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

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

key information

study id #: NCT03760029

condition: Duchenne Muscular Dystrophy

status: not yet recruiting

purpose:

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:<br>

• Group 1, subjects aged <6 years, 100 subjects; <br>

• Group 2, subjects aged >=6 years and <12 years, 180 subjects; <br>

• Group 3, subjects aged >=12 years, 50 subjects. <br>

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.

intervention: Visit frequency

mechanism of action: No pharmaceutical intervention

results: https://clinicaltrials.gov/ct2/show/results/NCT03760029

last updated: January 16, 2019

study details

start date: March 2019

estimated completion: August 2021

phase of development: N/A

size / enrollment: 330

primary outcomes:

  • Time to life altering clinical milestones due to DMD disease progression - failure to walk [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Time to life altering clinical milestones due to DMD disease progression - failure to stand [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Time to life altering clinical milestones due to DMD disease progression - failure to self feed [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in timed motor functions for ambulatory subjects - 4 stair climb [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in timed motor functions for ambulatory subjects - rise from floor (supine to stand) [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in timed motor functions for ambulatory subjects - 10 meter walk/run [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in timed motor functions for ambulatory subjects - 6 minute walk test [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in clinical evaluator determined scales - Expanded Hammersmith Functional Motor Scale [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in clinical evaluator determined scales - Northstar Ambulatory Assessment [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
    The Northstar Ambulatory Assessment will be performed in ambulatory children >=3 years old
  • Change from Baseline in clinical evaluator determined scales - Performance of Upper Limb [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
    The Performance of Upper Limb will be administered in subjects >=5 years old.
  • Change from Baseline in strength of muscle groups - knee extension [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in strength of muscle groups - elbow flexion [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in strength of muscle groups - elbow extension [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in strength of muscle groups - shoulder abduction. [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in pulmonary function tests - Forced Vital Capacity [ Time Frame: Change from baseline, 12, 24, 30 Months ]
  • Change from Baseline in pulmonary function tests - Forced Expiratory Volume in one Second [ Time Frame: Change from baseline, 12, 24, 30 Months ]
  • Change from Baseline in pulmonary function tests - Maximum Inspiratory Pressure [ Time Frame: Change from baseline, 12, 24, 30 Months ]
  • Change from Baseline in pulmonary function tests - Maximum Expiratory Pressure [ Time Frame: Change from baseline, 12, 24, 30 Months ]
  • Change from Baseline in pulmonary function tests - Peak cough flow [ Time Frame: Change from baseline, 12, 24, 30 Months ]
  • Change from Baseline in range of motion (ROM) at bilateral ankles and elbows [ Time Frame: Change from baseline, 6, 12, 18, 24, 30 Months ]
  • Change from Baseline in left ventricular ejection fraction (LVEF) [ Time Frame: Change from baseline, 12, 24, 30 Months ]
  • Change from Baseline in Wechsler Intelligence Scale for Children (WISC) score in ambulatory subjects aged >=6 years to <=16 years [ Time Frame: Chang from baseline, 24 Months ]
    The WISC is individually administered intelligence test for children between ages of 6 and 16. It generates a Full Scale Intelligence Quotient.

secondary outcomes:

  • Proportion of subjects with mutation of large deletion [ Time Frame: Baseline ]
  • Proportion of subjects with mutation of large duplication [ Time Frame: Baseline ]
  • Proportion of subjects with mutation of small insertion [ Time Frame: Baseline ]
  • Proportion of subjects with mutation of small deletion [ Time Frame: Baseline ]
  • Proportion of subjects with mutation of point mutation [ Time Frame: Baseline ]
  • Proportion of subjects with mutations involving key high frequency mutation sites [ Time Frame: Baseline ]
  • Change from Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) score [ Time Frame: Baseline, 12, 24, 30 Months ]
    The Pediatric Outcomes Data Collection Instrument is a patient-reported assessment of musculoskeletal health intended for use in children and adolescents.
  • Change from Baseline in EuroQoL 5 Dimension 3 Level (EQ-5D-3L) [ Time Frame: Baseline, 12, 24, 30 Months ]
    The EQ-5D-3L is instrument measuring health-related quality of life for patients >=16 years old.
  • Change from Baseline in EuroQoL 5 Dimension Youth (EQ-5D-Y) [ Time Frame: Baseline, 12, 24, 30 Months ]
    EQ-5D-Y is a generic instrument measuring health-related quality of life in children and adolescents younger than 16 years old.
  • Change from Baseline in Healthcare Resource Utilization (HRU) score [ Time Frame: Baseline, 12, 24, 30 Months ]
    The result collection is based on the HRU questionnaire.
  • Change from Baseline in Work Productivity and Activity Impairment Questionnaire adapted for Caregiving (WPAI:CG) impairment scores [ Time Frame: Baseline, 12, 24, 30 Months ]
    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.

inclusion criteria:
• 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).
• 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:
• 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.
• Presence or history of other musculoskeletal or neurologic disease or somatic disorder not related to DMD including pulmonary, cardiac, and cognitive diseases.
• Subjects >=4 years old who have not completed the recommended national immunization schedule and the varicella vaccination.
• 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.

study contacts

sponsor: Pfizer

contacts: Pfizer CT.gov Call Center, 1-800-718-1021, ClinicalTrials.gov_Inquiries@pfizer.com

investigators: Pfizer CT.gov Call Center