transformed onto a percentage scale, with 100 representing maximal disability, and lower scores representing decreasing disability.
Measured: 24 Months
Patient-Reported Outcomes Measurement Information System-57 (PROMIS57)
The PROMIS57 is an instrument developed by the NIH which generates scores for physical function, and the impact of physical limitations on daily life. 57 questions are summed into a total score, which is transformed into a normalized t-score with 50 representing normal, and lower scores representing increasing disability.
Measured: 24 Months
The Upper Extremity Functional Index
This index measures upper extremity dysfunction. 20 questions are combined into a total score, the score is transformed into a normalized score with 80 representing normal, and lower scores representing increasing disability.
Measured: 24 Months
The Facial Disability Index (FDI)
The FDI is a short 5 item questionnaire. The five questions are summed into total score which transformed onto a percentage scale, with 100 representing normal, and lower scores representing increasing disability.
Measured: 24 Months
Fall assessment
Fall assessment will be completed weekly for 3 months after the month 3 visit.
Measured: Total between Month 3 and Month 6 Visit
Domain Delta Anchor
Participants will be asked to complete a self-assessment "domain-delta" questionnaire at 3, 12, 18 and 24-month visits. The purpose of the "domain-delta" questionnaire is to determine each patient's perceived change in their health-related quality-of-life in the last 6-months. This questionnaire will inquire about total health as well as health related to 14 subdomains self-identified as important by patients during development of the FSHD Health Index. Participants indicate their perceived change by answering if an area "is a lot worse", "is a little worse", "there has been no change", "it is a little better", or "it is a lot better" for each subdomain.
Measured: 24 Months
Quantitative myometry (QMA)
Force will be measured on digital myometer, in KG-force.
Measured: 24 Months
Muscle Biopsy
Will be performed on 30 subjects at European and United Kingdom sites only. Participants will undergo one needle muscle biopsy of one lower extremity muscle (Vastus Lateralis (VL), Tibialis Anterior (TA), Medial Gastrocnemius (MG), or Lateral Gastrocnemius (LG)) at any study visit. For each biopsy, approximately three muscle tissue samples will be collected, two samples will be individually flash frozen in liquid nitrogen while the third sample will be put into media for myoblast cell cultures.
Measured: 24 Months
Blood biomarkers
DNA and RNA will be collected at Baseline and 3-month visits. Plasma and serum samples will be collected at every visit (Baseline, 3, 12, 18, and 24-months).
Measured: 24-Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2018
- Study start5 Mar 2018
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- 324 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), FSHD Society, Friends Research Institute, Inc., Muscular Dystrophy Association, AFM Telethon, University of Rochester, Leiden University Medical Center, Dyne Therapeutics
- Responsible party
- Sponsor
- Investigators
- Jeffrey Statland, MD, Principal investigator, University of Kansas Medical CenterRabi Tawil, MD, Principal investigator, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03458832
- Sponsor's protocol ID
- STUDY00140842
- Other IDs
- U01NS101944
- Acronym
- ReSolve
- Status last verified
- Sep 2026
- First submitted
- 11 Jan 2018
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Undecided
Publications
LoRusso S, Johnson NE, McDermott MP, Eichinger K, Butterfield RJ, Carraro E, Higgs K, Lewis L, Mul K, Sacconi S, Sansone VA, Shieh P, van Engelen B, Wagner K, Wang L, Statland JM, Tawil R; ReSolve Investigators and the FSHD CTRN18. Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study. BMC Neurol. 2019 Sep 10;19(1):224. doi: 10.1186/s12883-019-1452-x.
PubMed 31506080
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
- ClinicalTrials.gov ID
- NCT03458832
- Acronym / sponsor ID
- ReSolve / STUDY00140842
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Keywords
- muscular dystrophy., FSHD
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 324 participants (actual)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), FSHD Society, Friends Research Institute, Inc., Muscular Dystrophy Association, AFM Telethon, University of Rochester, Leiden University Medical Center, Dyne Therapeutics
- Study start
- 5 Mar 2018 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 8 Mar 2018 (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 primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy. As multiple drug companies pursue treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development, including creating disease-relevant outcome measures and optimizing inclusion criteria. This proposal will develop two new outcome measures (FSHD-COM and EIM) and optimize eligibility criteria by testing 320 patients across 14 international sites over a period of 24 months.
Detailed description
The overall aim of this study is to hasten drug development for facioscapulohumeral muscular dystrophy (FSHD). Recent breakthroughs in FSHD research have identified the primary disease mechanism as the aberrant expression of a normally silenced gene, DUX4, resulting in a toxic gain-of-function. This disease mechanism is particularly amenable to knock-down of DUX4 using epigenetic strategies or RNA therapies, as well as to other interventions targeting the downstream effects of DUX4 expression. There are many drug companies actively working towards disease-targeted therapies, and two clinical trials either under way now, or planned to start in early Fall 2016. However, meetings with industry, advocacy groups, and FSHD researchers have identified several gaps in the clinical trial arsenal, and clinical trial planning as a major goal for the community. Consequently, there is an urgent need to establish the tools necessary for the conduct of currently planned and expected therapeutic trials in FSHD.
To this end, the researchers propose to develop two novel clinical outcome assessments (COA), a composite functional outcome measure (FSHD-COM) and skeletal muscle biomarker, electrical impedance myography (EIM). In addition, there is broad consensus a better understanding of the relationship of genetic and demographic features to disease progression will be necessary for enumerating eligibility criteria.
The specific aims are to: 1. Determine the multi-site validity of the COAs, 2. Compare the responsiveness of new COAs to other FSHD outcomes and determine the minimal clinically meaningful changes, and 3. establish FSHD cohort characteristics useful for determining clinical trial eligibility criteria. To achieve these aims, the researchers are conducting a multicenter, prospective, 24 months study of 320 subjects.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FSHD-COM | – | Diagnostic Test: FSHD-specific functional rating scale, Device: Electrical Impedance MyographyAll participants will be asked to undergo FSHD-specific functional rating scale tests and procedures and Electrical Impedance Myography. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Participants with FSHD that are seen in the researchers clinic.
Key inclusion criteria (4 listed)
- Patients with genetically confirmed FSHD1 or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring
- Patients with symptomatic limb weakness
- Patients must be able to walk 30 feet without the support of another person or assistance (canes, walking sticks, and braces allowed; no walker).
- If taking over the counter supplements, willing to remain consistent with supplement regimen throughout the course of the study
Key exclusion criteria (5 listed)
- Patients with cardiac or respiratory dysfunction (deemed clinically unstable, or would interfere with safe testing, in the opinion of the Investigator)
- Patients with orthopedic conditions that preclude safe testing of muscle function
- Patients that regularly use available muscle anabolic/catabolic agents such as corticosteroids, oral testosterone or derivatives, or oral beta agonists
- Patients that have used an experimental drug in an FSHD clinical trial within the past 30 days
- Patients that are pregnant
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 |
|---|---|---|
| FSHD-specific functional rating scaleAlso: FSHD-COMThe FSHD-COM is composed of disease-relevant functional tasks such as leg function; shoulder and arm function; trunk function, hand function, and balance. | Diagnostic test | FSHD-COM |
| Electrical Impedance MyographyAlso: EIMEIM is a non-invasive, painless, and fast technique for obtaining information on how a patient's muscle structure is changing. EIM uses a small electrical current to measure the health of the underlying muscle. The patient will be asked to lie down and a trained clinical evaluator will perform testing on 16 total muscles (8 on each side) on your arms and legs. | Device | FSHD-COM |
Primary outcome
FSHD composite (FSHD-COM)
Time frame: 24 Months
The FSHD composite (FSHD-COM) is an 18-item evaluator-administered instrument comprised of individually validated functional motor tasks. The body regions represented match areas of importance identified by patients and include: leg function; shoulder and arm function; trunk function, hand function; and balance. Each item is scored on a 0-4 scale, with 0 representing unaffected/normal performance, and the divisions based on healthy population normative values, or the relative degree of ability to perform the functional task. The total scale has 72 points, with larger weight given to the two most frequently patient-cited areas of functional motor concern - leg function and shoulder and arm function.
Electrical Impedance Myography (EIM)
Time frame: 24 Months
EIM is administered using an investigational device manufactured by Skulpt, Inc (Boston, MA) that non-invasively measures the impedance of skeletal muscle over a frequency range between 1 kHz and 10 MHz (Figure 2). The impedance is measured at each frequency by applying low-intensity electrical current (<1 mA) via surface electrodes and measuring the resulting voltage signals using a second set of surface electrodes, converting them into 2 impedance parameters, the resistance and the reactance.
17 secondary outcomes
Motor Function Measure (MFM) Domain 1
Time frame: 24 Months
Facial Function
Time frame: 24 Months
Reachable Workspace (RWS)
Time frame: 24 Months
Manual Muscle Testing (MMT)
Time frame: 24 Months
Force Vital Capacity (FVC)
Time frame: 24 Months
Maximum Inspiratory Pressure (MIP) and Maximum Expiratory Pressure (MEP)
Time frame: 24 Months
Whole body and regional lean muscle mass (LMM)
Time frame: 24 Months
Severity Scores
Time frame: 24 Months
FSHD-Health Index (FSHD-HI)
Time frame: 24 Months
Patient-Reported Outcomes Measurement Information System-57 (PROMIS57)
Time frame: 24 Months
The Upper Extremity Functional Index
Time frame: 24 Months
The Facial Disability Index (FDI)
Time frame: 24 Months
Fall assessment
Time frame: Total between Month 3 and Month 6 Visit
Domain Delta Anchor
Time frame: 24 Months
Quantitative myometry (QMA)
Time frame: 24 Months
Muscle Biopsy
Time frame: 24 Months
Blood biomarkers
Time frame: 24-Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Derived from this study: PubMed 31506080LoRusso S, Johnson NE, McDermott MP, Eichinger K, Butterfield RJ, Carraro E, Higgs K, Lewis L, Mul K, Sacconi S, Sansone VA, Shieh P, van Engelen B, Wagner K, Wang L, Statland JM, Tawil R; ReSolve Investigators and the FSHD CTRN18. Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study. BMC Neurol. 2019 Sep 10;19(1):224. doi: 10.1186/s12883-019-1452-x.
- Individual participant dataSharing planned: Undecided
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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
Rabi Tawil, MD principal investigator
University of Rochester
14 sites in 6 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 University of Kansas Medical Center at 14 sites in 6 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 5 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: under way, not looking for participants, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, France, Germany, Italy, Netherlands and 1 more country. 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?
This study is still under way. Results are not available in the source record yet.
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 composite (FSHD-COM) (time frame: 24 Months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03458832, which also lists 1 linked publication.
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 17 Sep 2026.