Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?

NCT07892196 | Recruiting

• 1 other identifier: 25.12.2025 22/37
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Duchenne Muscular Dystrophy (DMD); Chronic Pain; Neuromuscular Diseases (NMD)

Keywords

duchenne muscular dystrophy; Quality of Life; Ambulation; Pain

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline (Month 0) and Month 6

Secondary Outcomes (10)

• Change in Pain Interference on Daily Activities From Baseline to 6 Months

  Baseline (Month 0) and Month 6

• Change in the Number of Anatomical Pain Sites From Baseline to 6 Months

  Baseline (Month 0) and Month 6

• Change in Pain Quality Descriptors From Baseline to 6 Months

  Baseline (Month 0) and Month 6

• Change in Pediatric Pain Coping Strategies From Baseline to 6 Months

  Baseline (Month 0) and Month 6

• Change in Ambulatory Motor Performance From Baseline to 6 Months

  Baseline (Month 0) and Month 6

Study Arms (2)

Ambulatory DMD Patients

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

Non-ambulatory DMD Patients

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

Interventions

Routine Clinical Follow-up and Pain Assessment OTHER

Observation of standard clinical care, natural disease progression, pain characteristics, functional status, and coping strategies over a 6-month period without experimental intervention.

Ambulatory DMD Patients; Non-ambulatory DMD Patients

Eligibility Criteria

• Age: 7 Years - 18 Years
• Sex: male
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• Genetically confirmed diagnosis of Duchenne Muscular Dystrophy (DMD)
• Aged 7 to 18 years
• Residing in Antalya, Turkey (geographical restriction applied to minimize patient travel burden and prevent loss to follow-up over the 6-month observation period)
• Intact hearing and vision capability sufficient to complete test instructions and self-report forms
• Voluntary willingness to participate, with signed informed consent from parents/legal guardians and informed assent from the child

You may not qualify if:

• Bedridden status (inability to sit or maintain basic seated posture)
• History of tracheostomy
• Severe intellectual disability that precludes understanding instructions
• Severe speech or communication impairment preventing self-report assessments
• Presence of co-existing psychiatric or neurodevelopmental conditions (e.g., autism spectrum disorder, attention deficit hyperactivity disorder \[ADHD\])

Sponsors & Collaborators

• Antalya Training and Research Hospital: lead

Study Sites (1)

University of Health Sciences Antalya Training and Research Hospital, Neuromuscular Diseases Center

Antalya, Antalya, 07100, Turkey (Türkiye)

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Chronic Pain; Neuromuscular Diseases; Pain

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• beyza deveci, MD

  +90 242 249 44 00

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2026
• First Posted: October 23, 2026
• Study Start: January 2, 2026
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): December 30, 2026
• Last Updated: October 23, 2026

Record last verified: 2026-10

Data Sharing

• IPD Sharing: Will not share

Locations

TU (1)