An observational study in Duchenne Muscular Dystrophy (DMD), Chronic Pain and Neuromuscular Diseases (NMD), sponsored by Antalya Training and Research Hospital. Recruiting at 1 site in Turkey (Türkiye). Open to male participants aged 7 Years to 18 Years. Per ClinicalTrials.gov, last updated 2026-10-23.
Sponsored by Antalya Training and Research Hospital · Observational
1. Brief Summary This prospective observational study aims to evaluate the 6-month longitudinal changes in chronic pain, pain characteristics, coping strategies, and functional performance in children and adolescents aged 7 to 18 years diagnosed with Duchenne Muscular Dystrophy (DMD), with a primary focus on comparing ambulatory and non-ambulatory disease stages.
Pediatric DMD patients followed at the University of Health Sciences Antalya Training and Research Hospital Neuromuscular Diseases Center will undergo comprehensive evaluations at baseline (Month 0) and at 6 months follow-up (Month 6). Pain parameters will be evaluated using the Adolescent Pediatric Pain Tool (APPT) to assess pain intensity, body localization, and sensory/affective/evaluative/temporal qualities, and the Brief Pain Inventory (BPI) 7-item Pain Interference Scale to determine pain interference with daily activities. Pain coping strategies will be assessed using the Pediatric Pain Coping Inventory (PPCI, adolescent and parent versions). Functional and motor assessments will include the North Star Ambulatory Assessment (NSAA), 6-Minute Walk Test (6MWT), stair climb/descend tests, Vignos and Brooke scales, handgrip dynamometry, manual muscle testing (MMT), and range of motion (ROM) and contracture evaluations, alongside anthropometrics and quality of life.
The central hypotheses are that non-ambulatory patients experience chronic pain more frequently, with longer duration, greater intensity, and across a higher number of anatomical sites than ambulatory patients. Furthermore, the study hypothesizes that anatomical pain sites, pain-aggravating and pain-relieving factors, and pain coping mechanisms differ significantly between ambulatory and non-ambulatory stages.
Duchenne Muscular Dystrophy (DMD) is an X-linked, progressive degenerative neuromuscular disorder. While motor decline is well documented, chronic pain and pain-related disability remain under-recognized secondary burdens. This single-center, prospective longitudinal observational study investigates the progression of chronic pain, pain interference, and coping mechanisms over a 6-month follow-up period in pediatric DMD patients and compares clinical phenotypes between ambulatory and non-ambulatory cohorts.
Study Hypotheses:
Chronic pain prevalence is significantly higher in non-ambulatory patients than in ambulatory patients.
The anatomical distribution of pain differs between non-ambulatory and ambulatory stages.
The total number of painful body regions is greater in non-ambulatory patients compared to ambulatory patients.
Non-ambulatory patients experience a significantly longer duration of chronic pain than ambulatory patients.
Chronic pain intensity is significantly higher in non-ambulatory patients than in ambulatory patients.
Pain-aggravating factors differ between ambulatory and non-ambulatory stages.
Pain-relieving (alleviating) factors differ between ambulatory and non-ambulatory stages.
Study Protocol and Setting:
Eligible pediatric patients aged 7-18 years with genetically confirmed DMD residing in Antalya will be recruited consecutively during scheduled outpatient visits at the University of Health Sciences Antalya Training and Research Hospital Neuromuscular Diseases Center. Restricting enrollment to Antalya residents aims to prevent follow-up attrition and minimize patient transport burden. Because DMD is a rare orphan disease, convenience sampling is employed without a formal a priori power calculation. Evaluations occur at baseline (Month 0) and 6 months follow-up (Month 6).
Assessment Instruments and Outcome Measures:
Adolescent Pediatric Pain Tool (APPT): Assesses anatomical pain localization using a pediatric body diagram, pain intensity using a 100 mm Visual Analog Scale (VAS) (0-4 mm: no pain; 5-44 mm: mild pain; 45-74 mm: moderate pain; 75-100 mm: severe pain), and pain quality descriptors across 4 dimensions (sensory, affective, evaluative, and temporal; 15 words total) scored as percentages per dimension.
Brief Pain Inventory (BPI) 7-Item Pain Interference Scale: Assesses the degree to which pain interferes with 7 daily activities over the last 24 hours (general activity, mood, walking ability, normal work, relations with others, sleep, and enjoyment of life) on a 0 (does not interfere) to 10 (completely interferes) numerical rating scale, calculated as a total interference score.
Pediatric Pain Coping Inventory (PPCI): Evaluates behavioral and cognitive pain coping strategies using the validated adolescent and parent proxy versions across subscales (cognitive self-instruction, problem solving, distraction, seeking social support, and catastrophizing/helplessness) rated on a 3-point Likert scale (0 = never, 1 = sometimes, 2 = often).
Motor and Functional Assessments: North Star Ambulatory Assessment (NSAA), 6-Minute Walk Test (6MWT), timed stair climb and descend tests, Brooke Upper Extremity Scale, Vignos Lower Extremity Scale, handgrip dynamometry, and Manual Muscle Testing (MMT).
Musculoskeletal Examination: Goniometric joint range of motion (ROM) and clinical joint contracture evaluations.
Quality of Life \& Anthropometrics: Standardized pediatric health-related quality of life questionnaires, body weight, height, and Body Mass Index (BMI).
Design Note:
Being a non-interventional observational study, no therapeutic allocation, randomization, or placebo is utilized; all participants receive standard medical care throughout the 6-month observation period.
480 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 109 are open to participants now.
This study's planned enrollment of 30 is below the median of 60 across 134 observational studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →Antalya Training and Research Hospital is the lead sponsor of 104 studies on the registry; 12 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Pediatric patients aged 7 to 18 years diagnosed with Duchenne Muscular Dystrophy who are regularly monitored and consecutively recruited during routine outpatient follow-up visits at the Neuromuscular Diseases Center of University of Health Sciences Antalya Training and Research Hospital, residing within the province of Antalya.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Pediatric patients aged 7-18 years with genetically confirmed Duchenne Muscular Dystrophy who maintain independent ambulatory capacity.
Other: Routine Clinical Follow-up and Pain Assessment
Pediatric patients aged 7-18 years with genetically confirmed Duchenne Muscular Dystrophy who have lost independent ambulation and are wheelchair-dependent.
Other: Routine Clinical Follow-up and Pain Assessment
Observation of standard clinical care, natural disease progression, pain characteristics, functional status, and coping strategies over a 6-month period without experimental intervention.
Change in Chronic Pain Intensity From Baseline to 6 Months
Assessed using the Visual Analog Scale (VAS) component of the Adolescent Pediatric Pain Tool (APPT). Pain intensity is marked along a 100 mm horizontal line, where 0 mm indicates 'no pain' and 100 mm indicates 'worst possible pain'. Pain levels are categorized as: 0-4 mm (no pain), 5-44 mm (mild pain), 45-74 mm (moderate pain), and 75-100 mm (severe pain). Higher scores reflect greater pain severity.
Time frame: Baseline (Month 0) and Month 6
Change in Pain Interference on Daily Activities From Baseline to 6 Months
ssessed using the 7-item Pain Interference Scale of the Brief Pain Inventory (BPI). It evaluates how much pain interferes with 7 daily life domains (general activity, mood, walking ability, normal work, relations with others, sleep, and enjoyment of life) over the past 24 hours. Each item is rated on an 11-point numeric scale from 0 (does not interfere) to 10 (completely interferes). The total score ranges from 0 to 70, with higher scores indicating greater pain interference in daily functioning.
Time frame: Baseline (Month 0) and Month 6
Change in the Number of Anatomical Pain Sites From Baseline to 6 Months
Assessed using the pediatric body diagram of the Adolescent Pediatric Pain Tool (APPT). Participants mark all anatomical regions where they experience pain. The total number of painful body areas is quantified, with higher numbers indicating more widespread pain.
Time frame: Baseline (Month 0) and Month 6
Change in Pain Quality Descriptors From Baseline to 6 Months
Assessed using the word descriptor component of the Adolescent Pediatric Pain Tool (APPT), which evaluates 4 dimensions of pain quality: sensory, affective, evaluative, and temporal across a total of 15 words. Scores are calculated as percentages of selected words relative to the total number of words within each dimension (0% to 100%). Higher percentage scores indicate greater endorsement of that specific pain dimension.
Time frame: Baseline (Month 0) and Month 6
Change in Pediatric Pain Coping Strategies From Baseline to 6 Months
Assessed using the Pediatric Pain Coping Inventory (PPCI, adolescent and parent-proxy versions). It measures coping strategies across 5 subscales: cognitive self-instruction, problem solving, distraction, seeking social support, and catastrophizing/helplessness. Items are scored on a 3-point Likert scale (0 = never, 1 = sometimes, 2 = often). Higher scores in each subscale indicate more frequent utilization of that specific coping strategy.
Time frame: Baseline (Month 0) and Month 6
Change in Ambulatory Motor Performance From Baseline to 6 Months
Assessed using the North Star Ambulatory Assessment (NSAA) in ambulatory participants. It evaluates 17 motor activities (such as standing, walking, rising from a chair, jumping, and running). Each item is scored on a 3-point scale: 0 (unable to achieve independently), 1 (achieves with modifications), and 2 (normal performance). Total raw scores range from 0 to 34, with higher scores reflecting better motor and functional capability.
Time frame: Baseline (Month 0) and Month 6
Change in Functional Exercise Capacity From Baseline to 6 Months
Evaluated using the modified 6-Minute Walk Test (6MWT) according to standard DMD protocols in ambulatory participants capable of walking at least 75 meters. Total distance walked in 6 minutes is recorded in meters. A greater distance indicates better functional exercise tolerance.
Time frame: Baseline (Month 0) and Month 6
Change in Lower and Upper Extremity Functional Disability Stages From Baseline to 6 Months
Lower extremity function is evaluated using the Vignos Scale (grades 1 to 10, ranging from 1 = walks and climbs stairs without assistance to 10 = bedridden). Upper extremity function is evaluated using the Brooke Scale (grades 1 to 6, ranging from 1 = arms abducted in a full circle to touch above head to 6 = no useful hand function). For both scales, higher numerical scores indicate more severe functional disability.
Time frame: Baseline (Month 0) and Month 6
Change in Muscle Strength From Baseline to 6 Months
Handgrip strength is measured bilaterally using an electronic hand dynamometer in kilograms (kg), recording the average and maximum of 3 trials. Proximal and distal muscle strength is assessed bilaterally using Manual Muscle Testing (MMT) with the Medical Research Council (MRC) scale (0 = no movement to 5 = normal strength). Total muscle strength is calculated as a composite percentage score (%MRC) using the standard formula: (Total score × 100) / (Number of tested muscles × 5). Higher values indicate greater muscle strength.
Time frame: Baseline (Month 0) and Month 6
Change in Timed Stair Climbing and Descending Performance From Baseline to 6 Months
Timed performance to ascend and descend a standard 4-step flight of stairs is measured in seconds using a digital stopwatch. Shorter completion times indicate superior functional mobility and lower extremity motor velocity.
Time frame: Baseline (Month 0) and Month 6
Change in Joint Range of Motion and Presence of Contractures From Baseline to 6 Months
Passive joint range of motion (ROM) is measured in degrees using a standard goniometer across cervical, shoulder, elbow, wrist, metacarpophalangeal, hip, knee, and ankle joints. Contracture is defined as passive loss of full normal joint ROM compared to age-matched reference standards. Lower degrees of passive ROM indicate greater joint contracture severity.
Time frame: Baseline (Month 0) and Month 6
Plan to share: No
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Started Jan 2026 (actual). Primary completion is estimated for Nov 2026, 10 months after the start.
Of 53 non-industry observational studies under Muscular Dystrophy, Duchenne, half reached primary completion within 37 months of starting (the median), counting those still running; a quarter within 11. This study's planned 10 months is below that.
Counting every sponsor, half of 64 studies reached it within 40 months; a quarter within 13 and three quarters within 120.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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