<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03760029</url>
  </required_header>
  <id_info>
    <org_study_id>C3391004</org_study_id>
    <nct_id>NCT03760029</nct_id>
  </id_info>
  <brief_title>A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy</brief_title>
  <official_title>A NATURAL HISTORY STUDY IN CHINESE MALE PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Pfizer</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Pfizer</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a multicenter, prospective, single cohort study designed to describe the natural&#xD;
      history of DMD in Chinese male patients. A total of approximately 330 subjects will be&#xD;
      enrolled with the target number of subjects in each group as below:&#xD;
&#xD;
        -  Group 1, Ambulatory subjects aged &lt;6 years, approximately 100 subjects;&#xD;
&#xD;
        -  Group 2, Ambulatory subjects aged &gt;=6 years, approximately 180 subjects;&#xD;
&#xD;
        -  Group 3, Non-ambulatory subjects, approximately 50 subjects. Subjects will visit sites&#xD;
           every 6 months. Each subject will be observed for at least 24 months. All subjects will&#xD;
           remain enrolled until the study completion date, such that some will have data collected&#xD;
           after Month 24. Subjects, who complete Visit 5/Month 24 at least 6 months prior to study&#xD;
           completion, will be asked to complete an additional visit at Month 30.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">July 24, 2019</start_date>
  <completion_date type="Actual">March 21, 2023</completion_date>
  <primary_completion_date type="Actual">March 21, 2023</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Other</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Time to life altering clinical milestones due to DMD disease progression - failure to walk</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Time to life altering clinical milestones due to DMD disease progression - failure to stand</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Time to life altering clinical milestones due to DMD disease progression - failure to self feed</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in timed motor functions for ambulatory subjects - rise from floor (supine to stand)</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in timed motor functions for ambulatory subjects - 10 meter walk/run</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in clinical evaluator determined scales - Northstar Ambulatory Assessment</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
    <description>The Northstar Ambulatory Assessment will be performed in ambulatory children &gt;=3 years old</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in clinical evaluator determined scales - Performance of Upper Limb 2.0</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
    <description>The Performance of Upper Limb 2.0 will be administered in subjects &gt;=10 years old.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in strength of muscle groups - knee extension</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in strength of muscle groups - elbow flexion</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in strength of muscle groups - elbow extension</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in strength of muscle groups - shoulder abduction.</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in pulmonary function tests - Percent predicted Forced Vital Capacity (%pFVC)</measure>
    <time_frame>Change from baseline, 12, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in pulmonary function tests - Forced Expiratory Volume in one Second</measure>
    <time_frame>Change from baseline, 12, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in pulmonary function tests - Maximum Inspiratory Pressure</measure>
    <time_frame>Change from baseline, 12, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in pulmonary function tests - Maximum Expiratory Pressure</measure>
    <time_frame>Change from baseline, 12, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in pulmonary function tests - Peak cough flow</measure>
    <time_frame>Change from baseline, 12, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in range of motion (ROM) at bilateral ankles and elbows</measure>
    <time_frame>Change from baseline, 6, 12, 18, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in left ventricular ejection fraction (LVEF)</measure>
    <time_frame>Change from baseline, 12, 24, 30 Months</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from Baseline in Wechsler Intelligence Scale for Children (WISC) score in ambulatory subjects aged &gt;=6 years to &lt;=16 years</measure>
    <time_frame>Chang from baseline, 24 Months</time_frame>
    <description>The WISC is individually administered intelligence test for children between ages of 6 and 16. It generates a Full Scale Intelligence Quotient.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Proportion of subjects with mutation of large deletion</measure>
    <time_frame>Baseline</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Proportion of subjects with mutation of large duplication</measure>
    <time_frame>Baseline</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Proportion of subjects with mutation of small insertion</measure>
    <time_frame>Baseline</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Proportion of subjects with mutation of small deletion</measure>
    <time_frame>Baseline</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Proportion of subjects with mutation of point mutation</measure>
    <time_frame>Baseline</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Proportion of subjects with mutations involving key high frequency mutation sites</measure>
    <time_frame>Baseline</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) score</measure>
    <time_frame>Baseline, 12, 24, 30 Months</time_frame>
    <description>The Pediatric Outcomes Data Collection Instrument is a patient-reported assessment of musculoskeletal health intended for use in children and adolescents.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from Baseline in EuroQoL 5 Dimension 3 Level (EQ-5D-3L)</measure>
    <time_frame>Baseline, 12, 24, 30 Months</time_frame>
    <description>The EQ-5D-3L is instrument measuring health-related quality of life for patients &gt;=16 years old.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from Baseline in EuroQoL 5 Dimension Youth (EQ-5D-Y)</measure>
    <time_frame>Baseline, 12, 24, 30 Months</time_frame>
    <description>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.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from Baseline in Healthcare Resource Utilization (HRU) score</measure>
    <time_frame>Baseline, 12, 24, 30 Months</time_frame>
    <description>The result collection is based on the HRU questionnaire.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from Baseline in Work Productivity and Activity Impairment Questionnaire adapted for Caregiving (WPAI:CG) impairment scores</measure>
    <time_frame>Baseline, 12, 24, 30 Months</time_frame>
    <description>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.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">312</enrollment>
  <condition>Duchenne Muscular Dystrophy</condition>
  <arm_group>
    <arm_group_label>Study arm</arm_group_label>
    <arm_group_type>Other</arm_group_type>
    <description>All subjects in this study will be observed for 24-30 months.</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Visit frequency</intervention_name>
    <description>All subjects need to visit sites more frequently than in routine clinical practice.</description>
    <arm_group_label>Study arm</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Chinese male patients with any age, diagnosed with DMD. Diagnosis must be confirmed in&#xD;
             subject's medical history and by genetic testing obtained during routine clinical care&#xD;
             for diagnostic purposes as reported from an appropriate regulated laboratory using a&#xD;
             clinically validated genetic test (genetic testing is not provided by the sponsor).&#xD;
&#xD;
          2. Subjects who are &gt;=4 years old must be receiving glucocorticosteroids for a minimum of&#xD;
             6 months prior to signing informed consent. There should be no significant change&#xD;
             (&lt;0.2 mg/kg) in dosage or dose regimen (not related to body weight change) for at&#xD;
             least 3 months immediately prior to signing the informed consent. Subjects who are&#xD;
             aged &gt;4 years will be exempt from this requirement; those not taking GC will be&#xD;
             eligible if the initiation of GC treatment in these subjects is considered&#xD;
             inappropriate in the opinion of Investigators.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Any injury which may impact functional testing. Previous injuries must be fully healed&#xD;
             prior to consenting. Prior lower limb fractures must be fully healed and at least 3&#xD;
             months from injury date.&#xD;
&#xD;
          2. Presence or history of other musculoskeletal or neurologic disease or somatic disorder&#xD;
             not related to DMD including pulmonary, cardiac, and cognitive diseases.&#xD;
&#xD;
          3. Subjects &gt;=4 years old who have not completed the varicella vaccination.&#xD;
&#xD;
          4. Participation in other studies involving investigational drug(s) for a minimum of 90&#xD;
             days prior to signing the informed consent and/or during study participation.&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <gender_based>Yes</gender_based>
    <minimum_age>0 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Pfizer CT.gov Call Center</last_name>
    <role>Study Director</role>
    <affiliation>Pfizer</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's Hospital of Chongqing Medical University</name>
      <address>
        <city>Chongqing</city>
        <state>Chongqing</state>
        <zip>400014</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Chongqing Medical University (Liangjiang Branch)</name>
      <address>
        <city>Chongqing</city>
        <state>Chongqing</state>
        <zip>401122</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The First Affiliated Hospital Of Fujian Medical University</name>
      <address>
        <city>Fuzhou</city>
        <state>Fujian</state>
        <zip>350005</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Peking University First Hospital</name>
      <address>
        <city>Beijing</city>
        <zip>100034</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Beijing Children's Hospital, Capital Medical University</name>
      <address>
        <city>Beijing</city>
        <zip>100045</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Huashan Hospital, Fudan University</name>
      <address>
        <city>Shanghai</city>
        <zip>200040</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Affiliated children's hospital of fudan university</name>
      <address>
        <city>Shanghai</city>
        <zip>201102</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Fudan University</name>
      <address>
        <city>Shanghai</city>
        <zip>201102</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <link>
    <url>https://pmiform.com/clinical-trial-info-request?StudyID=C3391004</url>
    <description>To obtain contact information for a study center near you, click here.</description>
  </link>
  <verification_date>April 2023</verification_date>
  <study_first_submitted>November 15, 2018</study_first_submitted>
  <study_first_submitted_qc>November 28, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">November 30, 2018</study_first_posted>
  <last_update_submitted>April 3, 2023</last_update_submitted>
  <last_update_submitted_qc>April 3, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">April 4, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscular Dystrophies</mesh_term>
    <mesh_term>Muscular Dystrophy, Duchenne</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
    <ipd_description>Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.</ipd_description>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03760029
org study id: C3391004
nct id: NCT03760029
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
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.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Other
masking: None (Open Label)
measure: 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
measure: 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
measure: 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
measure: 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
measure: 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
measure: Change from Baseline in clinical evaluator determined scales - Northstar Ambulatory Assessment
time frame: Change from baseline, 6, 12, 18, 24, 30 Months
description: The Northstar Ambulatory Assessment will be performed in ambulatory children >=3 years old
measure: Change from Baseline in clinical evaluator determined scales - Performance of Upper Limb 2.0
time frame: Change from baseline, 6, 12, 18, 24, 30 Months
description: The Performance of Upper Limb 2.0 will be administered in subjects >=10 years old.
measure: Change from Baseline in strength of muscle groups - knee extension
time frame: Change from baseline, 6, 12, 18, 24, 30 Months
measure: Change from Baseline in strength of muscle groups - elbow flexion
time frame: Change from baseline, 6, 12, 18, 24, 30 Months
measure: Change from Baseline in strength of muscle groups - elbow extension
time frame: Change from baseline, 6, 12, 18, 24, 30 Months
measure: Change from Baseline in strength of muscle groups - shoulder abduction.
time frame: Change from baseline, 6, 12, 18, 24, 30 Months
measure: Change from Baseline in pulmonary function tests - Percent predicted Forced Vital Capacity (%pFVC)
time frame: Change from baseline, 12, 24, 30 Months
measure: Change from Baseline in pulmonary function tests - Forced Expiratory Volume in one Second
time frame: Change from baseline, 12, 24, 30 Months
measure: Change from Baseline in pulmonary function tests - Maximum Inspiratory Pressure
time frame: Change from baseline, 12, 24, 30 Months
measure: Change from Baseline in pulmonary function tests - Maximum Expiratory Pressure
time frame: Change from baseline, 12, 24, 30 Months
measure: Change from Baseline in pulmonary function tests - Peak cough flow
time frame: Change from baseline, 12, 24, 30 Months
measure: Change from Baseline in range of motion (ROM) at bilateral ankles and elbows
time frame: Change from baseline, 6, 12, 18, 24, 30 Months
measure: Change from Baseline in left ventricular ejection fraction (LVEF)
time frame: Change from baseline, 12, 24, 30 Months
measure: 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
description: The WISC is individually administered intelligence test for children between ages of 6 and 16. It generates a Full Scale Intelligence Quotient.
measure: Proportion of subjects with mutation of large deletion
time frame: Baseline
measure: Proportion of subjects with mutation of large duplication
time frame: Baseline
measure: Proportion of subjects with mutation of small insertion
time frame: Baseline
measure: Proportion of subjects with mutation of small deletion
time frame: Baseline
measure: Proportion of subjects with mutation of point mutation
time frame: Baseline
measure: Proportion of subjects with mutations involving key high frequency mutation sites
time frame: Baseline
measure: Change from Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) score
time frame: Baseline, 12, 24, 30 Months
description: The Pediatric Outcomes Data Collection Instrument is a patient-reported assessment of musculoskeletal health intended for use in children and adolescents.
measure: Change from Baseline in EuroQoL 5 Dimension 3 Level (EQ-5D-3L)
time frame: Baseline, 12, 24, 30 Months
description: The EQ-5D-3L is instrument measuring health-related quality of life for patients >=16 years old.
measure: Change from Baseline in EuroQoL 5 Dimension Youth (EQ-5D-Y)
time frame: Baseline, 12, 24, 30 Months
description: 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.
measure: Change from Baseline in Healthcare Resource Utilization (HRU) score
time frame: Baseline, 12, 24, 30 Months
description: The result collection is based on the HRU questionnaire.
measure: Change from Baseline in Work Productivity and Activity Impairment Questionnaire adapted for Caregiving (WPAI:CG) impairment scores
time frame: Baseline, 12, 24, 30 Months
description: 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.
arm group label: Study arm
arm group type: Other
description: All subjects in this study will be observed for 24-30 months.
intervention type: Other
intervention name: Visit frequency
description: All subjects need to visit sites more frequently than in routine clinical practice.
arm group label: Study arm
criteria: 
gender: Male
gender based: Yes
minimum age: 0 Years
maximum age: N/A
healthy volunteers: No
last name: Pfizer CT.gov Call Center
role: Study Director
affiliation: Pfizer
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: China
url: https://pmiform.com/clinical-trial-info-request?StudyID=C3391004
description: To obtain contact information for a study center near you, click here.
responsible party type: Sponsor
mesh term: Muscular Dystrophies
mesh term: Muscular Dystrophy, Duchenne
sharing ipd: No
ipd description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.