Recruiting

Observational Study

Sponsor:

Jacqueline Montes

Code:

NCT07400198

Conditions

Spinal Muscular Atrophy Type 3

Ambulatory Spinal Muscular Atrophy

Eligibility Criteria

Sex: All

Age: 8 - 50

Healthy Volunteers: Not accepted

Study Details

Brief summary:

The objective of this study is to understand how spatiotemporal and kinetic gait parameters are associated with bone health in pwSMA who are receiving DMT. We hypothesize that gait parameters are associated with BMD and can determine fracture risk in pwSMA. This is an observational study that involves one in-clinic visit (approximately 3 hours) with remote follow-up visits (approximately 15 minutes) every 3 months for 1 year to collect fracture and medical history. Each of the assessments included in this study are non-interventional and are not intended as a treatment nor are they at the level of standard of care.

Conditions

Spinal Muscular Atrophy Type 3

Ambulatory Spinal Muscular Atrophy

Study ID

NCT07400198

Start date

Mar 5, 2026

Status verified date

Mar, 2026

Completion date

Jun 30, 2027

Anticipated

Primary completion date

Jun 30, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 8 - 50

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Confirmed diagnosis of 5qSMA
  • Between 8 and 50 years of age
  • Able to walk 10 meters without support or an assistive device
  • All participants who meet the inclusion criteria regardless of treatment status will be included, including those on adjuvant therapies, whether investigational or approved

Exclusion Criteria:

  • Injury or surgery within previous 3 months that would impact ability to perform in-clinic gait assessments
  • Unwilling or unable to comply with all study procedures
  • Age less than 8 or over 50 years

Study Design

Enrollment

22 participants

Anticipated

Interventions and Outcome Measures

Arms

Spinal Muscular Atrophy

Ambulatory children and adults at least 8 years old by the time of enrollment with genetically confirmed SMA.

Primary outcome measure

  • Dual X-Ray Absorptiometry (DXA) [ Time Frame: Baseline ]
  • Blood collection [ Time Frame: Baseline ]
  • Fracture history [ Time Frame: Baseline, months 3, 6, 9, and 12 ]
  • Fall history [ Time Frame: Baseline, months 3, 6, 9 and 12 ]
  • Collection of spatiotempoal and kinetic gait parameters using instrumented insoles (AI-Sole) [ Time Frame: Baseline ]
  • Six Minute Walk Test [ Time Frame: Baseline ]
  • IPAQ-SF [ Time Frame: Baseline ]

Central Contacts and Locations

Central contacts

Cara Kanner, PT, DPT, EdD

516-581-8150chy2112@cumc.columbia.edu

Locations

Columbia University Irving Medical Center

Recruiting

New York, New York, United States, 10032

Contacts

Jacqueline Montes

212-305-8916jm598@cumc.columbia.edu

Michael Lutzker

9257055133ml5121@cumc.columbia.edu

More Information

Sponsor

Jacqueline Montes

Last update posted

Mar 18, 2026

Last verified

Mar, 2026

Keywords

  • spinal muscular atrophy
  • SMA
  • instrumented insole
  • bone health
  • fracture
  • bone mineral density
  • gait
  • low bone mass

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Jacqueline Montes on 2026-03-18.