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

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)

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.

Study Overview

• Status: Terminated
• Conditions: Duchenne Muscular Dystrophy (DMD)
• Intervention / Treatment: Device: Activity Monitor

• Study Type: Observational
• Enrollment (Actual): 2

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 12 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Non-Probability Sample

Study Population

Ambulatory boys with Duchenne muscular dystrophy who receive care at Nationwide Children's Hospital

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): February 19, 2020
• Primary Completion (Actual): August 19, 2020
• Study Completion (Actual): August 19, 2020

Study Registration Dates

• First Submitted: January 31, 2020
• First Submitted That Met QC Criteria: January 31, 2020
• First Posted (Actual): February 5, 2020

Study Record Updates

• Last Update Posted (Actual): October 15, 2021
• Last Update Submitted That Met QC Criteria: October 14, 2021
• Last Verified: October 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: C3391005

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: No