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

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
5

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 1, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 25, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 30, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 31, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 16, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 13, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings