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

Duchenne Muscular Dystrophy (DMD) Clinical Trial

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04254172
• Other study ID #: C3391005
• Status: Terminated
• Start date: February 19, 2020
• Est. completion date: August 19, 2020

Study information

• Verified date: October 2021
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 2
• Est. completion date: August 19, 2020
• Est. primary completion date: August 19, 2020
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 12 Years

Eligibility

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy (DMD)
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Device:
• Activity Monitor
Wrist and ankle sensors to be worn continuously for 2-week intervals.

Locations

Country	Name	City	State
United States	Nationwide Children's Hospital	Columbus	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean change from baseline and variability of activity measures		baseline, 3, 6, 9, and 12 months
Secondary	Mean change from baseline in functional assessment scores obtained in the clinic		baseline, 3, 6, 9, and 12 months
Secondary	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

External Links

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