NCT03760029

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

A NATURAL HISTORY STUDY IN CHINESE MALE PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 312 (actual)
• Sponsor: Pfizer · Industry
• Sex: Male
• Age: 0 Years
• Healthy volunteers: Not accepted

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: * 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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
OTHER	Visit frequency	All subjects need to visit sites more frequently than in routine clinical practice.

Timeline

• Start date: 2019-07-24
• Primary completion: 2023-03-21
• Completion: 2023-03-21
• First posted: 2018-11-30
• Last updated: 2024-09-19
• Results posted: 2024-09-19

Locations

8 sites across 1 country: China