NSAA NON-Interventional Study Protocol

Completed

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Other: Remote Administration of NSAA

• Registration Number: NCT06054971
• Lead Sponsor: Red Nucleus Enterprise Solutions, LLC

Brief Summary

The purpose of this non-interventional study is to evaluate the feasibility of remotely administering the North Star Ambulatory Assessment (NSAA) to participants with Duchenne muscular dystrophy (DMD). The iTakeControl (iTC) software platform will be utilized to remotely administer and score the NSAAs.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-09-12
• Study First Submitted QC: 2023-09-19
• Study First Posted: 2023-09-26
• Study Start: 2023-10-02
• Status Verified: 2024-11
• Primary Completion: 2025-02-03
• Study Completion: 2025-03-13
• Last Update Submitted: 2025-03-18
• Last Update Posted: 2025-03-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 31

Inclusion Criteria

1. Evidence of a signed and dated informed consent document indicating that the participant's legally authorized representative (LAR) has been informed of all pertinent aspects of the study, along with evidence of age-appropriate child assent.
2. Confirmed diagnosis of DMD
3. Participant with DMD aged 4 to12 (inclusive) at t Enrollment date
4. Participant with DMD is ambulatory without assistive devices, braces, or aids throughout the study
5. Caregiver has access to and/or willingness to learn use of a smart phone and the iTC study mobile application
6. Participant and caregiver are based in the US throughout study
7. Participant and caregiver are fluent in the English language (verbally and in writing)

Exclusion Criteria

1. Non-ambulatory DMD participant at any study timepoint
2. Caregiver/participant unwilling or unable to administer/perform the NSAA
3. Anticipated deterioration of participant's ambulatory status during the study

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Group A	Remote Administration of NSAA	Group A will have Live telemedicine assessments completed prior to caregiver Asynchronous Video
Group B	Remote Administration of NSAA	Group B will have Caregiver Asynchronous Video first, followed by live telemedicine

Primary Outcome Measures

Name	Time	Method
Comparison of performance and ratings of the NSAA using three methods: 1) Live Telemedicine scores 2) Subsequent Recorded Telemedicine scores 3) Caregiver Asynchronous Video scores	6 Months

Trial Locations

Locations (1)

Red Nucleus Solutions; 🇺🇸 Malvern, Pennsylvania, United States