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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04254172
Recruitment Status : Terminated (This low-interventional study (involving no study drug) has been terminated early due to the COVID-19 pandemic, and not for any safety or efficacy reasons.)
First Posted : February 5, 2020
Last Update Posted : October 8, 2020
Sponsor:
Information provided by (Responsible Party):
Pfizer

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.

Condition or disease Intervention/treatment
Duchenne Muscular Dystrophy (DMD) Device: Activity Monitor

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Study Type : Observational
Actual Enrollment : 2 participants
Observational Model: Case-Only
Time Perspective: Prospective
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)
Actual Study Start Date : February 19, 2020
Actual Primary Completion Date : August 19, 2020
Actual Study Completion Date : August 19, 2020


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.




Primary Outcome Measures :
  1. Mean change from baseline and variability of activity measures [ Time Frame: baseline, 3, 6, 9, and 12 months ]

Secondary Outcome Measures :
  1. Mean change from baseline in functional assessment scores obtained in the clinic [ Time Frame: baseline, 3, 6, 9, and 12 months ]
  2. Comparison of mean changes from baseline and correlation coefficient between activity monitoring data and functional data obtained in clinic [ Time Frame: baseline, 3, 6, 9, and 12 months ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   4 Years to 12 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Ambulatory boys with Duchenne muscular dystrophy who receive care at Nationwide Children's Hospital
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04254172


Locations
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United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT04254172    
Other Study ID Numbers: C3391005
First Posted: February 5, 2020    Key Record Dates
Last Update Posted: October 8, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
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.

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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
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked