Self-Efficacy Enhancement Using a Multicomponent Support Group for Caregivers of Children With DMD/SMA

NCT07356063 | Not Yet Recruiting DMC

• 1 other identifier: 2025-11875-37040
• Study Type: interventional
• Target: 30
• Locations: 0 countries
• Sites: N/A

Brief Summary

The goal of this clinical trial is to learn if providing a multicomponent intervention improves the confidence of caregivers of children with DMD or SMA. The main question it aims to answer is: Does this intervention increase their self-efficacy scores over 8 weeks time? Researchers will compare scores at baseline (pre intervention) and after 8 weeks (post intervention)

Conditions

Duchenne Muscular Dystrophy (DMD); Spinal Muscular Atrophy (SMA)

Keywords

DMD; SMA; Self-Efficacy; Caregivers; Treat-NMD; Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• Change in Self-Efficacy Scores

  Change from Baseline in the Self-Efficacy Scores at 8 weeks. Care giver self efficacy tool is a 19-item self-report questionnaire adapted from the original validated scale developed for caregivers of individuals with DMD (DMD-CSES). The scale assesses caregiver self-efficacy across multiple domains of caregiving. Each item is rated on a 5-point Likert scale ranging from 1 (Strongly disagree) to 5 (Strongly agree). Total scores range from 19 to 95, calculated by summing responses across all items. Higher scores indicate greater caregiver self-efficacy (better outcome).

  Baseline and 8 weeks

Study Arms (1)

Multicomponent Support Group Intervention for Caregivers of Children with DMD or SMA

EXPERIMENTAL

Participants receive a structured, online multicomponent support group designed for primary caregivers of children with DMD or SMA enrolled in the Treat-NMD Registry of Pakistan. The intervention includes educational sessions, peer support discussions, and guidance from professionals in physiotherapy, respiratory care, occupational therapy, and psychosocial coping. Caregiver self-efficacy will be assessed before and after the intervention to measure changes associated with participation.

Behavioral: Multicomponent Support Group

Interventions

Multicomponent Support Group BEHAVIORAL

A multifaceted strategy will be employed. Multiple groups will be created for SMA and DMD patients. Each group will include up to 10 patients caregivers, 1 neurologist, 1 pediatric neurologist, 1 physiotherapist, 1 nutritionist, 1 speech therapist, 1 occupational therapist, 1 epidemiologist and 1 Research Associate. Sessions with each group will integrate peer support, coaching to address the multifaceted needs of patients, and time for open questions and answer sessions with the group. The session with each group will be 1 hours duration. It will start with 5 minutes talk from each expert about the basic needs for SMA and DMD patients. Following the first session, after a few weeks gap, another similar 1 hour session will be conducted which will be open to care givers to ask questions off the specialists or other care givers. In between these 2 sessions, short videos or pictorial clips for care will be sent to the participants to re-enforce what they learnt from sessions.

Multicomponent Support Group Intervention for Caregivers of Children with DMD or SMA

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Primary caregiver of a child diagnosed with SMA or DMD.
• Enrolled in the Treat-NMD registry- Pakistan.
• Access to a smart phone with internet connectivity.

You may not qualify if:

• Caregivers who are currently participating in another support group intervention.
• Caregivers who are unable to understand the language of communication (Urdu)

Sponsors & Collaborators

• Aga Khan University: lead

Related Links

• Treat-NMD is a global organisation that brings together a network of leading specialists, patient groups and industry representatives. This in turn aims to ensure preparedness for the trials and therapies of the future while also promoting best practice
• The Muscular Dystrophy Registry of Pakistan has been setup with a goal to improve outcomes and quality of life in patients with muscular dystrophies and related neuromuscular disorders. The registry is currently recruiting patients with Duchenne/Becker M

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Atrophy, Spinal; Muscular Dystrophies

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Spinal Cord Diseases; Central Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases

Study Officials

• Nashit Irfan Aziz, MBBS, MSc

  Aga Khan University

  PRINCIPAL INVESTIGATOR

• Sara Khan, MBBS, MD

  Aga Khan University

  PRINCIPAL INVESTIGATOR

• Wardah Khalid, MBBS, MSc, PhD

  Aga Khan University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Nashit Irfan Aziz, MBBS, MSc

CONTACT

+923142002888; nashit.irfan24@gmail.com

Sara Khan, MBBS, MD

CONTACT

sara.khan@aku.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Doctor

Model Details: Pre intervention: Baseline assessment of self efficacy scoring will be taken from each participant, where they will rate each item on a 5-point Likert scale, on Zoom call. Post intervention: After 2 months, the participants of each group will be contacted for post intervention scoring via Zoom call.

Study Record Dates

• First Submitted: January 11, 2026
• First Posted: January 21, 2026
• Study Start: February 23, 2026
• Primary Completion: March 23, 2026
• Study Completion: May 23, 2026
• Last Updated: January 22, 2026

Record last verified: 2026-01