Recruiting

Rehabilitation Intervention

Sponsor:

Holland Bloorview Kids Rehabilitation Hospital

Code:

NCT05638750

Conditions

Spinal Muscular Atrophy

Eligibility Criteria

Sex: All

Age: 0 - 6

Healthy Volunteers: Not accepted

Interventions

Rehabilitation Intervention

Study Details

Brief summary:

An outpatient rehabilitation program for children (6 months to less than 6 years old) with Spinal Muscular Atrophy (SMA) treated with genetic based therapies is being studied. Participants will participate in a 12-week therapy program where they receive 45 minutes each of occupational therapy and physical therapy each week. Home exercises will also be prescribed to be completed 5 days per week. At the end of the therapy program, there will be a 12-week period of no therapy where only home exercises will be completed. Assessments and program evaluation will occur at the beginning (Week 0) and end of the rehabilitation program (Week 24), then at the end of the no therapy block (week 24).

Conditions

Spinal Muscular Atrophy

Study ID

NCT05638750

Start date

Jan 1, 2022

Status verified date

Apr, 2024

Completion date

Dec, 2024

Anticipated

Primary completion date

Dec, 2024

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 6

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • SMA (Type I, II or III) diagnosis;
  • Receives genetic based therapy;
  • Aged 6 months to less than 6 years old;
  • Able to participate in weekly therapy at Holland Bloorview (i.e., can attend in-person sessions);
  • Able to bring appropriate respiratory equipment to weekly therapy sessions, if required;
  • Substitute decision makers (SDMs) must be able to speak and read English;
  • Child participant must be able to understand/follow directions in English, as age appropriate;
  • SDMs must consent to participate on behalf of their child.

Exclusion Criteria:

  • Live outside of Ontario;
  • Tracheostomy or use of daytime ventilation (excluding ventilation used during naps).

Study Design

Enrollment

10 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Intervention

Participants will participate in the rehabilitation intervention.

Interventions

Rehabilitation Intervention

12-week treatment intervention \& home exercised, then 12-week non-intervention period, follow-up visit at end of non-intervention period.

Primary outcome measure

  • Infant Toddler Quality of Life Questionnaire (ITQOL) [ Time Frame: Week 0, Week 12, Week 24 ]
  • PedsQL [ Time Frame: Week 0, Week 12, Week 24 ]
  • Canadian Occupational Performance Measure (COPM) [ Time Frame: Week 0, Week 12, Week 24 ]
  • Goal Attainment Scaling (GAS) [ Time Frame: Week 0, Week 12, Week 24 ]
  • Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) [ Time Frame: Week 0, Week 12, Week 24 ]
  • Hammersmith Functional Motor Scale - Expanded (HFMSE) [ Time Frame: Week 0, Week 12, Week 24 ]
  • Bayley Scales of Infant and Toddler DevelopmentTM, 4th Edition [ Time Frame: Week 0, Week 12, Week 24 ]
  • Peabody Developmental Motor Scales (PDMS), 2nd Edition [ Time Frame: Week 0, Week 12, Week 24 ]
  • Acceptability of Intervention Measure (AIM), Intervention Appropriateness Measure (IAM), Feasibility of Intervention Measure (FIM) [ Time Frame: Week 12 ]
  • Home Exercise Diary [ Time Frame: Weeks 1-24 ]

Central Contacts and Locations

Central contacts

Laura McAdam, MD

416-425-6220neuromuscular.research@hollandbloorview.ca

Locations

Holland Bloorview Kids Rehabilitation Hospital

Recruiting

Toronto, Ontario, Canada, M4G 1R8

Contacts

Laura McAdam, MD

416-425-6220lmcadam@hollandbloorview.ca

Principal Investigator:

Laura McAdam, MD

More Information

Sponsor

Holland Bloorview Kids Rehabilitation Hospital

Last update posted

Apr 9, 2024

Last verified

Apr, 2024

Keywords

  • young children
  • pediatric
  • rehabilitation
  • feasibility study
  • pilot study

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Holland Bloorview Kids Rehabilitation Hospital on 2024-04-09.