A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
NCT03760029
ABOUT THIS STUDY
FOR MORE INFORMATION
Contact a representative by phone, email, or visiting the study website. Please see the references below:
Pfizer Clinical Trials Contact Center
1-800-718-1021
1. 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).
2. 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.
1. 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.
2. Presence or history of other musculoskeletal or neurologic disease or somatic disorder
not related to DMD including pulmonary, cardiac, and cognitive diseases.
3. Subjects >=4 years old who have not completed the recommended national immunization
schedule and the varicella vaccination.
4. 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.
NEED INFO?
Questions about a trial? Call or email to reach a Pfizer Clinical Trial Contact Center Representative
TRY A NEW SEARCH
Search for Clinical Trials by condition, keyword or trial number. Share your location or enter your city or zip code to find studies near you.
Based on your search, you may also be interested in
- Los Angeles, California
- Los Angeles, California
- Los Angeles, California
- Sacramento, California
- Aurora, Colorado
- Tampa, Florida
- Chicago, Illinois
- Iowa City, Iowa
- Fairway, Kansas
- Fairway, Kansas
- Kansas City, Kansas
- Kansas City, Kansas
- Baltimore, Maryland
- Baltimore, Maryland
- Baltimore, Maryland
- Baltimore, Maryland
- Boston, Massachusetts
- Minneapolis, Minnesota
- Saint Louis, Missouri
- Durham, North Carolina
- Durham, North Carolina
- Cincinnati, Ohio
- Philadelphia, Pennsylvania
- Pittsburgh, Pennsylvania
- Salt Lake City, Utah
- Salt Lake City, Utah
- Salt Lake City, Utah
- Salt Lake City, Utah
- Salt Lake City, Utah
- Salt Lake City, Utah
- South Brisbane, Queensland
- Sofia,
- Sofia,
- Sofia,
- Sofia,
- Sofia,
- Calgary, Alberta
- Vancouver, British Columbia
- London, Ontario
- Montreal, Quebec
- Genova,
- Genova,
- Genova,
- Genova,
- Genova,
- Rome,
- Rome,
- Rome,
- Rome,
- Rome,
- Rome,
- Hyogo,
- Tokyo,
- Warszawa,
- Warszawa,
- Warszawa,
- Warszawa,
- Warszawa,
- Warszawa,
- Liverpool,
- Liverpool,
- London,
- London,
- Newcastle upon Tyne,
- Newcastle upon Tyne,
- Newcastle-upon-Tyne,
- Chongqing, Chongqing
- Chongqing, Chongqing
- Fuzhou, Fujian
- Beijing,
- Beijing,
- Shanghai,
- Shanghai,
- Shanghai,
- Salt Lake City, Utah
- Los Angeles, California
- Los Angeles, California
- Los Angeles, California
- Los Angeles, California
- Los Angeles, California
- Los Angeles, California
- Los Angeles, California
- Durham, North Carolina
- Durham, North Carolina
- Durham, North Carolina
- Durham, North Carolina
- Durham, North Carolina
- Durham, North Carolina
- Salt Lake City, Utah
- Salt Lake City, Utah
- Salt Lake City, Utah
- Salt Lake City, Utah
- Salt Lake City, Utah
- Salt Lake City, Utah
- Salt Lake City, Utah
- Roma,
- Jerusalem,
- Petach Tikvah,
- Esplugues de Llobregat, Barcelona
- Barcelona,
- Valencia,
Descriptive Information | |||||
---|---|---|---|---|---|
Brief Title ICMJE | A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy | ||||
Official Title ICMJE | A NATURAL HISTORY STUDY IN CHINESE MALE PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY | ||||
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 number of subjects in each group as below:
| ||||
Detailed Description | Not Provided | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Not Applicable | ||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Other | ||||
Condition ICMJE | Duchenne Muscular Dystrophy | ||||
Intervention ICMJE | Other: Visit frequency
All subjects need to visit sites more frequently than in routine clinical practice. | ||||
Study Arms ICMJE | Study arm
All subjects in this study will be observed for 24-30 months. Intervention: Other: Visit frequency | ||||
Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | |||||
Recruitment Information | |||||
Recruitment Status ICMJE | Recruiting | ||||
Estimated Enrollment ICMJE | 330 | ||||
Original Estimated Enrollment ICMJE | Same as current | ||||
Estimated Study Completion Date ICMJE | March 25, 2023 | ||||
Estimated Primary Completion Date | March 25, 2023 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
| ||||
Sex/Gender ICMJE |
| ||||
Ages ICMJE | Child, Adult, Older Adult | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE |
| ||||
Listed Location Countries ICMJE | China | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT03760029 | ||||
Other Study ID Numbers ICMJE | C3391004 | ||||
Has Data Monitoring Committee | No | ||||
U.S. FDA-regulated Product |
| ||||
IPD Sharing Statement ICMJE |
| ||||
Responsible Party | Pfizer | ||||
Study Sponsor ICMJE | Pfizer | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
| ||||
PRS Account | Pfizer | ||||
Verification Date | January 2021 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |