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"}}},"trialDetailsJson":{"title":"Details Page","returnSearch":"Back to search results","description":"Study Description","identifier":"ViiV Study ID","clinicalID":"Clinicaltrials.gov Identifier","EudraCtID":"EudraCT Number","resultsAvailable":"Has Results Available Below","docAvailable":"Has Results Document Available","studyOverview":"Study Overview","overviewSection":{"conditionsDisplay":"Medical Conditions","product":"Product","collaborators":"Collaborators","dates":"Date","studyType":"Type","phase":"Phase","gender":"Gender","age":"Age","acceptVolunteers":"Accepts Healthy Volunteers"},"studyDocuments":"Study Documents","noDocuments":"No documents available","studyDocumentsAdditionalText":"If you wish to request for Full Study Report, please contact - ","studyDocumentsAdditionalEmail":"GSKClinicalSupportHD@gsk.com","isReleased":{"firstPart":"This study has Protocol and Result summary on ClinicalTrial.gov. Click","secondPart":"to learn more.","linkText":"here"},"mapLocations":"Locations","centralContact":"Central Contact","centralPhone":"Central Contact Phone","centralEmail":"Central Contact Email","mapSection":{"site":"GSK Investigational Site","condition":"Condition","id":"Study ID","city":"City","state":"State","zip":"Zip","country":"Country"},"studyDesign":"Study Design","designSection":{"purpose":"Primary Purpose","allocation":"Allocation","study_design":"Study Design","study_classification":"Study Classification","masking":"Masking","masked_subject":"Masked Subject","masked_caregiver":"Masked Caregiver","masked_investigator":"Masked Investigator","masked_assessor":"Masked Assessor","primary_outcomes":"Primary Outcomes","secondary_outcomes":"Secondary Outcomes","arms":"Arms","interventions":"Interventions","keyword":"Keyword","inclusionTitle":"Inclusion Criteria","exclusionTitle":"Exclusion Criteria","criteriaText":"Inclusion/Exclusion Criteria","criteria_button":"Click to view inclusion/exclusion criteria","enrollment":"Enrollment","clinicalPublications":"Clinical Publications"},"popoversText":{"studyId":{"childMarkdownRemark":{"html":"A unique number for a study given by ViiV.
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The initial dose of study drug in double-blind period can be reduced once after Week 13 to the next lower weight-band due to safety or tolerability considerations, regardless of any changes in weight. In the open-label extension period, dose adjustments are permitted at the discretion of the investigator.","arm_type":"Experimental"},{"arm_group_label":"Placebo","arm_group_description":"Participants will receive placebo matching deflazacort orally for 26 weeks in the double-blind period, and deflazacort 0.6 mg/kg/day for an additional 26 weeks in the open-label extension period. In the open-label extension period, dose adjustments are permitted at the discretion of the investigator.","arm_type":"Placebo Comparator"}],"assignment":"Parallel Assignment","brief_summary":"This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. The study will include a 26-week double-blind, randomized, placebo-controlled period followed by a 26-week open-label extension period during which all participants will receive deflazacort.","brief_summary_OneLinkTx":"","brief_title_OneLinkTx":"","brief_title":"A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)","conditions":[{"DisplayValue":"Unmapped","DisplayValue_OneLinkTx":"","InternalValue":"CON_UNMAPPED","LookupValue":"Limb-Girdle Muscular Dystrophy","LookupValue_OneLinkTx":""}],"created_date":"06/07/2021 19:16:02","criteria":"Inclusion Criteria:\n\n - Genetic diagnosis of LGMD2I (confirmed mutation in the fukutin-related protein [FKRP] gene).\n - Ability to ascend 4 stairs greater than (>) 2.5 and less than (<) 8 seconds at screening and baseline.\n - Ability to understand the nature of the study and the consent form and to comply with study related procedures.\n - Must weigh between 35 to 112.5 kilograms (kg).\n\nExclusion Criteria:\n\n - Received greater than or equal to (>=) 4 weeks of continuous, systemic corticosteroid therapy within 3 months of study screening visit.\n - Presence of significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction <30 percent [%]) at screening.\n - Requires fulltime ventilator support.\n - History of chronic systemic fungal or viral infections.\n - History of acute bacterial infection (including tuberculosis) per discretion of the Investigator.\n - Diagnosis of diabetes mellitus (controlled and/or uncontrolled) defined as glycated hemoglobin (HbA1c) >=6.5%.\n - History of immunosuppression or other contraindications to glucocorticosteroid therapy.\n - Requires concomitant use or >1 week of drugs or substances that are moderate to strong cytochrome P3A4 (CYP3A4) inhibitors (for example, clarithromycin, fluconazole, diltiazem, verapamil, grapefruit juice) or moderate or strong CYP3A4 inducers (that is, rifampin, efavirenz, carbamazepine, 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arms.","intervention_name":"Deflazacort","intervention_type":"Drug"},{"arm_group_label":"Placebo","intervention_description":"Placebo matching deflazacort will be administered as per schedule specified in the respective arm.","intervention_name":"Placebo","intervention_type":"Drug"}],"interventional_subtype":"Treatment","is_fda_regulated":true,"is_high_value":false,"is_ind_study":false,"keywords":[],"last_follow_up_date":"08/31/2020 00:00:00","links":[],"locations":[{"City":"Baltimore","Latitude":"39.3042055","CountryDisplay":"United States","Longitude":"-76.557493","Name":"Hugo W Moser Research Institute at Kennedy Krieger Institute","StateDisplay":"MD","PostalCode":"21205","StatusDisplay":"Not yet recruiting","StatusDisplayInternal":"LOCSTATUS_NOT_YET_REC","StatusMapped":"Will Be Recruiting"},{"City":"Munich","Latitude":"48.1582691","CountryDisplay":"Germany","Longitude":"11.5786882","Name":"Ludwig-Maximilians University Munich, Friedrich-Baur-Institute","StateDisplay":null,"PostalCode":"80801","StatusDisplay":"Not yet recruiting","StatusDisplayInternal":"LOCSTATUS_NOT_YET_REC","StatusMapped":"Will Be Recruiting"},{"City":"Iowa City","Latitude":"41.659363","CountryDisplay":"United States","Longitude":"-91.5487099","Name":"University of Iowa Hospitals and Clinics","StateDisplay":"IA","PostalCode":"52242","StatusDisplay":"Not yet recruiting","StatusDisplayInternal":"LOCSTATUS_NOT_YET_REC","StatusMapped":"Will Be Recruiting"},{"City":"Kansas City","Latitude":"39.0599844","CountryDisplay":"United States","Longitude":"-94.6099922","Name":"The University of Kansas Medical Center","StateDisplay":"KS","PostalCode":"66160","StatusDisplay":"Not yet recruiting","StatusDisplayInternal":"LOCSTATUS_NOT_YET_REC","StatusMapped":"Will Be Recruiting"},{"City":"Copenhagen","Latitude":"55.6760968","CountryDisplay":"Denmark","Longitude":"12.5683372","Name":"Rigshospitalet, University of Copenhagen","StateDisplay":null,"PostalCode":"2200","StatusDisplay":"Not yet recruiting","StatusDisplayInternal":"LOCSTATUS_NOT_YET_REC","StatusMapped":"Will Be Recruiting"},{"City":"Atlanta","Latitude":"33.81842","CountryDisplay":"United States","Longitude":"-84.3594626","Name":"Rare Disease Research, LLC","StateDisplay":"GA","PostalCode":"30324","StatusDisplay":"Recruiting","StatusDisplayInternal":"LOCSTATUS_RECRUITING","StatusMapped":"Recruiting"},{"City":"Oslo","Latitude":"59.9138688","CountryDisplay":"Norway","Longitude":"10.7522454","Name":"Oslo University Hospital","StateDisplay":null,"PostalCode":"0424","StatusDisplay":"Not yet recruiting","StatusDisplayInternal":"LOCSTATUS_NOT_YET_REC","StatusMapped":"Will Be Recruiting"},{"City":"Ottawa","Latitude":"45.3929604","CountryDisplay":"Canada","Longitude":"-75.72175","Name":"Ottawa Hospital","StateDisplay":null,"PostalCode":"K1Y 4E9","StatusDisplay":"Not yet recruiting","StatusDisplayInternal":"LOCSTATUS_NOT_YET_REC","StatusMapped":"Will Be Recruiting"},{"City":"Moscow","Latitude":"55.8877653","CountryDisplay":"Russia","Longitude":"37.5148752","Name":"Pirogov Russian National Research Medical University","StateDisplay":null,"PostalCode":"125412","StatusDisplay":"Withdrawn","StatusDisplayInternal":"LOCSTATUS_TERMINATED","StatusMapped":"Terminated/Withdrawn"},{"City":"St Louis","Latitude":"38.6212468","CountryDisplay":"United States","Longitude":"-90.2526163","Name":"Washington University School of Medicine","StateDisplay":"MO","PostalCode":"63110","StatusDisplay":"Not yet recruiting","StatusDisplayInternal":"LOCSTATUS_NOT_YET_REC","StatusMapped":"Will Be Recruiting"},{"City":"Saint Petersburg","Latitude":"59.983695","CountryDisplay":"Russia","Longitude":"30.3488021","Name":"Saint-Petersburg State Pediatric Medical University","StateDisplay":null,"PostalCode":"194100","StatusDisplay":"Withdrawn","StatusDisplayInternal":"LOCSTATUS_TERMINATED","StatusMapped":"Terminated/Withdrawn"},{"City":"Marseille","Latitude":"43.296482","CountryDisplay":"France","Longitude":"5.36978","Name":"University Hospital La Timone","StateDisplay":null,"PostalCode":"13385","StatusDisplay":"Not yet recruiting","StatusDisplayInternal":"LOCSTATUS_NOT_YET_REC","StatusMapped":"Will Be Recruiting"},{"City":"Gothenburg","Latitude":"57.70887","CountryDisplay":"Sweden","Longitude":"11.97456","Name":"Sahlgrenska University Hospital","StateDisplay":null,"PostalCode":"41345","StatusDisplay":"Not yet recruiting","StatusDisplayInternal":"LOCSTATUS_NOT_YET_REC","StatusMapped":"Will Be Recruiting"}],"masking":"","maximum_age_raw":"N/A","maximum_age":"N/A","minimum_age":"18","minimum_age_raw":"18 Years","modified_date":"06/15/2021 18:05:49","number_of_arms":"2","observational_design":{"biospecimen_description":"","biospecimen_retention":"","number_of_groups":"","observational_study_design":"","patient_registry":"","target_duration_quantity":"","target_duration_units":"","timing":""},"official_title":"A Multicenter Randomized Placebo-Controlled Phase 3 Study on the Safety and Efficacy of Deflazacort (Emflaza®) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)","org_study_id":"Test_Attachment_Endpoint","overall_contact":{"email":"mharmon@ptcbio.com","last_name":"Harmon","phone":"908-912-9506"},"overall_status":"Recruiting - Interventional","oversight_info":{"exported_from_us":"Yes","fda_regulated_device":"No","fda_regulated_drug":"Yes","ped_postmarket_surv":"","has_dmc":false,"post_prior_to_approval":"","regulatory_authorities":[""]},"phase":"","primary_compl_date":"08/31/2020 00:00:00","primary_outcomes":[{"outcome_description":"","outcome_measure":"Change From Baseline in Time to Climb 4 Stairs at Week 26","outcome_time_frame":"Baseline, Week 26","outcome_safety_issue":null}],"product_name":"Deflazacort","product_name_OneLinkTx":"","secondary_ids":[],"secondary_outcomes":[{"outcome_description":"","outcome_measure":"Change From Baseline in Forced Vital Capacity (FVC) at Week 26","outcome_time_frame":"Baseline, Week 26","outcome_safety_issue":null},{"outcome_description":"","outcome_measure":"Change From Baseline in 2-Minute Walk Test at Week 26","outcome_time_frame":"Baseline, Week 26","outcome_safety_issue":null},{"outcome_description":"","outcome_measure":"Change From Baseline in Time to up and go at Week 26","outcome_time_frame":"Baseline, Week 26","outcome_safety_issue":null},{"outcome_description":"","outcome_measure":"Change From Baseline in Time to Descent 4 Stairs at Week 26","outcome_time_frame":"Baseline, Week 26","outcome_safety_issue":null},{"outcome_description":"","outcome_measure":"Change From Baseline in Time to Run/Walk 10 Meters at Week 26","outcome_time_frame":"Baseline, Week 26","outcome_safety_issue":null},{"outcome_description":"","outcome_measure":"Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) at Week 26","outcome_time_frame":"Baseline, Week 26","outcome_safety_issue":null},{"outcome_description":"","outcome_measure":"Change From Baseline in Hand-Held Myometry at Week 26","outcome_time_frame":"Baseline, Week 26","outcome_safety_issue":null},{"outcome_description":"","outcome_measure":"Number of Participants With Adverse Events","outcome_time_frame":"Baseline to Week 52","outcome_safety_issue":null},{"outcome_description":"","outcome_measure":"Number of Participants With Laboratory Abnormalities","outcome_time_frame":"Baseline to Week 52","outcome_safety_issue":null},{"outcome_description":"","outcome_measure":"Number of Participants With Electrocardiogram (ECG) Abnormalities","outcome_time_frame":"Baseline to Week 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(Lambda z [λz]) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort","outcome_time_frame":"Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13","outcome_safety_issue":null},{"outcome_description":"","outcome_measure":"Half-Life (t1/2) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort","outcome_time_frame":"Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13","outcome_safety_issue":null}],"sponsors":{"investigator_affiliation":"","investigator_username":"","lead_sponsor":"PTC Therapeutics","collaborators":[]},"start_date":"05/15/2019 00:00:00","study_type":"Interventional","therapeutic_area":"","citations":[],"is_section_801":true}},"pageContext":{"studyId":"Test_Attachment_Endpoint"}},"staticQueryHashes":["162689979","3332233851","3729204765","3769354840","399703114"]}