Spinal Muscular Atrophy and School Transitions

NCT07720518 | Not Yet Recruiting

• 1 other identifier: RL1 935
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

A qualitative questionnaire-based study to explore the experiences of children and young people with Spinal Muscular Atrophy (SMA) and their families regarding school transition and educational participation. The study objectives: To identify barriers to successful educational transition. To advocate for patients and families during the transitional process. To explore experiences of inclusion, access and support within school settings. To understand the coordination between education, healthcare and support services. To inform the development of practical, patient-centred guidance to improve educational transitions for children and young people with SMA.

Conditions

Spinal Muscular Atrophy

Keywords

SMA type 11 and 111

Outcome Measures

Primary Outcomes (1)

• Completion of the questionnaire to explore the experiences of children and young people with SMA and their families regarding school transition and educational participation.

  3 families will be invited to take place in a focus group discussion. Each family will be interviewed in a family unit on Microsoft Teams. These discussions will enable the development of a questionnaire which will be sent to 12 families to complete.

  1 month to complete the focus groups. 1 month to develop the questionnaire. 1 month to send questionnaires to 12 families and allow for the responses back.

Study Arms (2)

Focus group

Discussion regarding the development of a questionnaire

Completing questionnaire

Eligibility Criteria

• Age: Up to 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Participants with a genetic diagnosis of Spinal Muscular Atrophy 1, 11 or 111.

You may qualify if:

• Genetically confirmed diagnosis of Spinal Muscular Atrophy 1, 11 or 111
• In education, in either primary school, secondary school or further education
• Access to internet and either a smart phone, tablet or computer.

You may not qualify if:

• Not in education
• Not able to comply with the consent process
• Not cognitively able to complete the questionnaire.

Sponsors & Collaborators

• Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust: lead

Study Sites (1)

The Robert Jones & Agnes Hunt Orthopaedic Hospital

Oswestry, Shropshire, SY10 7AG, United Kingdom

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Officials

• Nicholas Emery

  Neuromuscular Clinical Specialist Physiotherapist

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Julie Steen

CONTACT

01691 404418; julie.steen@nhs.net

Study Design

• Study Type: observational
• Observational Model: FAMILY BASED
• Time Perspective: PROSPECTIVE
• Target Duration: 1 Month
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 17, 2026
• First Posted: July 22, 2026
• Study Start (Estimated): August 10, 2026
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): February 28, 2027
• Last Updated: July 22, 2026

Record last verified: 2026-07

Locations

GB (1)