Nctid:
NCT06617429
Payload:
{"hasResults"=>false, "derivedSection"=>{"miscInfoModule"=>{"versionHolder"=>"2024-10-04"}, "conditionBrowseModule"=>{"meshes"=>[{"id"=>"D000017204", "term"=>"Angelman Syndrome"}, {"id"=>"D000013577", "term"=>"Syndrome"}], "ancestors"=>[{"id"=>"D000004194", "term"=>"Disease"}, {"id"=>"D000010335", "term"=>"Pathologic Processes"}, {"id"=>"D000009069", "term"=>"Movement Disorders"}, {"id"=>"D000002493", "term"=>"Central Nervous System Diseases"}, {"id"=>"D000009422", "term"=>"Nervous System Diseases"}, {"id"=>"D000000015", "term"=>"Abnormalities, Multiple"}, {"id"=>"D000000013", "term"=>"Congenital Abnormalities"}, {"id"=>"D000025063", "term"=>"Chromosome Disorders"}, {"id"=>"D000030342", "term"=>"Genetic Diseases, Inborn"}, {"id"=>"D000096803", "term"=>"Imprinting Disorders"}], "browseLeaves"=>[{"id"=>"M16355", "name"=>"Syndrome", "asFound"=>"Syndrome", "relevance"=>"HIGH"}, {"id"=>"M19508", "name"=>"Angelman Syndrome", "asFound"=>"Angelman Syndrome", "relevance"=>"HIGH"}, {"id"=>"M12029", "name"=>"Movement Disorders", "relevance"=>"LOW"}, {"id"=>"M5742", "name"=>"Central Nervous System Diseases", "relevance"=>"LOW"}, {"id"=>"M12", "name"=>"Congenital Abnormalities", "relevance"=>"LOW"}, {"id"=>"M14", "name"=>"Abnormalities, Multiple", "relevance"=>"LOW"}, {"id"=>"M23023", "name"=>"Chromosome Disorders", "relevance"=>"LOW"}, {"id"=>"M23686", "name"=>"Genetic Diseases, Inborn", "relevance"=>"LOW"}, {"id"=>"T389", "name"=>"Angelman Syndrome", "asFound"=>"Angelman Syndrome", "relevance"=>"HIGH"}], "browseBranches"=>[{"name"=>"Symptoms and General Pathology", "abbrev"=>"BC23"}, {"name"=>"All Conditions", "abbrev"=>"All"}, {"name"=>"Nervous System Diseases", "abbrev"=>"BC10"}, {"name"=>"Diseases and Abnormalities at or Before Birth", "abbrev"=>"BC16"}, {"name"=>"Rare Diseases", "abbrev"=>"Rare"}]}}, "protocolSection"=>{"designModule"=>{"phases"=>["PHASE3"], "studyType"=>"INTERVENTIONAL", "designInfo"=>{"allocation"=>"RANDOMIZED", "maskingInfo"=>{"masking"=>"QUADRUPLE", "whoMasked"=>["PARTICIPANT", "CARE_PROVIDER", "INVESTIGATOR", "OUTCOMES_ASSESSOR"]}, "primaryPurpose"=>"TREATMENT", "interventionModel"=>"PARALLEL"}, "enrollmentInfo"=>{"type"=>"ESTIMATED", "count"=>120}}, "statusModule"=>{"overallStatus"=>"NOT_YET_RECRUITING", "startDateStruct"=>{"date"=>"2024-12", "type"=>"ESTIMATED"}, "expandedAccessInfo"=>{"hasExpandedAccess"=>false}, "statusVerifiedDate"=>"2024-09", "completionDateStruct"=>{"date"=>"2027-11", "type"=>"ESTIMATED"}, "lastUpdateSubmitDate"=>"2024-09-24", "studyFirstSubmitDate"=>"2024-09-24", "studyFirstSubmitQcDate"=>"2024-09-24", "lastUpdatePostDateStruct"=>{"date"=>"2024-09-27", "type"=>"ACTUAL"}, "studyFirstPostDateStruct"=>{"date"=>"2024-09-27", "type"=>"ACTUAL"}, "primaryCompletionDateStruct"=>{"date"=>"2026-07", "type"=>"ESTIMATED"}}, "outcomesModule"=>{"primaryOutcomes"=>[{"measure"=>"Change from Baseline in Bayley-4 Cognitive Raw Score Without Caregiver Input at Day 338", "timeFrame"=>"Baseline, Day 338"}], "secondaryOutcomes"=>[{"measure"=>"Net Response in Multidomain Responder Index (MDRI)", "timeFrame"=>"Day 338"}, {"measure"=>"Change from Baseline in ABC-C Hyperactivity/Noncompliance Subscale Score at Day 338", "timeFrame"=>"Baseline, Day 338"}, {"measure"=>"Change from Baseline in Bayley-4 Receptive Communication Raw Score at Day 338", "timeFrame"=>"Baseline, Day 338"}, {"measure"=>"Change from Baseline in ORCA Total Mastery Communication T-Score at Day 338", "timeFrame"=>"Baseline, Day 338"}, {"measure"=>"Change from Baseline in ASA Sleep Rating Raw Score at Day 338", "timeFrame"=>"Baseline, Day 338"}, {"measure"=>"Number of Participants with Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs), Severity of AEs and Relationship to Investigational Drug, Procedure, and Premedication", "timeFrame"=>"2 Years"}]}, "oversightModule"=>{"isFdaRegulatedDrug"=>true, "isFdaRegulatedDevice"=>false}, "conditionsModule"=>{"conditions"=>["Angelman Syndrome"]}, "referencesModule"=>{"seeAlsoLinks"=>[{"url"=>"https://www.ultrarareadvocacy.com/conditions-we-study/angelman-syndrome-as/", "label"=>"Ultragenyx Patient Advocacy Website"}]}, "descriptionModule"=>{"briefSummary"=>"The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with Angelman Syndrome (AS)."}, "eligibilityModule"=>{"sex"=>"ALL", "stdAges"=>["CHILD"], "maximumAge"=>"17 years", "minimumAge"=>"4 years", "healthyVolunteers"=>false, "eligibilityCriteria"=>"Inclusion Criteria:\n\n* Signed informed consent from parent(s) or legal guardian(s)\n* Confirmed diagnosis of AS with genetic confirmation of full maternal ubiquitin-protein ligase E3A (UBE3A) gene deletion causing AS in the region of 15q11.2 q13\n* Able to ambulate independently, or with assistance at the Screening Visit (note, a child whose primary means of mobility is by wheelchair is excluded from the study)\n* Platelet count, prothrombin time / international normalized ratio, and partial thromboplastin time within 1.5x the normal limits at the Screening Visit\n* Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, and all study procedures, including LP procedure and tolerating anesthesia without intubation\n* From the time of informed consent through to at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102\n\nExclusion Criteria:\n\n* Any change in medications or diet/supplements intended to treat symptoms of AS (eg, sleeping aids, antiseizure medications, supplements, dietary change including ketogenic or low-glycemic index diet, other) within the month prior to the Screening Visit (excluding weight-based adjustments)\n* Any condition that creates an increased risk of unsuccessful LP\n* Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors)\n* Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects\n* Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results\n* Any clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition or infection that, in the judgment of the Investigator, will pose a safety risk, make the subject unsuitable for participation in, and/or unable to complete the study procedures\n* Any laboratory abnormality, that, in the Investigator's opinion, could adversely affect the safety of the subject, make it unlikely that the course of treatment or follow up would be completed, or impair the assessment of study result\n* Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study\n* Use of any investigational product or investigational medical device within 6 months or 5 half-lives prior to the Screening Visit or any prior use of gene therapy or ASO regardless of duration since last administration\n* Concurrent participation in any study, including observational natural history studies"}, "identificationModule"=>{"nctId"=>"NCT06617429", "acronym"=>"Aspire", "briefTitle"=>"Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS", "organization"=>{"class"=>"INDUSTRY", "fullName"=>"Ultragenyx Pharmaceutical Inc"}, "officialTitle"=>"A Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects With Angelman Syndrome", "orgStudyIdInfo"=>{"id"=>"GTX-102-CL301"}, "secondaryIdInfos"=>[{"id"=>"2024-512600-19-00", "type"=>"CTIS"}]}, "armsInterventionsModule"=>{"armGroups"=>[{"type"=>"EXPERIMENTAL", "label"=>"GTX-102", "description"=>"Participants will receive GTX-102 via lumbar puncture (LP) during both the double-blind and open-label period", "interventionNames"=>["Drug: GTX-102"]}, {"type"=>"SHAM_COMPARATOR", "label"=>"Sham-LP then GTX-102", "description"=>"Participants will receive sham procedure during the double-blind period and then will receive GTX-102 via LP during the open-label period", "interventionNames"=>["Drug: GTX-102", "Procedure: Sham-LP"]}], "interventions"=>[{"name"=>"GTX-102", "type"=>"DRUG", "description"=>"antisense oligonucleotide", "armGroupLabels"=>["GTX-102", "Sham-LP then GTX-102"]}, {"name"=>"Sham-LP", "type"=>"PROCEDURE", "description"=>"Small needle prick on the lower back at the location where the LP injection is normally made", "armGroupLabels"=>["Sham-LP then GTX-102"]}]}, "contactsLocationsModule"=>{"centralContacts"=>[{"name"=>"Patients Contact: Trial Recruitment", "role"=>"CONTACT", "email"=>"trialrecruitment@ultragenyx.com", "phone"=>"1-888-756-8657"}, {"name"=>"HCPs Contact: Medical Information", "role"=>"CONTACT", "email"=>"medinfo@ultragenyx.com", "phone"=>"1-888-756-8657"}], "overallOfficials"=>[{"name"=>"Medical Director", "role"=>"STUDY_DIRECTOR", "affiliation"=>"Ultragenyx Pharmaceuticals Inc."}]}, "ipdSharingStatementModule"=>{"ipdSharing"=>"NO"}, "sponsorCollaboratorsModule"=>{"leadSponsor"=>{"name"=>"Ultragenyx Pharmaceutical Inc", "class"=>"INDUSTRY"}, "responsibleParty"=>{"type"=>"SPONSOR"}}}}
