Clinical trial NCT03760029: A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
Official title: A NATURAL HISTORY STUDY IN CHINESE MALE PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2018-11-30
Enrollment: 312
Sex: MALE
Minimum age (years): 0
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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:

* 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Age of Participants When They Failed to Walk
timeFrame: Up to Month 30
measure: Age of Participants When They Failed to Stand
timeFrame: Up to Month 30
measure: Age of Participants When They Failed to Self-feed
timeFrame: Up to Month 30
measure: Change From Baseline in Northstar Ambulatory Assessment (NSAA) Total Score at Month 6: Ambulatory Participants Aged >=3 Years
timeFrame: Baseline (Day 1) and Month 6
measure: Change From Baseline in NSAA Total Score at Month 12: Ambulatory Participants Aged >=3 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in NSAA Total Score at Month 18: Ambulatory Participants Aged >=3 Years
timeFrame: Baseline (Day 1) and Month 18
measure: Change From Baseline in NSAA Total Score at Month 24: Ambulatory Participants Aged >=3 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in NSAA Total Score at Month 30: Ambulatory Participants Aged >=3 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Performance of Upper Limb (PUL) 2.0 Total Score at Month 6: Participants Aged >=10 Years
timeFrame: Baseline (Day 1) and Month 6
measure: Change From Baseline in PUL 2.0 Total Score at Month 12: Participants Aged >=10 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in PUL 2.0 Total Score at Month 18: Participants Aged >=10 Years
timeFrame: Baseline (Day 1) and Month 18
measure: Change From Baseline in PUL 2.0 Total Score at Month 24: Participants Aged >=10 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in PUL 2.0 Total Score at Month 30: Participants Aged >=10 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Rise From Floor Velocity at Month 6: Ambulatory Participants Aged >=3 Years Only
timeFrame: Baseline (Day 1) and Month 6
measure: Change From Baseline in Rise From Floor Velocity at Month 12: Ambulatory Participants Aged >=3 Years Only
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in Rise From Floor Velocity at Month 18: Ambulatory Participants Aged >=3 Years Only
timeFrame: Baseline (Day 1) and Month 18
measure: Change From Baseline in Rise From Floor Velocity at Month 24: Ambulatory Participants Aged >=3 Years Only
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in Rise From Floor Velocity at Month 30: Ambulatory Participants Aged >=3 Years Only
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in 10 Meter Walk or Run Velocity at Month 6: Ambulatory Participants Aged >=3 Years
timeFrame: Baseline (Day 1) and Month 6
measure: Change From Baseline in 10 Meter Walk or Run Velocity at Month 12: Ambulatory Participants Aged >=3 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in 10 Meter Walk or Run Velocity at Month 18: Ambulatory Participants Aged >=3 Years
timeFrame: Baseline (Day 1) and Month 18
measure: Change From Baseline in 10 Meter Walk or Run Velocity at Month 24: Ambulatory Participants Aged >=3 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in 10 Meter Walk or Run Velocity at Month 30: Ambulatory Participants Aged >=3 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Knee Extension of Muscle Strength at Month 6: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 6
measure: Change From Baseline in Knee Extension of Muscle Strength at Month 12: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in Knee Extension of Muscle Strength at Month 18: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 18
measure: Change From Baseline in Knee Extension of Muscle Strength at Month 24: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in Knee Extension of Muscle Strength at Month 30: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Elbow Flexion of Muscle Strength at Month 6: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 6
measure: Change From Baseline in Elbow Flexion of Muscle Strength at Month 12: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in Elbow Flexion of Muscle Strength at Month 18: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 18
measure: Change From Baseline in Elbow Flexion of Muscle Strength at Month 24: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in Elbow Flexion of Muscle Strength at Month 30: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Elbow Extension of Muscle Strength at Month 6: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 6
measure: Change From Baseline in Elbow Extension Muscle Strength at Month 12: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in Elbow Extension Muscle Strength at Month 18: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 18
measure: Change From Baseline in Elbow Extension Muscle Strength at Month 24: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in Elbow Extension Muscle Strength at Month 30: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Shoulder Abduction of Muscle Strength at Month 6: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 6
measure: Change From Baseline in Shoulder Abduction of Muscle Strength at Month 12: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in Shoulder Abduction of Muscle Strength at Month 18: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 18
measure: Change From Baseline in Shoulder Abduction of Muscle Strength at Month 24: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in Shoulder Abduction of Muscle Strength at Month 30: Participants Aged >=5 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Range of Motion (ROM) at Bilateral Ankles at Month 6
timeFrame: Baseline (Day 1) and Month 6
measure: Change From Baseline in ROM at Bilateral Ankles at Month 12
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in ROM at Bilateral Ankles at Month 18
timeFrame: Baseline (Day 1) and Month 18
measure: Change From Baseline in ROM at Bilateral Ankles at Month 24
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in ROM at Bilateral Ankles at Month 30
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in ROM at Bilateral Elbows at Month 6
timeFrame: Baseline (Day 1) and Month 6
measure: Change From Baseline in ROM at Bilateral Elbows at Month 12
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in ROM at Bilateral Elbows at Month 18
timeFrame: Baseline (Day 1) and Month 18
measure: Change From Baseline in ROM at Bilateral Elbows at Month 24
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in ROM at Bilateral Elbows at Month 30
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Percent Predicted Forced Vital Capacity (%pFVC) at Month 12: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in %pFVC at Month 12: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Percent Predicted Forced Expiratory Volume in One Second (%pFEV1) at Month 12: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in %pFEV1 at Month 12: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Maximum Inspiratory Pressure at Month 12: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in Maximum Inspiratory Pressure at Month 24: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in Maximum Inspiratory Pressure at Month 30: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Maximum Expiratory Pressure at Month 12: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in Maximum Expiratory Pressure at Month 24: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in Maximum Expiratory Pressure at Month 30: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 12: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in LVEF at Month 12: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years (Unplanned Analysis)
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Wechsler Intelligence Scale for Children (WISC)-IV Score at Month 24: Ambulatory Participants >= 6 to <=16 Years
timeFrame: Baseline (Day 1) and Month 24
Secondary outcomes:
measure: Number of Participants With Type of DMD Mutation
timeFrame: Up to Month 30
measure: Number of Participants With Each Affected Exon by Mutation Types
timeFrame: Up to Month 30
measure: Number of Participants With DMD Mutations Affecting Any Exon Between Exon 9 and Exon 13 or Deletion That Affects Both Exon 29 and Exon 30
timeFrame: Up to Month 30
measure: Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30
timeFrame: Baseline (Day 1) and Months 6, 12, 18, 24 and 30
measure: Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30
timeFrame: Baseline (Day 1) and Months 6, 12, 18, 24 and 30
measure: Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30
timeFrame: Baseline (Day 1) and Months 6, 12, 18, 24 and 30
measure: Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12
timeFrame: Months 12
measure: Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24
timeFrame: Month 24
measure: Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30
timeFrame: Month 30
measure: Change From Baseline in EQ-5D-3L Visual Analogue Score (VAS) Assessment at Months 12, 24 and 30
timeFrame: Baseline (Day 1) and Months 12, 24 and 30
measure: Change From Baseline in EQ-5D-3L Index Score at Months 12, 24 and 30
timeFrame: Baseline (Day 1) and Months 12, 24, and 30
measure: Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12
timeFrame: Month 12
measure: Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24
timeFrame: Month 24
measure: Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30
timeFrame: Month 30
measure: Change From Baseline in EQ-5D-Y VAS Assessment at Months 12, 24, and 30
timeFrame: Baseline (Day 1) and Months 12, 24, and 30
measure: Change From Baseline in EQ-5D-Y Index Score at Months 12, 24, and 30
timeFrame: Baseline (Day 1) and Months 12, 24, and 30
measure: Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30
timeFrame: Baseline (Day 1) and Months 12, 24, and 30
measure: Change From Baseline in HRU Survey Responses: Number of Nights in Hospital Due to Disease/Medication for Disease at Months 12, 24, and 30
timeFrame: Baseline (Day 1) and Months 12, 24, and 30
measure: Change From Baseline in Out-of-Pocket Money of HRU Survey at Months 12, 24 and 30
timeFrame: Baseline (Day 1) and Months 12, 24, and 30
measure: Change From Baseline in Percent Activity Impairment as Per Work Productivity and Activity Impairment Questionnaire Adapted for Caregiving (WPAI:CG) Caregiver Unchanged at Months 12, 24, and 30
timeFrame: Baseline (Day 1) and Months 12, 24, and 30
measure: Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 12
timeFrame: Baseline (Day 1) and Month 12
measure: Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 24
timeFrame: Baseline (Day 1) and Month 24
measure: Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 30
timeFrame: Baseline (Day 1) and Month 30
measure: Change From Baseline in Percent Overall Work Impairment WPAI: CG Caregiver Unchanged at Months 12, 24, and 30
timeFrame: Baseline (Day 1) and Months 12, 24, and 30
measure: Percent Activity Impairment Score as Per WPAI: CG at Months 12, 24, and 30
timeFrame: Months 12, 24, and 30
measure: Percent Work Time Missed Score as Per WPAI: CG at Month 12
timeFrame: Month 12
measure: Percent Work Time Missed Score as Per WPAI: CG at Month 24
timeFrame: Month 24
measure: Percent Work Time Missed Score as Per WPAI: CG at Month 30
timeFrame: Month 30
measure: Percent Overall Work Impairment Scores as Per WPAI: CG at Months 12, 24, and 30
timeFrame: Months 12, 24, and 30
Other outcomes:
Locations:
city: Chongqing
country: China
facility: Children's Hospital of Chongqing Medical University
state: Chongqing
city: Chongqing
country: China
facility: Children's Hospital of Chongqing Medical University (Liangjiang Branch)
state: Chongqing
city: Fuzhou
country: China
facility: The First Affiliated Hospital Of Fujian Medical University
state: Fujian
city: Beijing
country: China
facility: Peking University First Hospital
city: Beijing
country: China
facility: Beijing Children's Hospital, Capital Medical University
city: Shanghai
country: China
facility: Huashan Hospital, Fudan University
city: Shanghai
country: China
facility: Affiliated children's hospital of fudan university
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: