Facioscapulohumeral Muscular DystrophyNCT05178706
Effectiveness of Upper Extremity Rehabilitation in pwFSHD (Patient With Facioscapulohumeral Dystrophia)
An observational study of Facioscapulohumeral Muscular Dystrophy, run by Istanbul University - Cerrahpasa at 1 site in Turkey (Türkiye).
Trial summary
Facioscapulohumeral Muscular Dystrophy (FSHD) is one of the most common forms of muscular dystrophy, characterized by pronounced skeletal weakness and with a broad spectrum of diseases. Read more about this study
- Who is it for?
- Ages 18 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy
- What is being tested?
- upper extremity rehabilitation program and Results of assessment
- How many people
- 36 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 22 Feb 2022to 10 Jun 2022
- 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 50 years
- All sexes
- Healthy volunteers accepted
- Adult
Study population: Patients who have been diagnosed with FSHD, patients who have undergone scapulothoracic arthrodesis, healthy people for control group
Who may be able to join
- Individuals to be included in all groups 18-50 years
- Score between 0.5 and 3.5 according to CSS (Clinical Severity Score) of the cases to be included in the intervention groups.
- Be rated between 3-5 according to FAS (functional ambulation scale) of the cases to be included in intervention groups
- For patients who will be included in the intervention group who have undergone surgery, bilateral scapulothoracic arthrodesis surgery within 3 months at the earliest
- Individuals who will be included in the control group consisting of healthy individuals have not undergone any orthopedic surgery
Who cannot join
- Having received regular physiotherapy for 1 month, at least 2 days a week in the last 6 months
- Presence of additional neurological problem/problems
- Presence of any other upper limb problem/problems and surgery
- Presence of lower limb problems/problems that can cause balance and walking problems
- Having undergone Spinal fusion surgery
- Having lower back pain described 6 and above according to VAS (Visual Analog Scale) in intervention and control groups
- Having a level of visual and auditory problems that will prevent communication
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
Istanbul University-Cerrahpaşa
Istanbul, 34500
About this study
Official title: Does the Upper Extremity Rehabilitation Program Implemented on Individuals With Facioscapulohumeral Dystrophy, Followed by Various Interventions, Have an Impact on Function, Balance, and Gait?
Facioscapulohumeral Muscular Dystrophy (FSHD) is one of the most common forms of muscular dystrophy, characterized by pronounced skeletal weakness and with a broad spectrum of diseases. It is a hereditary disease seen in 3-5/100,000 of society, usually starting with weakness in the facial and shoulder muscles and progressing to the trunk, pelvis and leg muscles, giving symptoms in the twenties. In FSHD, which shows slow progression and can lead to loss of ambulation ability in about 20% of patients, patients may have difficulty performing activities above shoulder level with the influence of the periscapular area.
The goal of FSHD treatment is to improve muscle strength and/or function. Treatments include medical, conservative and surgical methods. The aim of surgical methods is to improve shoulder function and prevent pain caused by the movements of the scapula. The publications on physiotherapy interventions and aerobic exercise are available as conservative treatment. In patients diagnosed with FSHD, conservative treatment is frequently used to improve muscle strength, regulate function and improve the quality of life of patients.
Read more
Patients with FSHD use their affected upper extremities asymmetrically, which leads to the development of restrictive compensation mechanisms in the development of symmetrical postural control. Postural control deficits may occur due to limited use of the affected scapula in individuals with FSHD. Accordingly, in cases with FSHD, there is the use of atypical movements for balance and mobility. It is not yet known whether people with FSHD really have poorer dynamic stability during self-initiated whole-body movements such as walking, and at what stage of the disease these difficulties arise.
Accordingly, the aim of this study was to examine the effects of rehabilitation approaches applied to the upper limb on upper limb function, balance and walking in patients with FSHD.
H1: Within the group of patients with FSHD patients underwent surgery arthrodesis surgery scapulothoracic applied to pre-treatment with the parameters of the rehabilitation program for the evaluation of upper limb functionality after applying the upper extremities, postural control and gait parameters examined, there is statistical difference between the groups.
Read the detailed description
Voluntary participants who have been diagnosed with FSHD and agreed to voluntary health subjects will be included in the study. Signed voluntary consent will be obtained from participants. Participants will be divided into tree groups. One group will include patients who have undergone scapulothoracic arthrodesis surgery, while the other group will include patients who have not undergone surgery and control group will include participants who have agree to participate the study. A progressive rehabilitation program for the upper extremities will be applied to patients in a group of individuals who have undergone and have not undergone surgery. Both groups will undergo some outcome measures to assess their upper extremity function, balance and walking.
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Being tested
- upper extremity rehabilitation program, Results of assessment
- Keywords
- Neuromuscular Disease, Scapulothoracic Arthrodesis, Exercise Therapy, Kinetic Chain
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
upper extremity rehabilitation programOther
Patients with FSHD who have undergone and have not undergone surgery will be given activity training by the same physiotherapist after they agree to participate in the study. Both groups will be given the same exercises, the same number of repetitions and the same frequency. A two-phase exercise protocol will be established and progression will be performed after 4 weeks. The exercises will be based on the upper extremity and will include various types of exercises and the primary affected November muscles in the upper extremity. It is planned that the exercises will be accompanied by a physiotherapist 2 days a week, with a total of 16 sessions for 8 weeks. It is planned that there will be a total of 8-10 exercises in 10 repetitions in the exercise program. The total length of the exercise session will be 30 minutes. It is planned that the same physiotherapist will be one-on-one with the patient from the beginning to the end of the exercises.
Results of assessmentDiagnostic test
The total follow-up period of participants included in intervention programs will be 8 weeks. Evaluations will be evaluated by physiotherapist before and at the end of treatment.
| Study group | Role | What they receive |
|---|---|---|
| non-operative conservative treatment | 9 individuals diagnosed with FSHD who have not undergone unilateral or bilateral surgery who meet the inclusion criteria.
application of determined outcome scales and rehabilitation program on patients Receives: Other: upper extremity rehabilitation program; Diagnostic Test: Results of assessment | |
| scapulothoracic arthrodesis | 9 individuals diagnosed with FSHD who have undergone bilateral surgery who meet the inclusion criteria.
application of determined outcome scales and rehabilitation program on patients Receives: Other: upper extremity rehabilitation program; Diagnostic Test: Results of assessment | |
| healthy control | 18 participants for measuring the normative datas; application of determined outcome scales Receives: Diagnostic Test: Results of assessment |
What is being measured
The primary outcome is the main question the study is designed to answer.
change from baseline 10 Meter Walk Test value at 8 weeks
during the test, the patient is asked to walk at a normal walking speed at a distance of 10 meters determined before the test. The time it has traveled the specified distance is recorded. It is a long-term performance test used for dynamic balance assessment.
Measured: baseline and every four weeks up to eight week
change from baseline Gait Analyzer for walking speed at 8 weeks (m/sec)
It is a smartphone-based application for analyzing walking parameters in real time. It can measure walking speed, step time, step length, cadence and symmetry.
Measured: baseline and every four weeks up to eight week
change from baseline Gait Analyzer for step time at 8 weeks (sec)
It is a smartphone-based application for analyzing walking parameters in real time. It can measure walking speed, step time, step length, cadence and symmetry.
Measured: baseline and every four weeks up to eight week
change from baseline Gait Analyzer for step lenght at 8 weeks (m)
It is a smartphone-based application for analyzing walking parameters in real time. It can measure walking speed, step time, step length, cadence and symmetry.
Measured: baseline and every four weeks up to eight week
change from baseline Gait Analyzer for cadance at 8 weeks (step/min)
It is a smartphone-based application for analyzing walking parameters in real time. It can measure walking speed, step time, step length, cadence and symmetry.
Measured: baseline and every four weeks up to eight week
change from baseline Gait Analyzer for symmetry at 8 weeks (%)
It is a smartphone-based application for analyzing walking parameters in real time. It can measure walking speed, step time, step length, cadence and symmetry.
Measured: baseline and every four weeks up to eight week
Secondary outcomes (8)
change from baseline Timed up and Go Test at 8 weeks
The test is a measure of dynamic equilibrium. It requires individuals to get up from a chair, walk 3 feet, turn and sit. The time from the moment the individual lifts the pelvis from the chair until he or she returns with the pelvis in the chair is recorded in seconds.
Measured: baseline and every four weeks up to eight week
change from baseline Single Leg Stance Test at 8 weeks
It is used to evaluate static posture and balance control. The patient must stand unaided on one leg from the moment the other foot leaves the ground until the foot touches the ground again or the arms are separated from the hips.
Measured: baseline and every four weeks up to eight week
change from baseline 30 Seconds Sit Up Test at 8 weeks
It is a test that evaluates the patient's sit-up activity, lower limb strength and dynamic balance. A 30-second period is initiated when the patient cuts the contact of his pelvis from the chair in which he is sitting. He is constantly asked to sit on the chair and get up from there within 30 seconds. It is scored by counting how many repetitions the patient applies this command within 30 seconds
Measured: baseline and every four weeks up to eight week
change from baseline Hand-Held Dynamometer values at 8 weeks
It is an efficient, objective, precise and affordable alternative to muscle strength measurement. A small portable device is held by the examiner and placed towards the patient's limb during maximum isometric contraction.
Measured: baseline and every four weeks up to eight week
change from baseline Pedometer values at 8 weeks
It is a proven, inexpensive system that helps to measure the steps of individuals with the help of sensors attached to the ankle.
Measured: baseline and every four weeks up to eight week
change from baseline Patient Satisfaction Questionnare at 8 weeks
It is a scale in which the fifth likert scale is used and patients report their satisfaction with the results of the treatments they receive. The points given are between -2 and +2.The higher scores indicate a better result.
Measured: every four weeks up to eight week
Disabilities of Arm, Shoulder and Hand
It is a questionnaire that examines the patient's ability to perform certain upper extremity activities. This questionnaire is a self-report questionnaire in which patients evaluate their difficulties and situations in daily life
Measured: baseline and every four weeks up to eight week
Berg Balance Scale
This scale evaluates the static and dynamic balance by observing the patient's performance for each instruction and giving a score between 0-4. Better scores indicate better balance.
Measured: baseline and every four weeks up to eight week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2022
- Study start22 Feb 2022
- Primary completion23 Apr 2022
- Study completion10 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)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 Istanbul University - CerrahpasaThe 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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Istanbul University - Cerrahpasa
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Aysenur Erekdag, Istanbul University - Cerrahpasa
- Investigators
- Ipek Yeldan Karagoz, PhD, Study director, Istanbul University - Cerrahpasa
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05178706
- Sponsor's protocol ID
- 60260273-615.98-
- Status last verified
- Oct 2023
- First submitted
- 20 Nov 2021
- Last updated
- 12 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Fecek C, Emmady PD. Facioscapulohumeral Muscular Dystrophy. 2023 Jun 26. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2026 Jan-. Available from http://www.ncbi.nlm.nih.gov/books/NBK559028/
PubMed 32644454Rijken NH, van Engelen BG, Weerdesteyn V, Geurts AC. Clinical Functional Capacity Testing in Patients With Facioscapulohumeral Muscular Dystrophy: Construct Validity and Interrater Reliability of Antigravity Tests. Arch Phys Med Rehabil. 2015 Dec;96(12):2201-6. doi: 10.1016/j.apmr.2015.08.429. Epub 2015 Sep 9.
PubMed 26363336Tawil R, Van Der Maarel SM. Facioscapulohumeral muscular dystrophy. Muscle Nerve. 2006 Jul;34(1):1-15. doi: 10.1002/mus.20522.
PubMed 16508966Kang PB, Morrison L, Iannaccone ST, Graham RJ, Bonnemann CG, Rutkowski A, Hornyak J, Wang CH, North K, Oskoui M, Getchius TS, Cox JA, Hagen EE, Gronseth G, Griggs RC; Guideline Development Subcommittee of the American Academy of Neurology and the Practice Issues Review Panel of the American Association of Neuromuscular & Electrodiagnostic Medicine. Evidence-based guideline summary: evaluation, diagnosis, and management of congenital muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology and the Practice Issues Review Panel of the American Association of Neuromuscular & Electrodiagnostic Medicine. Neurology. 2015 Mar 31;84(13):1369-78. doi: 10.1212/WNL.0000000000001416.
PubMed 25825463Tawil R, Kissel JT, Heatwole C, Pandya S, Gronseth G, Benatar M; Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology; Practice Issues Review Panel of the American Association of Neuromuscular & Electrodiagnostic Medicine. Evidence-based guideline summary: Evaluation, diagnosis, and management of facioscapulohumeral muscular dystrophy: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology and the Practice Issues Review Panel of the American Association of Neuromuscular & Electrodiagnostic Medicine. Neurology. 2015 Jul 28;85(4):357-64. doi: 10.1212/WNL.0000000000001783.
PubMed 26215877Kord D, Liu E, Horner NS, Athwal GS, Khan M, Alolabi B. Outcomes of scapulothoracic fusion in facioscapulohumeral muscular dystrophy: A systematic review. Shoulder Elbow. 2020 Apr;12(2):75-90. doi: 10.1177/1758573219866195. Epub 2019 Aug 14.
PubMed 32313557Iosa M, Mazza C, Pecoraro F, Aprile I, Ricci E, Cappozzo A. Control of the upper body movements during level walking in patients with facioscapulohumeral dystrophy. Gait Posture. 2010 Jan;31(1):68-72. doi: 10.1016/j.gaitpost.2009.08.247. Epub 2009 Sep 25.
PubMed 19782569Rijken NH, van Engelen BG, de Rooy JW, Weerdesteyn V, Geurts AC. Gait propulsion in patients with facioscapulohumeral muscular dystrophy and ankle plantarflexor weakness. Gait Posture. 2015 Feb;41(2):476-81. doi: 10.1016/j.gaitpost.2014.11.013. Epub 2014 Dec 2.
PubMed 25687333Eagle M. Report on the muscular dystrophy campaign workshop: exercise in neuromuscular diseases Newcastle, January 2002. Neuromuscul Disord. 2002 Dec;12(10):975-83. doi: 10.1016/s0960-8966(02)00136-0. No abstract available.
PubMed 12467755Statland JM, Tawil R. Risk of functional impairment in Facioscapulohumeral muscular dystrophy. Muscle Nerve. 2014 Apr;49(4):520-7. doi: 10.1002/mus.23949. Epub 2014 Feb 10.
PubMed 23873337
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Does the Upper Extremity Rehabilitation Program Implemented on Individuals With Facioscapulohumeral Dystrophy, Followed by Various Interventions, Have an Impact on Function, Balance, and Gait?
- ClinicalTrials.gov ID
- NCT05178706
- Acronym / sponsor ID
- 60260273-615.98-
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Keywords
- Neuromuscular Disease, Scapulothoracic Arthrodesis, Exercise Therapy, Kinetic Chain
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Istanbul University - CerrahpasaUniversity, hospital or other
- Study start
- 22 Feb 2022 (actual)
- Primary completion
- 23 Apr 2022 (actual)
- Study completion
- 10 Jun 2022 (actual)
- First posted
- 5 Jan 2022 (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.
Facioscapulohumeral Muscular Dystrophy (FSHD) is one of the most common forms of muscular dystrophy, characterized by pronounced skeletal weakness and with a broad spectrum of diseases. It is a hereditary disease seen in 3-5/100,000 of society, usually starting with weakness in the facial and shoulder muscles and progressing to the trunk, pelvis and leg muscles, giving symptoms in the twenties. In FSHD, which shows slow progression and can lead to loss of ambulation ability in about 20% of patients, patients may have difficulty performing activities above shoulder level with the influence of the periscapular area.
The goal of FSHD treatment is to improve muscle strength and/or function. Treatments include medical, conservative and surgical methods. The aim of surgical methods is to improve shoulder function and prevent pain caused by the movements of the scapula. The publications on physiotherapy interventions and aerobic exercise are available as conservative treatment. In patients diagnosed with FSHD, conservative treatment is frequently used to improve muscle strength, regulate function and improve the quality of life of patients.
Patients with FSHD use their affected upper extremities asymmetrically, which leads to the development of restrictive compensation mechanisms in the development of symmetrical postural control. Postural control deficits may occur due to limited use of the affected scapula in individuals with FSHD. Accordingly, in cases with FSHD, there is the use of atypical movements for balance and mobility. It is not yet known whether people with FSHD really have poorer dynamic stability during self-initiated whole-body movements such as walking, and at what stage of the disease these difficulties arise.
Accordingly, the aim of this study was to examine the effects of rehabilitation approaches applied to the upper limb on upper limb function, balance and walking in patients with FSHD.
H1: Within the group of patients with FSHD patients underwent surgery arthrodesis surgery scapulothoracic applied to pre-treatment with the parameters of the rehabilitation program for the evaluation of upper limb functionality after applying the upper extremities, postural control and gait parameters examined, there is statistical difference between the groups.
Detailed description
Voluntary participants who have been diagnosed with FSHD and agreed to voluntary health subjects will be included in the study. Signed voluntary consent will be obtained from participants. Participants will be divided into tree groups. One group will include patients who have undergone scapulothoracic arthrodesis surgery, while the other group will include patients who have not undergone surgery and control group will include participants who have agree to participate the study. A progressive rehabilitation program for the upper extremities will be applied to patients in a group of individuals who have undergone and have not undergone surgery. Both groups will undergo some outcome measures to assess their upper extremity function, balance and walking.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| non-operative conservative treatment | – | Other: upper extremity rehabilitation program, Diagnostic Test: Results of assessment9 individuals diagnosed with FSHD who have not undergone unilateral or bilateral surgery who meet the inclusion criteria. application of determined outcome scales and rehabilitation program on patients |
| scapulothoracic arthrodesis | – | Other: upper extremity rehabilitation program, Diagnostic Test: Results of assessment9 individuals diagnosed with FSHD who have undergone bilateral surgery who meet the inclusion criteria. application of determined outcome scales and rehabilitation program on patients |
| healthy control | – | Diagnostic Test: Results of assessment18 participants for measuring the normative datas; application of determined outcome scales |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
- Study population
- Patients who have been diagnosed with FSHD, patients who have undergone scapulothoracic arthrodesis, healthy people for control group
Key inclusion criteria (5 listed)
- Individuals to be included in all groups 18-50 years
- Score between 0.5 and 3.5 according to CSS (Clinical Severity Score) of the cases to be included in the intervention groups.
- Be rated between 3-5 according to FAS (functional ambulation scale) of the cases to be included in intervention groups
- For patients who will be included in the intervention group who have undergone surgery, bilateral scapulothoracic arthrodesis surgery within 3 months at the earliest
- Individuals who will be included in the control group consisting of healthy individuals have not undergone any orthopedic surgery
Key exclusion criteria (7 listed)
- Having received regular physiotherapy for 1 month, at least 2 days a week in the last 6 months
- Presence of additional neurological problem/problems
- Presence of any other upper limb problem/problems and surgery
- Presence of lower limb problems/problems that can cause balance and walking problems
- Having undergone Spinal fusion surgery
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 |
|---|---|---|
| upper extremity rehabilitation programPatients with FSHD who have undergone and have not undergone surgery will be given activity training by the same physiotherapist after they agree to participate in the study. Both groups will be given the same exercises, the same number of repetitions and the same frequency. A two-phase exercise protocol will be established and progression will be performed after 4 weeks. The exercises will be based on the upper extremity and will include various types of exercises and the primary affected November muscles in the upper extremity. It is planned that the exercises will be accompanied by a physiotherapist 2 days a week, with a total of 16 sessions for 8 weeks. It is planned that there will be a total of 8-10 exercises in 10 repetitions in the exercise program. The total length of the exercise session will be 30 minutes. It is planned that the same physiotherapist will be one-on-one with the patient from the beginning to the end of the exercises. | Other | non-operative conservative treatment, scapulothoracic arthrodesis |
| Results of assessmentThe total follow-up period of participants included in intervention programs will be 8 weeks. Evaluations will be evaluated by physiotherapist before and at the end of treatment. | Diagnostic test | healthy control, non-operative conservative treatment, scapulothoracic arthrodesis |
Primary outcome
change from baseline 10 Meter Walk Test value at 8 weeks
Time frame: baseline and every four weeks up to eight week
during the test, the patient is asked to walk at a normal walking speed at a distance of 10 meters determined before the test. The time it has traveled the specified distance is recorded. It is a long-term performance test used for dynamic balance assessment.
change from baseline Gait Analyzer for walking speed at 8 weeks (m/sec)
Time frame: baseline and every four weeks up to eight week
It is a smartphone-based application for analyzing walking parameters in real time. It can measure walking speed, step time, step length, cadence and symmetry.
change from baseline Gait Analyzer for step time at 8 weeks (sec)
Time frame: baseline and every four weeks up to eight week
It is a smartphone-based application for analyzing walking parameters in real time. It can measure walking speed, step time, step length, cadence and symmetry.
change from baseline Gait Analyzer for step lenght at 8 weeks (m)
Time frame: baseline and every four weeks up to eight week
It is a smartphone-based application for analyzing walking parameters in real time. It can measure walking speed, step time, step length, cadence and symmetry.
change from baseline Gait Analyzer for cadance at 8 weeks (step/min)
Time frame: baseline and every four weeks up to eight week
It is a smartphone-based application for analyzing walking parameters in real time. It can measure walking speed, step time, step length, cadence and symmetry.
change from baseline Gait Analyzer for symmetry at 8 weeks (%)
Time frame: baseline and every four weeks up to eight week
It is a smartphone-based application for analyzing walking parameters in real time. It can measure walking speed, step time, step length, cadence and symmetry.
8 secondary outcomes
change from baseline Timed up and Go Test at 8 weeks
Time frame: baseline and every four weeks up to eight week
change from baseline Single Leg Stance Test at 8 weeks
Time frame: baseline and every four weeks up to eight week
change from baseline 30 Seconds Sit Up Test at 8 weeks
Time frame: baseline and every four weeks up to eight week
change from baseline Hand-Held Dynamometer values at 8 weeks
Time frame: baseline and every four weeks up to eight week
change from baseline Pedometer values at 8 weeks
Time frame: baseline and every four weeks up to eight week
change from baseline Patient Satisfaction Questionnare at 8 weeks
Time frame: every four weeks up to eight week
Disabilities of Arm, Shoulder and Hand
Time frame: baseline and every four weeks up to eight week
Berg Balance Scale
Time frame: baseline and every four weeks up to eight week
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 Oct 2023.
- Background: PubMed 32644454Fecek C, Emmady PD. Facioscapulohumeral Muscular Dystrophy. 2023 Jun 26. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2026 Jan-. Available from http://www.ncbi.nlm.nih.gov/books/NBK559028/
- Background: PubMed 26363336Rijken NH, van Engelen BG, Weerdesteyn V, Geurts AC. Clinical Functional Capacity Testing in Patients With Facioscapulohumeral Muscular Dystrophy: Construct Validity and Interrater Reliability of Antigravity Tests. Arch Phys Med Rehabil. 2015 Dec;96(12):2201-6. doi: 10.1016/j.apmr.2015.08.429. Epub 2015 Sep 9.
- Background: PubMed 16508966Tawil R, Van Der Maarel SM. Facioscapulohumeral muscular dystrophy. Muscle Nerve. 2006 Jul;34(1):1-15. doi: 10.1002/mus.20522.
- Background: PubMed 25825463Kang PB, Morrison L, Iannaccone ST, Graham RJ, Bonnemann CG, Rutkowski A, Hornyak J, Wang CH, North K, Oskoui M, Getchius TS, Cox JA, Hagen EE, Gronseth G, Griggs RC; Guideline Development Subcommittee of the American Academy of Neurology and the Practice Issues Review Panel of the American Association of Neuromuscular & Electrodiagnostic Medicine. Evidence-based guideline summary: evaluation, diagnosis, and management of congenital muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology and the Practice Issues Review Panel of the American Association of Neuromuscular & Electrodiagnostic Medicine. Neurology. 2015 Mar 31;84(13):1369-78. doi: 10.1212/WNL.0000000000001416.
- Background: PubMed 26215877Tawil R, Kissel JT, Heatwole C, Pandya S, Gronseth G, Benatar M; Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology; Practice Issues Review Panel of the American Association of Neuromuscular & Electrodiagnostic Medicine. Evidence-based guideline summary: Evaluation, diagnosis, and management of facioscapulohumeral muscular dystrophy: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology and the Practice Issues Review Panel of the American Association of Neuromuscular & Electrodiagnostic Medicine. Neurology. 2015 Jul 28;85(4):357-64. doi: 10.1212/WNL.0000000000001783.
- Background: PubMed 32313557Kord D, Liu E, Horner NS, Athwal GS, Khan M, Alolabi B. Outcomes of scapulothoracic fusion in facioscapulohumeral muscular dystrophy: A systematic review. Shoulder Elbow. 2020 Apr;12(2):75-90. doi: 10.1177/1758573219866195. Epub 2019 Aug 14.
- Background: PubMed 19782569Iosa M, Mazza C, Pecoraro F, Aprile I, Ricci E, Cappozzo A. Control of the upper body movements during level walking in patients with facioscapulohumeral dystrophy. Gait Posture. 2010 Jan;31(1):68-72. doi: 10.1016/j.gaitpost.2009.08.247. Epub 2009 Sep 25.
- Background: PubMed 25687333Rijken NH, van Engelen BG, de Rooy JW, Weerdesteyn V, Geurts AC. Gait propulsion in patients with facioscapulohumeral muscular dystrophy and ankle plantarflexor weakness. Gait Posture. 2015 Feb;41(2):476-81. doi: 10.1016/j.gaitpost.2014.11.013. Epub 2014 Dec 2.
- Background: PubMed 12467755Eagle M. Report on the muscular dystrophy campaign workshop: exercise in neuromuscular diseases Newcastle, January 2002. Neuromuscul Disord. 2002 Dec;12(10):975-83. doi: 10.1016/s0960-8966(02)00136-0. No abstract available.
- Background: PubMed 23873337Statland JM, Tawil R. Risk of functional impairment in Facioscapulohumeral muscular dystrophy. Muscle Nerve. 2014 Apr;49(4):520-7. doi: 10.1002/mus.23949. Epub 2014 Feb 10.
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Facioscapulohumeral Muscular Dystrophy (FSHD)
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.
Ipek Yeldan Karagoz, PhD study director
Istanbul University - Cerrahpasa
1 site 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 Istanbul University - Cerrahpasa at 1 site in Turkey (Türkiye).
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 5 inclusion and 7 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 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Turkey (Türkiye). 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?
change from baseline 10 Meter Walk Test value at 8 weeks (time frame: baseline and every four weeks up to eight week). The study lists 6 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 NCT05178706, which also lists 10 linked publications.
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 Oct 2023.