A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy

Study Overview
=================
Brief Summary
-----------------
The purpose of this low interventional study is to collect data on everyday movement in boys with Duchenne muscular dystrophy (DMD) using wearable activity sensors. The activity sensors could provide useful information beyond what is currently collected by functional (movement, strength) assessments in clinic. This information can help with the understanding of the impact of DMD, and perhaps with how possible treatments can affect this impact.

Official Title
-----------------
A SINGLE-SITE, PROSPECTIVE, NATURAL HISTORY LOW INTERVENTIONAL STUDY TO ESTABLISH NORMATIVE DATA OF REAL-WORLD ACTIVITY MEASURES USING WEARABLE SENSORS IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY (DMD)

Conditions
-----------------
Duchenne Muscular Dystrophy (DMD)

Intervention / Treatment
-----------------
* Device: Activity Monitor


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing Body weight between 15 and 50 kg Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry Ability to rise from floor within seven (7) seconds and ability to walk Exclusion Criteria: Current exposure to systemic immunosuppressant agents other than glucocorticoids. Prior exposure to any gene therapy agent, including exon-skipping and missense agents. Exposure to other investigational drugs within 30 days or 5 half-lives, whichever is longer. Any injury which may impact functional testing per investigator's judgement. Previous injuries must be fully healed prior to consenting. Prior lower limb fractures must be fully healed and at least 3 months from injury date at screening. Any planned surgeries which may impact physical activity and performance. Presence or history of musculoskeletal or neurological disease in addition to DMD. Any known allergies or skin reactions to stainless steel, versaflex, and silicon that may cause possible discomfort by wearable sensors. Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, cancer, autoimmune or allergic disease that may interfere with the study conduct as per investigator's judgment, excluding untreated, asymptomatic, seasonal allergies at time of screening. Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are Pfizer employees, including their family members, directly involved in the conduct of the study.

Ages Eligible for Study
-----------------
Minimum Age: 4 Years
Maximum Age: 12 Years

Sexes Eligible for Study
-----------------
Male

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Single cohort<br>There is no randomization or stratification in this study. All subjects will complete the same study assessments. | Device: Activity Monitor<br>* Wrist and ankle sensors to be worn continuously for 2-week intervals.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change from baseline and variability of activity measures |  | baseline, 3, 6, 9, and 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change from baseline in functional assessment scores obtained in the clinic |  | baseline, 3, 6, 9, and 12 months | 
| Comparison of mean changes from baseline and correlation coefficient between activity monitoring data and functional data obtained in clinic |  | baseline, 3, 6, 9, and 12 months |