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A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

Last updated on December 6, 2018

FOR MORE INFORMATION
Study Location
Contact
1-800-718-1021
Eligibility criteria
Condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Duchenne Muscular Dystrophy
Sex
Male
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
0 +
Inclusion criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

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
( 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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details

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.

NCT03760029
Pfizer
Not yet recruiting
A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

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A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
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, subjects aged <6 years, 100 subjects;
  • Group 2, subjects aged >=6 years and <12 years, 180 subjects;
  • Group 3, subjects aged >=12 years, 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.
Not Provided
Interventional
Not Applicable
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Duchenne Muscular Dystrophy
Other: Visit frequency
All subjects need to visit sites more frequently than in routine clinical practice.
Study arm
All subjects in this study will be observed for 24-30 months.
Intervention: Other: Visit frequency
Not Provided


*   Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Not yet recruiting
330
Same as current
August 2021
August 2021   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  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.

Exclusion Criteria:

  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.
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Child, Adult, Older Adult
No

Contact: Pfizer CT.gov Call Center 1-800-718-1021 [email protected]
Not Provided
 
 
NCT03760029
C3391004
No
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Plan Description: Information relating to our policy on data sharing and the process for requesting data can be found at the following link: http://www.pfizer.com/research/clinical_trials/trial_data_and_results/da...
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
December 2018

ICMJE     Data element required by the

International Committee of Medical Journal Editors
and the
World Health Organization ICTRP

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting thte study website. To get updates and notications about this trail, sign up using the form below.

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1-800-718-1021

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