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

NCT ID: NCT04254172

Last Updated: 2021-10-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Total Enrollment

2 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-02-19

Study Completion Date

2020-08-19

Brief Summary

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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.

Detailed Description

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Conditions

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Duchenne Muscular Dystrophy (DMD)

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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Single cohort

There is no randomization or stratification in this study. All subjects will complete the same study assessments.

Activity Monitor

Intervention Type DEVICE

Wrist and ankle sensors to be worn continuously for 2-week intervals.

Interventions

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Activity Monitor

Wrist and ankle sensors to be worn continuously for 2-week intervals.

Intervention Type DEVICE

Eligibility Criteria

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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.
Minimum Eligible Age

4 Years

Maximum Eligible Age

12 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Countries

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United States

Related Links

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https://pmiform.com/clinical-trial-info-request?StudyID=C3391005

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

Other Identifiers

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C3391005

Identifier Type: -

Identifier Source: org_study_id