NSAA NON-Interventional Study Protocol

NCT06054971 | Completed

• 1 other identifier: NSAA-RWE-001
• Study Type: observational
• Target: 31
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

Group A

Group A will have Live telemedicine assessments completed prior to caregiver Asynchronous Video

Other: Remote Administration of NSAA

Group B

Group B will have Caregiver Asynchronous Video first, followed by live telemedicine

Other: Remote Administration of NSAA

Interventions

Remote Administration of NSAA OTHER

Remote Administration of NSAA

Group A; Group B

Eligibility Criteria

• Age: 4 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Individuals with confirmed diagnosis of DMD

You may qualify if:

• 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.
• Confirmed diagnosis of DMD
• Participant with DMD aged 4 to12 (inclusive) at t Enrollment date
• Participant with DMD is ambulatory without assistive devices, braces, or aids throughout the study
• Caregiver has access to and/or willingness to learn use of a smart phone and the iTC study mobile application
• Participant and caregiver are based in the US throughout study
• Participant and caregiver are fluent in the English language (verbally and in writing)

You may not qualify if:

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

Sponsors & Collaborators

• Red Nucleus Enterprise Solutions, LLC: lead
• Pfizer: collaborator

Study Sites (1)

Red Nucleus Solutions

Malvern, Pennsylvania, 19355, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 12, 2023
• First Posted: September 26, 2023
• Study Start: October 2, 2023
• Primary Completion: February 3, 2025
• Study Completion: March 13, 2025
• Last Updated: March 21, 2025

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)