HABIT-ILE + FST in Children With SMA

March 13, 2026 updated by: Teachers College, Columbia University

Feasibility and Preliminary Effectiveness of Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) + Functional Strength Training (FST) in Children With Spinal Muscular Atrophy (SMA): a Pilot Study

This single-arm pilot study will assess the feasibility and preliminary effectiveness of an intensive motor skill intervention (HABIT-ILE) combined with functional strength training (FST) in children with SMA who are receiving disease-modifying therapies. Participants will attend one 6-hour HABIT-ILE + FST session each weekend (Saturday or Sunday) for 15 weeks, for a total of 90 hours of training. Feasibility will be evaluated through questionnaires, and effectiveness through standardized motor function assessments.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

Spinal Muscular Atrophy (SMA) is a severe neuromuscular disorder marked by the progressive degeneration of alpha motor neurons in the spinal cord and brainstem, resulting in proximal muscle atrophy and weakness. Based on age of onset and the motor function achieved, children were historically categorized into functional groups. However, recent advances leading to the development of disease-modifying therapies (DMTs) have transformed the management of SMA. Outcomes are now primarily determined by the timing of DMT initiation, with early treatment-ideally before symptom onset-showing significant efficacy in improving motor function and survival. Despite these therapeutic breakthroughs, rehabilitation remains a cornerstone of care for children with SMA. Current clinical guidelines emphasize physical activity, muscle strengthening, and stretching. However, few controlled studies have rigorously evaluated these interventions, and even fewer have examined their combined effects with DMTs. This gap underscores the need for innovative, evidence-based rehabilitation strategies that can complement pharmacological treatments and further promote functional outcomes. Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) is an intervention that integrates bimanual coordination with postural control and gross motor training. Grounded in motor skill learning principles, HABIT-ILE delivers high-intensity, structured practice of progressively increased motor difficulty tasks and functional activities, emphasizing voluntary active movement. The approach has demonstrated efficacy in children aged 6 months to 18 years with cerebral palsy (CP), a non-progressive neurodevelopmental disorder caused by early brain injury. Although SMA and CP differ in pathophysiology, both conditions involve motor impairments that may respond to intensive, task-specific motor training. In individuals with SMA, central neuroplastic mechanisms may help compensate for peripheral motor deficits, while targeted motor training could optimize recruitment and efficiency of residual motor units in the muscle. Strength training, in particular, has demonstrated beneficial effects in individuals with SMA, suggesting that emphasizing skill training requiring increasing endurance and progressively increasing the weights of objects participants handled, may be especially advantageous. In this context, augmenting HABIT-ILE with a functional strength training (FST) component tailored to individual goals may further enhance motor outcomes in this population. The aim of this pilot proposal is to evaluate the feasibility and preliminary efficacy of HABIT-ILE + FST in children with SMA receiving DMTs. Children with SMA will attend one 6-hour HABIT-ILE + FST session each weekend (either Saturday or Sunday) for 15 weeks, adding up to a total of 90 hours. It is hypothesized that this intervention will be well tolerated by children, enhance the acquisition of new motor skills, and foster greater functional independence in daily activities.

Study Type

Interventional

Enrollment (Estimated)

5

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • New York, New York, United States, 10027
        • Center for Cerebral Palsy Research, Teachers College, Columbia University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Diagnosis of SMA
  • Able to participate in all pre and post intervention assessments
  • Able to understand and speak English
  • Able to understand directions (no significant cognitive delay or serious behavioral issues that would limit participation)
  • Receiving a stable dose of disease-modifying therapies for at least 6 months prior to enrollment.

Exclusion Criteria:

  • Orthopedic surgery within the last year (confound)
  • Initiation of any new pharmaceutical treatment during the study period
  • Participation in a clinical trial of an investigational therapy

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Combined HABIT-ILE + FST
Participants receive a combined intervention consisting of Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) and Functional Strength Training (FST). Both components are delivered as an integrated, intensive motor learning-based program aimed at improving upper and lower limb function in children with SMA .Subject will participate in a HABIT-ILE + FST camp format for 6 hours/day, one day/week for 15 weeks.
Other: Combined Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) + functional strength training (FST)
Goal-directed, task-specific training for both the upper and lower extremity, and postural control with targeted strengthening exercises to enhance motor control and facilitate the achievement of functional goals

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Feasibility questionnaire
Time Frame: Immediately after the intervention
Feasibility questionnaire completed by both the child and their parents to assess the acceptability of the intervention (including child satisfaction and physical comfort, and parent feedback), as well as its demand and practicality.
Immediately after the intervention

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Hammersmith Functional Motor Scale -Expanded (HFMSE)
Time Frame: 4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Gross motor function measure; range: 0 to 66; higher scores indicate better motor function
4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Revised Upper Limb Module (RULM)
Time Frame: 4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Upper limb function measure; range: 0 to 37; higher scores indicate better uper limb function
4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Endurance Shuttle box and blocks test (ESBBT)
Time Frame: 4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Dexterity and upper extremity endurance/fatigability; range: O to 20 minutes (time to limitation); higher times correspond to better endurance
4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
6-Minute Walk Test (6MWT)
Time Frame: 4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Walking endurance measure; range: 0 meters to the maximim distance the participant can walk in 6 minutes; higher distances reflect better ambulatory function and endurance.
4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
10 Meter Walking Test (10MWT)
Time Frame: 4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Assesses gait speed by recording the time required to walk 10 meters, which is then expressed in meters per second. Scores range from 0 m/s (unable to walk) up to approximately 2 m/s in healthy individuals. Higher walking speed reflects better ambulatory function.
4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Timed Up and Go (TUG)
Time Frame: 4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Functional mobility and dynamic balance measure. The TUG measures the time required to rise from a chair, walk 3 meters, turn around, return, and sit down. The outcome is expressed in seconds, with lower times reflecting better functional mobility and balance
4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
30-Second Sit-to-Stand Test (30STS)
Time Frame: 4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Lower limb strength and endurance measure; range: 0 (unable to stand) to higher values reflecting better lower limb strength and endurance
4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Trunk Control Measurement Scale (TCMS)
Time Frame: 4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Static and dynamic trunk control measure; range: 0 to 58 points; higher scores indicating better trunk control
4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Spinal Muscular Atrophy EFFORT (SMA EFFORT)
Time Frame: 4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Questionnaire of perceived physical fatigability (ages 12 years and older); different subscales; higher scores indicating greater perceived fatigue
4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Canadian Occupational Performance Measure (COPM)
Time Frame: 4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Interview of occupational performance and satisfaction; range: 1 to 10 for each item, with higher scores indicating better performance or greater satisfaction.
4 assessments: Baseline 1, baseline 2 (1 week), post intervention (16 weeks), 3 months follow-up
Daily activity logs
Time Frame: Every day during the intervention (assessed up to 15 weeks)
Implementation feasibility measure
Every day during the intervention (assessed up to 15 weeks)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Teachers College, Columbia University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 1, 2025

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

July 1, 2027

Study Registration Dates

First Submitted

September 25, 2025

First Submitted That Met QC Criteria

October 30, 2025

First Posted (Actual)

October 31, 2025

Study Record Updates

Last Update Posted (Actual)

March 16, 2026

Last Update Submitted That Met QC Criteria

March 13, 2026

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 25-429

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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