- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03760029
A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
April 3, 2023 updated by: Pfizer
A NATURAL HISTORY STUDY IN CHINESE MALE PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY
This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients. A total of approximately 330 subjects will be enrolled with the target number of subjects in each group as below:
- Group 1, Ambulatory subjects aged <6 years, approximately 100 subjects;
- Group 2, Ambulatory subjects aged >=6 years, approximately 180 subjects;
- Group 3, Non-ambulatory subjects, approximately 50 subjects. Subjects will visit sites every 6 months. Each subject will be observed for at least 24 months. All subjects will remain enrolled until the study completion date, such that some will have data collected after Month 24. Subjects, who complete Visit 5/Month 24 at least 6 months prior to study completion, will be asked to complete an additional visit at Month 30.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
312
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Beijing, China, 100034
- Peking University First Hospital
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Beijing, China, 100045
- Beijing Children's Hospital, Capital Medical University
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Shanghai, China, 200040
- Huashan Hospital, Fudan University
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Shanghai, China, 201102
- Children's Hospital of Fudan University
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Shanghai, China, 201102
- Affiliated children's hospital of fudan university
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Chongqing
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Chongqing, Chongqing, China, 400014
- Children's Hospital of Chongqing Medical University
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Chongqing, Chongqing, China, 401122
- Children's Hospital of Chongqing Medical University (Liangjiang Branch)
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Fujian
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Fuzhou, Fujian, China, 350005
- The First Affiliated Hospital of Fujian Medical University
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 second and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
Male
Description
Inclusion Criteria:
- Chinese male patients with any age, diagnosed with DMD. Diagnosis must be confirmed in subject's medical history and by genetic testing obtained during routine clinical care for diagnostic purposes as reported from an appropriate regulated laboratory using a clinically validated genetic test (genetic testing is not provided by the sponsor).
- Subjects who are >=4 years old must be receiving glucocorticosteroids for a minimum of 6 months prior to signing informed consent. There should be no significant change (<0.2 mg/kg) in dosage or dose regimen (not related to body weight change) for at least 3 months immediately prior to signing the informed consent. Subjects who are aged >4 years will be exempt from this requirement; those not taking GC will be eligible if the initiation of GC treatment in these subjects is considered inappropriate in the opinion of Investigators.
Exclusion Criteria:
- Any injury which may impact functional testing. 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.
- Presence or history of other musculoskeletal or neurologic disease or somatic disorder not related to DMD including pulmonary, cardiac, and cognitive diseases.
- Subjects >=4 years old who have not completed the varicella vaccination.
- Participation in other studies involving investigational drug(s) for a minimum of 90 days prior to signing the informed consent and/or during study participation.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Other
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Other: Study arm
All subjects in this study will be observed for 24-30 months.
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All subjects need to visit sites more frequently than in routine clinical practice.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Time to life altering clinical milestones due to DMD disease progression - failure to walk
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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Change from baseline, 6, 12, 18, 24, 30 Months
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Time to life altering clinical milestones due to DMD disease progression - failure to stand
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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Change from baseline, 6, 12, 18, 24, 30 Months
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Time to life altering clinical milestones due to DMD disease progression - failure to self feed
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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Change from baseline, 6, 12, 18, 24, 30 Months
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Change from Baseline in timed motor functions for ambulatory subjects - rise from floor (supine to stand)
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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Change from baseline, 6, 12, 18, 24, 30 Months
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Change from Baseline in timed motor functions for ambulatory subjects - 10 meter walk/run
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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Change from baseline, 6, 12, 18, 24, 30 Months
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Change from Baseline in clinical evaluator determined scales - Northstar Ambulatory Assessment
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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The Northstar Ambulatory Assessment will be performed in ambulatory children >=3 years old
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Change from baseline, 6, 12, 18, 24, 30 Months
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Change from Baseline in clinical evaluator determined scales - Performance of Upper Limb 2.0
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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The Performance of Upper Limb 2.0 will be administered in subjects >=10 years old.
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Change from baseline, 6, 12, 18, 24, 30 Months
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Change from Baseline in strength of muscle groups - knee extension
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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Change from baseline, 6, 12, 18, 24, 30 Months
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Change from Baseline in strength of muscle groups - elbow flexion
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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Change from baseline, 6, 12, 18, 24, 30 Months
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Change from Baseline in strength of muscle groups - elbow extension
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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Change from baseline, 6, 12, 18, 24, 30 Months
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Change from Baseline in strength of muscle groups - shoulder abduction.
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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Change from baseline, 6, 12, 18, 24, 30 Months
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Change from Baseline in pulmonary function tests - Percent predicted Forced Vital Capacity (%pFVC)
Time Frame: Change from baseline, 12, 24, 30 Months
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Change from baseline, 12, 24, 30 Months
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Change from Baseline in pulmonary function tests - Forced Expiratory Volume in one Second
Time Frame: Change from baseline, 12, 24, 30 Months
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Change from baseline, 12, 24, 30 Months
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Change from Baseline in pulmonary function tests - Maximum Inspiratory Pressure
Time Frame: Change from baseline, 12, 24, 30 Months
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Change from baseline, 12, 24, 30 Months
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Change from Baseline in pulmonary function tests - Maximum Expiratory Pressure
Time Frame: Change from baseline, 12, 24, 30 Months
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Change from baseline, 12, 24, 30 Months
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Change from Baseline in pulmonary function tests - Peak cough flow
Time Frame: Change from baseline, 12, 24, 30 Months
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Change from baseline, 12, 24, 30 Months
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Change from Baseline in range of motion (ROM) at bilateral ankles and elbows
Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months
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Change from baseline, 6, 12, 18, 24, 30 Months
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Change from Baseline in left ventricular ejection fraction (LVEF)
Time Frame: Change from baseline, 12, 24, 30 Months
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Change from baseline, 12, 24, 30 Months
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Change from Baseline in Wechsler Intelligence Scale for Children (WISC) score in ambulatory subjects aged >=6 years to <=16 years
Time Frame: Chang from baseline, 24 Months
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The WISC is individually administered intelligence test for children between ages of 6 and 16.
It generates a Full Scale Intelligence Quotient.
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Chang from baseline, 24 Months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Proportion of subjects with mutation of large deletion
Time Frame: Baseline
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Baseline
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Proportion of subjects with mutation of large duplication
Time Frame: Baseline
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Baseline
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Proportion of subjects with mutation of small insertion
Time Frame: Baseline
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Baseline
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Proportion of subjects with mutation of small deletion
Time Frame: Baseline
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Baseline
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Proportion of subjects with mutation of point mutation
Time Frame: Baseline
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Baseline
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Proportion of subjects with mutations involving key high frequency mutation sites
Time Frame: Baseline
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Baseline
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Change from Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) score
Time Frame: Baseline, 12, 24, 30 Months
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The Pediatric Outcomes Data Collection Instrument is a patient-reported assessment of musculoskeletal health intended for use in children and adolescents.
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Baseline, 12, 24, 30 Months
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Change from Baseline in EuroQoL 5 Dimension 3 Level (EQ-5D-3L)
Time Frame: Baseline, 12, 24, 30 Months
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The EQ-5D-3L is instrument measuring health-related quality of life for patients >=16 years old.
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Baseline, 12, 24, 30 Months
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Change from Baseline in EuroQoL 5 Dimension Youth (EQ-5D-Y)
Time Frame: Baseline, 12, 24, 30 Months
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EQ-5D-Y is a generic instrument measuring health-related quality of life in children and adolescents younger than 16 years old and able to read and complete the questionnaire.
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Baseline, 12, 24, 30 Months
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Change from Baseline in Healthcare Resource Utilization (HRU) score
Time Frame: Baseline, 12, 24, 30 Months
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The result collection is based on the HRU questionnaire.
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Baseline, 12, 24, 30 Months
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Change from Baseline in Work Productivity and Activity Impairment Questionnaire adapted for Caregiving (WPAI:CG) impairment scores
Time Frame: Baseline, 12, 24, 30 Months
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WPAI:CG will measure the impact of a subject with DMD on a caregiver's work productivity and regular activities.
It will be administered as questionnaire.
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Baseline, 12, 24, 30 Months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
July 24, 2019
Primary Completion (Actual)
March 21, 2023
Study Completion (Actual)
March 21, 2023
Study Registration Dates
First Submitted
November 15, 2018
First Submitted That Met QC Criteria
November 28, 2018
First Posted (Actual)
November 30, 2018
Study Record Updates
Last Update Posted (Actual)
April 4, 2023
Last Update Submitted That Met QC Criteria
April 3, 2023
Last Verified
April 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- C3391004
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
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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