Outpatient Rehabilitation Intervention for Young Children With SMA

NCT ID: NCT05638750

Last Updated: 2024-04-09

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-01-01
• Study Completion Date: 2024-12-31

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).

Detailed Description

The standard of care for SMA has historically been physical therapy (PT) and occupational therapy (OT) often focused on strategies that reduce the risk of secondary side effects such as joint tightness. Recently, three genetic based therapies: nusinersen, onasemnogene abeparvovec and risdiplam, have been approved as treatment by Health Canada. Genetic based therapies have provided improvements in physical function for children with SMA. Currently, there is no evidence-based guidance regarding rehabilitation to increase function for children with SMA that have received genetic based therapies. Additionally, there is a lack of published evidence regarding the type of rehabilitation programs and the impact that rehabilitation has on physical function.

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 proposed and its feasibility being evaluated. 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 Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Intervention

Participants will participate in the rehabilitation intervention.

Group Type: EXPERIMENTAL

Rehabilitation Intervention

Intervention Type: OTHER

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

Interventions

Rehabilitation Intervention

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

Intervention Type: OTHER

Eligibility Criteria

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).

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Holland Bloorview Kids Rehabilitation Hospital

OTHER

Sponsor Role: lead

Responsible Party

Laura McAdam

Medical Director, Child Development Program

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, Canada

Site Status: RECRUITING

Countries

Canada

Central Contacts

Laura McAdam, MD

Role: CONTACT

neuromuscular.research@hollandbloorview.ca

416-425-6220 ext. 6605

Facility Contacts

Laura McAdam, MD

Role: primary

lmcadam@hollandbloorview.ca

416-425-6220 ext. 6605

References

Ippolito C, Canthiya L, Floreani A, Luckhart K, Hoffman A, McAdam L. Twice-Weekly Outpatient Rehabilitation Intervention for Young Children With Spinal Muscular Atrophy Treated With Genetic-Based Therapies: Protocol for a Feasibility Study. JMIR Res Protoc. 2023 Nov 2;12:e46363. doi: 10.2196/46363.

Reference Type: DERIVED

PMID: 37917140 (View on PubMed)

Other Identifiers

0550

Identifier Type: -

Identifier Source: org_study_id