Status:
COMPLETED
A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
Lead Sponsor:
Pfizer
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
Phase:
NA
Brief Summary
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...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
July 24 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 21 2023
Estimated Enrollment :
312 Patients enrolled
Trial Details
Trial ID
NCT03760029
Start Date
July 24 2019
End Date
March 21 2023
Last Update
September 19 2024
Active Locations (8)
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1
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, China, 400014
2
Children's Hospital of Chongqing Medical University (Liangjiang Branch)
Chongqing, Chongqing Municipality, China, 401122
3
The First Affiliated Hospital Of Fujian Medical University
Fuzhou, Fujian, China, 350005
4
Peking University First Hospital
Beijing, China, 100034