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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03760029
• Other study ID #: C3391004
• Status: Completed
• Phase: N/A
• Start date: July 24, 2019
• Est. completion date: March 21, 2023

Study information

• Verified date: April 2023
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 312
• Est. completion date: March 21, 2023
• Est. primary completion date: March 21, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 0 Years and older

Eligibility

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 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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Other:
• Visit frequency
All subjects need to visit sites more frequently than in routine clinical practice.

Locations

Country	Name	City	State
China	Beijing Children's Hospital, Capital Medical University	Beijing
China	Peking University First Hospital	Beijing
China	Children's Hospital of Chongqing Medical University	Chongqing	Chongqing
China	Children's Hospital of Chongqing Medical University (Liangjiang Branch)	Chongqing	Chongqing
China	The First Affiliated Hospital Of Fujian Medical University	Fuzhou	Fujian
China	Affiliated children's hospital of fudan university	Shanghai
China	Children's Hospital of Fudan University	Shanghai
China	Huashan Hospital, Fudan University	Shanghai

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time to life altering clinical milestones due to DMD disease progression - failure to walk		Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Time to life altering clinical milestones due to DMD disease progression - failure to stand		Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Time to life altering clinical milestones due to DMD disease progression - failure to self feed		Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Change from Baseline in timed motor functions for ambulatory subjects - rise from floor (supine to stand)		Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Change from Baseline in timed motor functions for ambulatory subjects - 10 meter walk/run		Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Change from Baseline in clinical evaluator determined scales - Northstar Ambulatory Assessment	The Northstar Ambulatory Assessment will be performed in ambulatory children >=3 years old	Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Change from Baseline in clinical evaluator determined scales - Performance of Upper Limb 2.0	The Performance of Upper Limb 2.0 will be administered in subjects >=10 years old.	Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Change from Baseline in strength of muscle groups - knee extension		Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Change from Baseline in strength of muscle groups - elbow flexion		Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Change from Baseline in strength of muscle groups - elbow extension		Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Change from Baseline in strength of muscle groups - shoulder abduction.		Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Change from Baseline in pulmonary function tests - Percent predicted Forced Vital Capacity (%pFVC)		Change from baseline, 12, 24, 30 Months
Primary	Change from Baseline in pulmonary function tests - Forced Expiratory Volume in one Second		Change from baseline, 12, 24, 30 Months
Primary	Change from Baseline in pulmonary function tests - Maximum Inspiratory Pressure		Change from baseline, 12, 24, 30 Months
Primary	Change from Baseline in pulmonary function tests - Maximum Expiratory Pressure		Change from baseline, 12, 24, 30 Months
Primary	Change from Baseline in pulmonary function tests - Peak cough flow		Change from baseline, 12, 24, 30 Months
Primary	Change from Baseline in range of motion (ROM) at bilateral ankles and elbows		Change from baseline, 6, 12, 18, 24, 30 Months
Primary	Change from Baseline in left ventricular ejection fraction (LVEF)		Change from baseline, 12, 24, 30 Months
Primary	Change from Baseline in Wechsler Intelligence Scale for Children (WISC) score in ambulatory subjects aged >=6 years to <=16 years	The WISC is individually administered intelligence test for children between ages of 6 and 16. It generates a Full Scale Intelligence Quotient.	Chang from baseline, 24 Months
Secondary	Proportion of subjects with mutation of large deletion		Baseline
Secondary	Proportion of subjects with mutation of large duplication		Baseline
Secondary	Proportion of subjects with mutation of small insertion		Baseline
Secondary	Proportion of subjects with mutation of small deletion		Baseline
Secondary	Proportion of subjects with mutation of point mutation		Baseline
Secondary	Proportion of subjects with mutations involving key high frequency mutation sites		Baseline
Secondary	Change from Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) score	The Pediatric Outcomes Data Collection Instrument is a patient-reported assessment of musculoskeletal health intended for use in children and adolescents.	Baseline, 12, 24, 30 Months
Secondary	Change from Baseline in EuroQoL 5 Dimension 3 Level (EQ-5D-3L)	The EQ-5D-3L is instrument measuring health-related quality of life for patients >=16 years old.	Baseline, 12, 24, 30 Months
Secondary	Change from Baseline in EuroQoL 5 Dimension Youth (EQ-5D-Y)	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.	Baseline, 12, 24, 30 Months
Secondary	Change from Baseline in Healthcare Resource Utilization (HRU) score	The result collection is based on the HRU questionnaire.	Baseline, 12, 24, 30 Months
Secondary	Change from Baseline in Work Productivity and Activity Impairment Questionnaire adapted for Caregiving (WPAI:CG) impairment scores	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.	Baseline, 12, 24, 30 Months

External Links

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