Clinical trial NCT04254172: A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy
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)
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Duchenne Muscular Dystrophy (DMD)
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2020-02-05
Enrollment: 2
Sex: MALE
Minimum age (years): 4
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Ambulatory boys with Duchenne muscular dystrophy who receive care at Nationwide Children's Hospital

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.


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.

Primary outcomes:
measure: Mean change from baseline and variability of activity measures
timeFrame: baseline, 3, 6, 9, and 12 months
Secondary outcomes:
measure: Mean change from baseline in functional assessment scores obtained in the clinic
timeFrame: baseline, 3, 6, 9, and 12 months
measure: Comparison of mean changes from baseline and correlation coefficient between activity monitoring data and functional data obtained in clinic
timeFrame: baseline, 3, 6, 9, and 12 months
Other outcomes:
Locations:
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: