NSAA NON-Interventional Study Protocol

Red Nucleus Enterprise Solutions

Status: Completed

Conditions: Duchenne Muscular Dystrophy

Treatments: Other: Remote Administration of NSAA

Study type: Observational

Funder types: Industry

Identifiers

NCT06054971

NSAA-RWE-001

Details and patient eligibility

About

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.

Enrollment: 31 patients

Sex: All

Ages: 4 to 12 years old

Volunteers: No Healthy Volunteers

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

Trial design

31 participants in 2 patient groups

Group A

Description:

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

Treatment:

Other: Remote Administration of NSAA

Group B

Description:

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

Treatment:

Other: Remote Administration of NSAA