HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness

Feasibility and Preliminary Effectiveness of HABIT-ILE + Functional Strength Training in Children With Spinal Muscular Atrophy (SMA): a Prospective Single-group Intervention Trial.

This single-arm pilot study will assess the 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 a HABIT-ILE + FST summer camp for 6 hours per day over a 3-week period, totaling 90 hours of training.

Study Overview

• Status: Recruiting
• Conditions: Spinal Muscular Atrophy (SMA)
• Intervention / Treatment: Other: Combined Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) + functional strength training (FST)

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 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 aims of this prospective single-group intervention trial are to determine the effects of HABIT-ILE+FST on motor function in children with SMA receiving DMTs, and to assess retention of gains at 6 months, and to evaluate the effects of HABIT-ILE+FST on muscle fatigability using both clinical assessments and surface electromyography (sEMG). Participants will complete 90 hours of HABIT-ILE + FST training over a 3-week period (6 hours/day). 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): 20
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Andrew M Gordon, PhD; Phone Number: +1(212) 678-3332; Email: ag275@tc.columbia.edu
• Study Contact Backup: Name: Astrid Carton de Tournai, PhD; Email: apc2187@tc.columbia.edu

Study Locations

• United States
  • New York
    • New York, New York, United States, 10021
      • Recruiting
      • Center for Cerebral Palsy Research, Teachers College, Columbia University
      • Contact: Astrid Carton de Tournai, PhD; Phone Number: (212) 678-3332; Email: cpresearch@tc.columbia.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Have a confirmed SMA diagnosis
• Be 5-17 years old
• Complete all pre- and post-intervention assessments
• Understand and speak English
• Follow instructions without major cognitive or behavioral issues
• Have received a stable disease-modifying therapy (DMT) dose for ≥6 months
• Be classified as a sitter or walker (SMA type 2 or 3) with supported standing ability (score ≥1 on Item 18 of the HFMSE) without KAFOs, AFOs, or external standing devices

Exclusion Criteria:

• Orthopedic surgery within the past year
• New pharmaceutical treatment during the study
• Concurrent participation in another clinical trial

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: 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 over 3 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
Hammersmith Functional Motor Scale - Expanded (HFMSE)	Gross motor function measure; range: 0 to 66; higher scores indicate better motor function	3 assessments: Baseline, immediately after the intervention, 3 months follow-up

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility questionnaire	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
Daily activity logs	Implementation feasibility measure	Every day during the intervention (assessed up to 15 weeks)
Surface electromyography (sEMG)	Surface electromyography (sEMG) will be recorded from muscles of both lower extremities to assess rehabilitation-induced neuroplasticity and neuromuscular adaptations, including muscle fatigability and motor unit recruitment, during standardized endurance tasks.	3 assessments: Baseline, immediately after the intervention, 3 months follow-up
Revised Upper Limb Module (RULM)	Upper limb function measure; range: 0 to 37; higher scores indicate better uper limb function	3 assessments: Baseline, immediately after the intervention, 3 months follow-up
Endurance Shuttle box and blocks test (ESBBT)	Dexterity and upper extremity endurance/fatigability; range: O to 20 minutes (time to limitation); higher times correspond to better endurance	3 assessments: Baseline, immediately after the intervention, 3 months follow-up
6-Minute Walk Test (6MWT)	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.	3 assessments: Baseline, immediately after the intervention, 3 months follow-up
10 Meter Walking Test (10MWT)	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.	3 assessments: Baseline, immediately after the intervention, 3 months follow-up
Timed Up and Go (TUG)	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.	3 assessments: Baseline, immediately after the intervention, 3 months follow-up
30-Second Sit-to-Stand Test (30STS)	Lower limb strength and endurance measure; range: 0 (unable to stand) to higher values reflecting better lower limb strength and endurance	3 assessments: Baseline, immediately after the intervention, 3 months follow-up
Trunk Control Measurement Scale (TCMS)	Static and dynamic trunk control measure; range: 0 to 58 points; higher scores indicating better trunk control	3 assessments: Baseline, immediately after the intervention, 3 months follow-up
Spinal Muscular Atrophy EFFORT (SMA EFFORT)	Questionnaire of perceived physical fatigability (ages 12 years and older); different subscales; higher scores indicating greater perceived fatigue	3 assessments: Baseline, immediately after the intervention, 3 months follow-up
Canadian Occupational Performance Measure (COPM)	Interview of occupational performance and satisfaction; range: 1 to 10 for each item, with higher scores indicating better performance or greater satisfaction.	3 assessments: Baseline, immediately after the intervention, 3 months follow-up

Collaborators and Investigators

• Sponsor: Teachers College, Columbia University

Publications and helpful links

General Publications

• Mercuri E, Finkel RS, Muntoni F, Wirth B, Montes J, Main M, Mazzone ES, Vitale M, Snyder B, Quijano-Roy S, Bertini E, Davis RH, Meyer OH, Simonds AK, Schroth MK, Graham RJ, Kirschner J, Iannaccone ST, Crawford TO, Woods S, Qian Y, Sejersen T; SMA Care Group. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018 Feb;28(2):103-115. doi: 10.1016/j.nmd.2017.11.005. Epub 2017 Nov 23.
• Bleyenheuft Y, Ebner-Karestinos D, Surana B, Paradis J, Sidiropoulos A, Renders A, Friel KM, Brandao M, Rameckers E, Gordon AM. Intensive upper- and lower-extremity training for children with bilateral cerebral palsy: a quasi-randomized trial. Dev Med Child Neurol. 2017 Jun;59(6):625-633. doi: 10.1111/dmcn.13379. Epub 2017 Jan 30.
• Bleyenheuft Y, Gordon AM. Hand-arm bimanual intensive therapy including lower extremities (HABIT-ILE) for children with cerebral palsy. Phys Occup Ther Pediatr. 2014 Nov;34(4):390-403. doi: 10.3109/01942638.2014.932884. Epub 2014 Oct 1.

Study record dates

Study Major Dates

• Study Start (Estimated): March 15, 2026
• Primary Completion (Estimated): February 28, 2028
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: March 13, 2026
• First Submitted That Met QC Criteria: March 18, 2026
• First Posted (Actual): March 23, 2026

Study Record Updates

• Last Update Posted (Actual): March 23, 2026
• Last Update Submitted That Met QC Criteria: March 18, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Rehabilitation; Pediatric; Motor control; Intensive; Functional Strenght Training; HABIT-ILE
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; Motor Neuron Disease; Muscular Atrophy, Spinal
• Other Study ID Numbers: 25-429-2

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