<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/NCT04475926</url>
  </required_header>
  <id_info>
    <org_study_id>SRP-LGMD-501-NHS</org_study_id>
    <nct_id>NCT04475926</nct_id>
  </id_info>
  <brief_title>A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice</brief_title>
  <official_title>Journey: A Global, Multicenter, Longitudinal Study of the Natural History of Subjects With Limb Girdle Muscular Dystrophy (LGMD) Type 2E (LGMD2E/R4), Type 2D (LGMD2D/R3), and Type 2C (LGMD2C/R5)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Sarepta Therapeutics, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Sarepta Therapeutics, Inc.</source>
  <brief_summary>
    <textblock>
      This study will follow participants who are screened and confirmed with a genetic diagnosis&#xD;
      of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D&#xD;
      (LGMD2D/R3), or Limb-girdle muscular dystrophy type 2C (LGMD2C/R5). These enrolled&#xD;
      participants will be followed to evaluate mobility and pulmonary function for up to 3 years&#xD;
      after enrollment. Additional participant data will be collected from the time the individual&#xD;
      began experiencing LGMD symptoms to the present.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">April 22, 2021</start_date>
  <completion_date type="Anticipated">December 24, 2025</completion_date>
  <primary_completion_date type="Anticipated">December 24, 2025</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>North Star Assessment for Dysferlinopathy (NSAD) Total Score</measure>
    <time_frame>Baseline up to Month 36</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Time to Rise from the Floor</measure>
    <time_frame>Baseline up to Month 36</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Time of 10-Meter Walk/Run [10MWR]</measure>
    <time_frame>Baseline up to Month 36</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Time to Ascend 4 Steps</measure>
    <time_frame>Baseline up to Month 36</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Dimension of the Performance of the Upper Limb (PUL)</measure>
    <time_frame>Baseline up to Month 36</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Timed Up and Go (TUG)</measure>
    <time_frame>Baseline up to Month 36</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Time of 100-Meter Walk/Run (100MWR)</measure>
    <time_frame>Baseline up to Month 36</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Pulmonary Function Test: Forced Vital Capacity (FVC)</measure>
    <time_frame>Baseline up to Month 36</time_frame>
  </primary_outcome>
  <number_of_groups>3</number_of_groups>
  <enrollment type="Anticipated">90</enrollment>
  <condition>Limb-girdle Muscular Dystrophy</condition>
  <arm_group>
    <arm_group_label>LGMD2E/R4 Cohort</arm_group_label>
    <description>Participants with LGMD2E/R4 will be enrolled in this cohort. Enrollment will be capped according to age as follows: 4 to 7 years age range, 8 to 16 years age range, and ≥17 years age range through the course of the study.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>LGMD2D/R3 Cohort</arm_group_label>
    <description>Participants with LGMD2D/R3 will be enrolled in this cohort. Enrollment will be capped according to age as follows: 4 to 7 years age range, 8 to 16 years age range, and ≥17 years age range through the course of the study.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>LGMD2C/R5 Cohort</arm_group_label>
    <description>Participants with LGMD2C/R5 will be enrolled in this cohort. Enrollment will be capped according to age as follows: 4 to 7 years age range, 8 to 16 years age range, and ≥17 years age range through the course of the study.</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        The population for this study is ambulatory or non-ambulatory participants ≥ 4 years of age&#xD;
        with confirmed genetic diagnosis of LGMD2E/R4, LGMD2D/R3, or LGMD2C/R5.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        - Male or female participant ≥ 4 years of age with confirmed genetic diagnosis of&#xD;
        LGMD2E/R4, LGMD2D/R3, or LGMD2C/R5.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Demonstrates cognitive delay or impairment that could confound motor development, in&#xD;
             the opinion of the Investigator.&#xD;
&#xD;
          -  Has a medical condition, in the opinion of the Investigator, that might compromise&#xD;
             participants ability to comply with study requirements.&#xD;
&#xD;
          -  Is participating in other interventional study(ies) at the time of enrollment in this&#xD;
             study.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>4 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Medical Director</last_name>
    <role>Study Director</role>
    <affiliation>Sarepta Therapeutics, Inc.</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4</last_name>
    <phone>1-888-SAREPTA (1-888-727-3782)</phone>
    <email>SareptAlly@sarepta.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Barrow Neurological Institute</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85013</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <email>SareptAlly@sarepta.com</email>
    </contact>
    <investigator>
      <last_name>Erik Ortega, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Arkansas Children's</name>
      <address>
        <city>Little Rock</city>
        <state>Arkansas</state>
        <zip>72202</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>501-364-1850</phone>
      <email>AVeerapandiyan@uams.edu</email>
    </contact>
    <investigator>
      <last_name>Aravindhan Veerapandiyan, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of California San Diego</name>
      <address>
        <city>La Jolla</city>
        <state>California</state>
        <zip>92037</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>858-966-5819</phone>
      <email>ckonersman@health.ucsd.edu</email>
    </contact>
    <investigator>
      <last_name>Chamindra Konersman, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of California, Davis Health Dept of PM&amp;R</name>
      <address>
        <city>Sacramento</city>
        <state>California</state>
        <zip>95817</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>916-734-5057</phone>
      <email>NMRL@ucdavis.edu</email>
    </contact>
    <investigator>
      <last_name>Craig McDonald, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Anne &amp; Robert H. Lurie Children's Hospital of Chicago</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60611</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>312-227-2201</phone>
    </contact>
    <investigator>
      <last_name>Vamshi Rao, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Nationwide Children's Hospital</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43205</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>614-722-6881</phone>
      <email>Linda.Lowes@nationwidechildrens.org</email>
    </contact>
    <investigator>
      <last_name>Linda Lowes, PT, PhD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Oregon Health and Science University</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97068</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <email>SareptAlly@sarepta.com</email>
    </contact>
    <investigator>
      <last_name>Erika Finanger, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>The Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <email>SareptAlly@sarepta.com</email>
    </contact>
    <investigator>
      <last_name>John Brandsema, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Texas Southwestern Medical Center</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75201</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <email>SareptAlly@sarepta.com</email>
    </contact>
    <investigator>
      <last_name>Kaitlin Batley, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Utah Hospital</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84112</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>801-585-9399</phone>
      <email>Russell.Butterfield@hsc.utah.edu</email>
    </contact>
    <investigator>
      <last_name>Russell Butterfield, MD, PhD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of the King's Daughters</name>
      <address>
        <city>Norfolk</city>
        <state>Virginia</state>
        <zip>23507</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>757-668-5743</phone>
      <email>Melinda.Griggs@chkd.org; Erika.Paradiso@chkd.org</email>
    </contact>
    <investigator>
      <last_name>Crystal Proud, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University Hospital Gent</name>
      <address>
        <city>Gent</city>
        <state>Oost-Vlaanderen</state>
        <zip>9000</zip>
        <country>Belgium</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>+3293324544</phone>
      <email>jan.debleecker@ugent.be</email>
    </contact>
    <investigator>
      <last_name>Jan De Bleecker, MD, PHD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>UZ Leuven</name>
      <address>
        <city>Leuven</city>
        <state>Vlaams Brabant</state>
        <zip>3000</zip>
        <country>Belgium</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>+3216344278</phone>
      <email>kristl.claeys@uzleuven.be</email>
    </contact>
    <investigator>
      <last_name>Kristl Claeys, MD, PHD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Hôpital Universitaire des Enfants Reine Fabiola</name>
      <address>
        <city>Brussels</city>
        <zip>1020</zip>
        <country>Belgium</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <email>SareptAlly@sarepta.com</email>
    </contact>
    <investigator>
      <last_name>Nicolas Deconinck, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Hospital de Clinicas de Porto Alegre (HCPA) - PPDS</name>
      <address>
        <city>Porto Alegre</city>
        <state>Rio Grande Do Sul</state>
        <zip>90035-903</zip>
        <country>Brazil</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <email>SareptAlly@sarepta.com</email>
    </contact>
    <investigator>
      <last_name>Jonas Morales Saute, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Children's Hospital - London Health Science Centre</name>
      <address>
        <city>London</city>
        <state>Ontario</state>
        <zip>N6C 2R5</zip>
        <country>Canada</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>(519) 685-8332</phone>
      <email>Craig.Campbell@lhsc.on.ca</email>
    </contact>
    <investigator>
      <last_name>Craig Campbell, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Zentrum für Kinderheilkunde und Jugendmedizin Uniklinikum Giessen Marburg (UKGM), Standort Giessen</name>
      <address>
        <city>Giessen</city>
        <state>Hessen</state>
        <zip>35392</zip>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>+49 (0)641-985-56843</phone>
      <email>andreas.hahn@paediat.med.uni-giessen.de</email>
    </contact>
    <investigator>
      <last_name>Andreas Hahn, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Essen</name>
      <address>
        <city>Essen</city>
        <state>Nordrhein-Westfalen</state>
        <zip>D-45147</zip>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>+4920172383652</phone>
      <email>andrea.gangfuss@uk-essen.de</email>
    </contact>
    <investigator>
      <last_name>Andrea Gangfuss, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Istituto Giannina Gaslini</name>
      <address>
        <city>Genova</city>
        <state>Liguria</state>
        <zip>16147</zip>
        <country>Italy</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>+3901056362912</phone>
      <email>claudio2246@gmail.com</email>
    </contact>
    <investigator>
      <last_name>Claudio Bruno, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Azienda Ospedale Università Padova</name>
      <address>
        <city>Padova</city>
        <state>Veneto</state>
        <zip>35129</zip>
        <country>Italy</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <email>SareptAlly@sarepta.com</email>
    </contact>
    <investigator>
      <last_name>Elena Pegoraro, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico</name>
      <address>
        <city>Milan</city>
        <zip>20122</zip>
        <country>Italy</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>39-02-55033802</phone>
      <email>giacomo.comi@unimi.it</email>
    </contact>
    <investigator>
      <last_name>Giacomo Comi, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Hospital Sant Joan de Déu Universidad de Barcelona</name>
      <address>
        <city>Barcelona</city>
        <zip>08950</zip>
        <country>Spain</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <email>SareptAlly@sarepta.com</email>
    </contact>
    <investigator>
      <last_name>Ortez Gonzalez, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Karolinska Universitetssjukhuset Solna</name>
      <address>
        <city>Stockholm</city>
        <state>Stockholms Lan</state>
        <zip>17176</zip>
        <country>Sweden</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <email>SareptAlly@sarepta.com</email>
    </contact>
    <investigator>
      <last_name>Thomas Sejersen, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Lokman Hekim Etlik Hastanesi</name>
      <address>
        <city>Ankara</city>
        <zip>06100</zip>
        <country>Turkey</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>90 532 234 12 26</phone>
      <email>htopalog@hacettepe.edu.tr</email>
    </contact>
    <investigator>
      <last_name>Haluk Topaloglu, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>UCL Institute of Child Health &amp; Great Ormond Street Hospital for Children</name>
      <address>
        <city>London</city>
        <zip>WC1N 1EH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>44(0) 20 7905 2872</phone>
      <email>g.baranello@ucl.ac.uk</email>
    </contact>
    <investigator>
      <last_name>Giovanni Baranello, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Institute of Genetic Medicine, International Centre for Life</name>
      <address>
        <city>Newcastle Upon Tyne</city>
        <zip>NE1 3BZ</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <phone>0191 241 8652</phone>
      <email>Jordi.Diaz-Manera@newcastle.ac.uk</email>
    </contact>
    <investigator>
      <last_name>Jordi Diaz-Manera, MD,</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>Belgium</country>
    <country>Brazil</country>
    <country>Canada</country>
    <country>Germany</country>
    <country>Italy</country>
    <country>Spain</country>
    <country>Sweden</country>
    <country>Turkey</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://clinicaltrials.sarepta.com/journeyLGMD</url>
    <description>Click here to access the website, https://clinicaltrials.sarepta.com/journeyLGMD, for additional information for the study.</description>
  </link>
  <verification_date>April 2023</verification_date>
  <study_first_submitted>July 14, 2020</study_first_submitted>
  <study_first_submitted_qc>July 14, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 17, 2020</study_first_posted>
  <last_update_submitted>April 6, 2023</last_update_submitted>
  <last_update_submitted_qc>April 6, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">April 10, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>North Star Assessment for Dysferlinopathy (NSAD)</keyword>
  <keyword>Performance of Upper Limb (PUL)</keyword>
  <keyword>Pulmonary function tests (PFTs)</keyword>
  <keyword>Ambulatory</keyword>
  <keyword>Non-Ambulatory</keyword>
  <keyword>Limb-girdle</keyword>
  <keyword>LGMD</keyword>
  <keyword>sarcoglycanopathy</keyword>
  <keyword>β -sarcoglycan</keyword>
  <keyword>Muscular Dystrophy</keyword>
  <keyword>α -sarcoglycan</keyword>
  <keyword>γ -sarcoglycan</keyword>
  <keyword>LGMD-2D/R3</keyword>
  <keyword>LGMD-2E/R4</keyword>
  <keyword>LGMD-2C/R5</keyword>
  <keyword>Clinical Outcomes Assessment</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscular Dystrophies</mesh_term>
    <mesh_term>Muscular Dystrophies, Limb-Girdle</mesh_term>
    <mesh_term>Sarcoglycanopathies</mesh_term>
  </condition_browse>
  <!-- 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/NCT04475926
org study id: SRP-LGMD-501-NHS
nct id: NCT04475926
lead sponsor: 
This study will follow participants who are screened and confirmed with a genetic diagnosis
      of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D
      (LGMD2D/R3), or Limb-girdle muscular dystrophy type 2C (LGMD2C/R5). These enrolled
      participants will be followed to evaluate mobility and pulmonary function for up to 3 years
      after enrollment. Additional participant data will be collected from the time the individual
      began experiencing LGMD symptoms to the present.
observational model: Cohort
time perspective: Prospective
measure: North Star Assessment for Dysferlinopathy (NSAD) Total Score
time frame: Baseline up to Month 36
measure: Time to Rise from the Floor
time frame: Baseline up to Month 36
measure: Time of 10-Meter Walk/Run [10MWR]
time frame: Baseline up to Month 36
measure: Time to Ascend 4 Steps
time frame: Baseline up to Month 36
measure: Dimension of the Performance of the Upper Limb (PUL)
time frame: Baseline up to Month 36
measure: Timed Up and Go (TUG)
time frame: Baseline up to Month 36
measure: Time of 100-Meter Walk/Run (100MWR)
time frame: Baseline up to Month 36
measure: Pulmonary Function Test: Forced Vital Capacity (FVC)
time frame: Baseline up to Month 36
arm group label: LGMD2E/R4 Cohort
description: Participants with LGMD2E/R4 will be enrolled in this cohort. Enrollment will be capped according to age as follows: 4 to 7 years age range, 8 to 16 years age range, and ≥17 years age range through the course of the study.
arm group label: LGMD2D/R3 Cohort
description: Participants with LGMD2D/R3 will be enrolled in this cohort. Enrollment will be capped according to age as follows: 4 to 7 years age range, 8 to 16 years age range, and ≥17 years age range through the course of the study.
arm group label: LGMD2C/R5 Cohort
description: Participants with LGMD2C/R5 will be enrolled in this cohort. Enrollment will be capped according to age as follows: 4 to 7 years age range, 8 to 16 years age range, and ≥17 years age range through the course of the study.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 4 Years
maximum age: N/A
healthy volunteers: No
last name: Medical Director
role: Study Director
affiliation: Sarepta Therapeutics, Inc.
last name: Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
phone: 1-888-SAREPTA (1-888-727-3782)
email: SareptAlly@sarepta.com
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
country: Belgium
country: Brazil
country: Canada
country: Germany
country: Italy
country: Spain
country: Sweden
country: Turkey
country: United Kingdom
country: United States
url: https://clinicaltrials.sarepta.com/journeyLGMD
description: Click here to access the website, https://clinicaltrials.sarepta.com/journeyLGMD, for additional information for the study.
responsible party type: Sponsor
mesh term: Muscular Dystrophies
mesh term: Muscular Dystrophies, Limb-Girdle
mesh term: Sarcoglycanopathies