Status:

COMPLETED

NSAA NON-Interventional Study Protocol

Lead Sponsor:

Red Nucleus Enterprise Solutions, LLC

Collaborating Sponsors:

Pfizer

Conditions:

Duchenne Muscular Dystrophy

Eligibility:

All Genders

4-12 years

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)...

Eligibility Criteria

Inclusion

  • 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)

Exclusion

  • 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

Key Trial Info

Start Date :

October 2 2023

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

March 13 2025

Estimated Enrollment :

31 Patients enrolled

Trial Details

Trial ID

NCT06054971

Start Date

October 2 2023

End Date

March 13 2025

Last Update

March 21 2025

Active Locations (1)

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Red Nucleus Solutions

Malvern, Pennsylvania, United States, 19355