Duchenne Muscular Dystrophy (DMD)SEE-NMDNCT07356063
Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy
A study testing Multicomponent Intervention in people with Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA), run by Aga Khan University at 1 site in Pakistan.
Trial 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… Read more about this study
- Who is it for?
- Ages Up to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA)
- What is being tested?
- Multicomponent Intervention
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 1 sitePakistan
- Study dates
- 15 Aug 2026to 4 Dec 2026
- Phase
- Interventional
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 Up to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- Caregivers who are currently participating in another support group intervention.
- Caregivers who are unable to understand the language of communication (Urdu)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pre-Post Single-Arm Pilot Trial of a Digital Multicomponent Intervention for Self-Efficacy Enhancement Among Online Support Groups of Primary Caregivers of Children With Neuro-Muscular Disorders: SEE-NMD Protocol
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)
Read the detailed description
Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD) are progressive genetic neuromuscular disorders that impose substantial caregiving demands on families. In Pakistan, where disease-modifying therapies remain largely inaccessible, care is predominantly supportive, frequently resulting in psychological distress and diminished caregiver confidence. An individual's belief in their capacity to execute required behaviours, self-efficacy, is a recognised determinant of caregiving quality, yet evidence on caregiver self-efficacy in SMA and DMD within low-resource settings remains sparse. This protocol describes the methods for an exploratory, single-arm, pre-post trial. The trial aims to generate preliminary evidence on changes in self-efficacy following on the effectiveness of a multicomponent, digitally delivered intervention and its implementation feasibility among primary caregivers of children with SMA and DMD in Pakistan.
- Being tested
- Multicomponent Intervention
- Keywords
- DMD, SMA, Self-Efficacy, Caregivers, Treat-NMD, Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Atrophy, Spinal, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Multicomponent InterventionBehavioural
Multiple support groups will be created for SMA and DMD patients, separately. Within SMA and DMD, further subgroups will be created on the basis of caregivers educational level and socioeconomic background.
Each support group will include up to 10-14 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 sessions with each group will be 30-60 minutes in duration. In between these live sessions, short video clips, pictorial guides, audios will be sent to the participants to re-enforce what they could learn to improve care of their child and potentially increase their self-efficacy levels.
| Study group | Role | What they receive |
|---|---|---|
| Support Group for Caregivers | Experimental | A support group comprising of the primary caregivers of children with DMD or SMA enrolled in the Treat-NMD Registry of Pakistan, will be given the Intervention. Their self-efficacy will be assessed before and after the intervention to measure any changes associated with participation. Receives: Behavioral: Multicomponent Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Self-Efficacy Scores
Change from Baseline in the Self-Efficacy Scores at 8 weeks. Care giver self efficacy tool is a 20-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).
Measured: Baseline and 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2026
- Study start15 Aug 2026
- Primary completion20 Nov 2026 (estimated)
- Study completion4 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A single-arm, pre-post study design will recruit primary caregivers of children with SMA and DMD enrolled in the Treat-NMD Registry of Pakistan, with 40 caregivers per condition (80 total) analyzed as separate disease-specific cohorts. An eight-week intervention will be delivered via online support groups, comprising educational video clips, expert-led live sessions, pictorial guides, and progressively tapering audio reminders. The primary outcome, self-efficacy, will be measured using a culturally adapted, content-validated Urdu version of the DMD Caregiver Self-Efficacy Scale (DMD-CSES), assessed at baseline (T0) and eight weeks post-intervention (T1). 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 phone call. Post intervention: After 2 months, the participants of each group will be contacted for post intervention scoring via phone call.
- Masking
- None (open label)
- Enrolment
- 34 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Aga Khan University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sara Khan, Aga Khan University
- Investigators
- Nashit Irfan Aziz, MBBS, MSc, Principal investigator, Aga Khan UniversitySara Khan, MBBS, MD, Principal investigator, Aga Khan UniversityWardah Khalid, MBBS, MSc, PhD, Principal investigator, Aga Khan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07356063
- Sponsor's protocol ID
- 2025-11875-37040
- Other IDs
- 2025
- Acronym
- SEE-NMD
- Status last verified
- Jan 2026
- First submitted
- 11 Jan 2026
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Because DMD and SMA are rare diseases, and number counts might be traceable with collected information.