[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100316977":3},{"organization":4,"outcomesModule":7,"designInfo":64,"detailedDescription":76,"studyPopulation":54,"armGroups":77,"interventions":94,"overallOfficials":108,"centralContacts":112,"locations":118,"responsibleParty":234,"collaborators":54,"id":236,"slug":237,"hasResults":238,"nctId":239,"briefTitle":240,"officialTitle":241,"acronym":54,"eligibilityCriteria":242,"healthyVolunteers":238,"sex":243,"minAge":244,"maxAge":245,"enrollmentInfo":246,"targetDuration":54,"studyType":249,"phases":250,"briefSummary":253,"conditions":254,"keywords":54,"overallStatus":195,"whyStopped":54,"lastUpdateSubmitDate":256,"lastUpdatePostDateStruct":257,"startDateStruct":260,"completionDateStruct":262,"leadSponsor":264,"locationsCount":265},{"fullName":5,"class":6},"Travere Therapeutics, Inc.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":29,"otherOutcomes":54},[9,13,16,19,23,26],{"measure":10,"description":11,"timeFrame":12},"Incidence of AEs","Incidence of AEs (by type, severity and relationship to study drug)","• Cohorts 1-6: Through double-blind study completion, approximately 10 months per patient • Cohort 7: First dose through End of Treatment (up to 32 weeks)",{"measure":14,"description":15,"timeFrame":12},"Anti-pegtibatinase antibodies","Presence and levels of anti-pegtibatinase antibodies in plasma as measured by antibody titers",{"measure":17,"description":18,"timeFrame":12},"Anti-PEG antibodies","Presence and levels of anti-PEG antibodies in plasma as measured by antibody titers",{"measure":20,"description":21,"timeFrame":22},"Incidence of hypermethioninemia (Cohort 7 only)","The number and percentage of participants who develop hypermethioninemia during treatment, based on plasma methionine concentrations exceeding the protocol-defined threshold. Participants meeting the protocol-defined threshold may undergo dietary management or study treatment modifications, as appropriate.","First dose through End of Treatment (up to Week 32)",{"measure":24,"description":25,"timeFrame":22},"Incidence of hypomethioninemia (Cohort 7 only)","The number and percentage of participants who develop hypomethioninemia during treatment, based on plasma methionine concentrations below the protocol-defined threshold. Participants meeting the protocol-defined threshold may receive dietary protein supplementation or study treatment modifications, as appropriate.",{"measure":27,"description":28,"timeFrame":22},"The proportion of participants requiring dietary protein rescue (Cohort 7 only)","The proportion of participants who require initiation of dietary protein supplementation during study treatment to manage protocol-defined low plasma methionine concentrations.",[30,33,36,40,43,46,49,52,55,57,60,62],{"measure":31,"description":32,"timeFrame":12},"Changes in pegtibatinase levels","Changes in pegtibatinase levels following single and repeat administration at specified timepoints",{"measure":34,"description":35,"timeFrame":12},"Changes in Met cycle metabolites levels - tHcy","Changes in total homocysteine levels in micromoles",{"measure":37,"description":38,"timeFrame":39},"Changes in Met cycle metabolites levels - total Cys (Cohorts 1-6 Only)","Changes in total cysteine levels in micromoles","Through double-blind study completion, approximately 10 months per patient",{"measure":41,"description":42,"timeFrame":39},"Changes in Met cycle metabolites levels - Me (Cohorts 1-6 Only)","Changes in methionine levels in micromoles",{"measure":44,"description":45,"timeFrame":39},"Changes in Met cycle metabolites levels - Cth (Cohorts 1-6 Only)","Changes in cystathionine levels in micromoles",{"measure":47,"description":48,"timeFrame":39},"Changes in Met cycle metabolites levels - Phe (Cohorts 1-6 Only)","Changes in phenylalanine levels in micromoles",{"measure":50,"description":51,"timeFrame":39},"Descriptive ophthalmology examination findings (Cohorts 1-6 Only)","Comprehensive ophthalmological examination (for each eye: visual acuity \\[myopia, hyperopia, exotropia\\], slit lamp examination \\[ectopic lentis, cataracts, corneal abrasion, and uveitis\\], retinal examination \\[retinal degeneration, retinal detachment, retinitis pigmentosa, uveitis)\\]). Assessment of presence and severity of findings.",{"measure":53,"description":54,"timeFrame":39},"Bone densitometry using dual-energy X-ray absorptionmetry (DEXA) scans (Cohorts 1-6 Only)",null,{"measure":56,"description":54,"timeFrame":39},"Cognitive assessments using the National Institutes of Health Toolbox Cognition Battery score (Cohorts 1-6 Only)",{"measure":58,"description":59,"timeFrame":39},"Patient Reported Outcome (PRO): Quality of Life in Neurological Disorders [Neuro-QoL] (Cohorts 1-6 Only)","The Quality of Life in Neurological Disorders \\[Neuro-QoL\\] includes Anxiety Short Form, Depression Short Form, Satisfaction with Social Roles Short Form, Cognition Function Short Form for 18+ years of age; Anxiety Short Form, Depression Short Form, Social Relations - Interaction with Peers Short Form, and Cognitive Function Short Form for Ages 12 to 17 years old",{"measure":61,"description":54,"timeFrame":39},"Patient Reported Outcome (PRO): Quality of Life by 36-Item Short Form Survey [SF-36] (Cohorts 1-6 Only)",{"measure":63,"description":54,"timeFrame":39},"Patient Reported Outcome (PRO): Quality of Life by EuroQol 5-Dimentional Instrument [EQ 5D] (Cohorts 1-6 Only)",{"allocation":65,"interventionModel":66,"interventionModelDescription":67,"primaryPurpose":68,"observationalModel":54,"timePerspective":54,"maskingInfo":69},"RANDOMIZED","PARALLEL","Cohorts 1-6 (completed) used a randomized, double-blind, placebo-controlled, parallel-group dose-escalation design. Cohort 7 (recruiting) is an open-label, single-arm pediatric cohort with intra-participant sequential dose escalation from 1.0 mg\u002Fkg to 2.5 mg\u002Fkg administered twice weekly. Participants who meet protocol-defined criteria progress to the next dose level. Participants who complete treatment or do not meet dose-escalation criteria may transition to the ENSEMBLE long-term extension study or continue treatment until ENSEMBLE is available at their highest tolerated dose.","TREATMENT",{"masking":70,"maskingDescription":54,"whoMasked":71},"QUADRUPLE",[72,73,74,75],"PARTICIPANT","CARE_PROVIDER","INVESTIGATOR","OUTCOMES_ASSESSOR","Primary Objective - Cohorts 1-6\n\nResearchers are performing this study to learn if pegtibatinase is safe and tolerable (how it makes participants feel).\n\nResearchers will use medical examination, blood tests, and urine tests to measure side effects (unwanted health problems that may or may not be related to the study treatment), changes in laboratory tests and in the electrical activity of the heart (as measured by electrocardiogram or \"ECG\"), and if the body makes antibodies to fight against pegtibatinase. Antibodies are proteins that the body makes that may stop pegtibatinase from working or may cause side effects.\n\nSecondary Objectives - Cohorts 1-6\n\nResearchers also want to learn about:\n\n* What are the levels of pegtibatinase in the body after single and repeated doses?\n* How does pegtibatinase affect levels of certain substances that are produced when the body breaks down and creates homocysteine?\n* What are the effects of pegtibatinase on the eyes, bones, mental health, and cognitive function? Researchers will use blood tests, physical examinations, and questionnaires to answer these questions.\n\nCohort 7 Objectives\n\nGroup 7 is open to children aged 5 to 11 years old (or \"pediatric participants\") who meet the requirements for the study.\n\nResearchers are performing this part of the study with Group 7 to learn if pegtibatinase is safe and tolerable (how it makes participants feel) and if it increases antibody levels when it is given to children with HCU.\n\nResearchers will use blood tests and physical examinations to measure side effects that happen during the study, changes in laboratory tests, electrical activity of the heart, antibody levels, vital signs, and the number of pediatric participants that have too high or too low levels of methionine. Researchers will use questionnaires to measure the number of pediatric participants that need more protein in their diet.\n\nResearchers are performing this part of the study with Group 7 because they also want to learn about:\n\n* What are the levels of pegtibatinase in the pediatric participant's body after single and repeated doses?\n* How does pegtibatinase affect levels of total homocysteine and methionine in the pediatric participant's body? Researchers will use blood tests to answer these questions.\n\nStudy Population and Treatments\n\nThis study will include children and adults from 5 to 65 years old with HCU. Participants in Groups 1 to 6 were aged 12 to 65 years old, and Group 7 participants will be aged 5 to 11 years old. Participants in the study will take their standard of care treatment.\n\nGroups 1 to 6 have completed this study already. For these groups, 24 participants aged 12 to 65 years old who met the requirements of the study were split into 1 of the 6 groups. For each group, 3 participants were chosen to take pegtibatinase for every 1 participant chosen to take an injection that does not have any medicine in it (or \"placebo\"). Participants were assigned to pegtibatinase or placebo by chance, like the flip of a coin. This is called \"randomization\". Neither the researchers nor the participants knew which treatment they were getting until the study was completed. This is known as a \"double-blind\" approach.\n\nGroup 7 is open and will be conducted globally. It will include 10 to 15 pediatric participants aged 5 to 11 years old who meet the requirements of the study. All participants in this group will receive pegtibatinase. All participants will know that this is the treatment that they receive. This is called an \"open label\" approach. The treatment period will be separated into 3 parts: Part A, Part B, and Part C. Each part will test a different dose of pegtibatinase. After each part, pediatric participants who meet specific requirements will move to the next part. If the pediatric participants do not meet specific requirements to move to the next part, they will have the option to join the ENSEMBLE study (NCT06431893) and continue taking that same dose.\n\nPegtibatinase or placebo will be given as an injection under the skin (or \"subcutaneous injection\"). Doses for each group are shown below.\n\n* Group 1: 0.33 mg\u002Fkg pegtibatinase or placebo 1 time a week.\n* Group 2: 0.66 mg\u002Fkg pegtibatinase or placebo 1 time a week.\n* Group 3: 1.0 mg\u002Fkg pegtibatinase or placebo 1 time a week.\n* Group 4: 1.0 mg\u002Fkg pegtibatinase or placebo 2 times a week.\n* Group 5: 1.5 mg\u002Fkg pegtibatinase or placebo 2 times a week.\n* Group 6: 2.5 mg\u002Fkg pegtibatinase or placebo 2 times a week.\n* Group 7:\n* Part A: 1.0 mg\u002Fkg pegtibatinase 2 times a week for 8 weeks;\n* Part B: 1.5 mg\u002Fkg pegtibatinase 2 times a week for 8 weeks;\n* Part C: 2.5 mg\u002Fkg pegtibatinase 2 times a week for 4 weeks.\n\nStudy Duration and Visits\n\nParticipants in Groups 1 to 6 were in the study for up to 158 weeks, including the screening period of up to 8 weeks, a double-blind treatment period of up to 12 weeks, and an extension period of up to 138 weeks. A continuation study called ENSEMBLE was available to participants. Participants were offered to join the ENSEMBLE study before they finished the extension period of the COMPOSE study.\n\nPediatric participants in Group 7 may be in the study for up to 42 weeks, including the screening period of up to 10 weeks, open-label treatment period of up to 20 weeks, and up to 12 weeks of additional treatment at the same dose if the ENSEMBLE study is not yet open.\n\nIf participants in Group 7 meet all the requirements of the study, they will have up to 53 visits to a study center or at home. If the ENSEMBLE study is not yet open at their site, they can have up to 23 more visits to continue treatment.\n\nThese visits can include:\n\n* Blood and urine tests\n* Physical examinations\n* Questionnaires\n* Injections\n* Questions about how they are feeling or any problems they are having\n\nSafety \u002F Adverse Events\n\nResearchers will keep track of any medical problems that a participant has during a study (or \"adverse event\"). All participants in this study will have regular laboratory tests, health checkups, and site visits to watch for health risks and measure safety.\n\nBenefit-Risk Conclusion Researchers have worked to reduce risks to participants in this study. They believe the risks of taking pegtibatinase in this study are justified by the potential benefits that they think pegtibatinase may have for people with HCU.",[78,84,89],{"label":79,"type":80,"description":81,"interventionNames":82},"Pegtibatinase (Cohort 1-6)","ACTIVE_COMPARATOR","Double-Blind Treatment Cohorts (≥12 to ≤65 years)",[83],"Drug: Pegtibatinase",{"label":85,"type":86,"description":81,"interventionNames":87},"Placebo (Cohort 1-6)","PLACEBO_COMPARATOR",[88],"Drug: Placebo",{"label":90,"type":91,"description":92,"interventionNames":93},"Pegtibatinase (Cohort 7)","EXPERIMENTAL","Pediatric Open-label Treatment Cohort (≥5 to \\\u003C12 years)",[83],[95,104],{"type":96,"name":97,"description":98,"armGroupLabels":99,"otherNames":100},"DRUG","Pegtibatinase","Pegtibatinase sterile solution for subcutaneous injection",[79,90],[101,102,103],"TVT-058","OT-58","PEG modified CBS, PEG htCBS C15S, htCBS C15S ME-200GS",{"type":96,"name":105,"description":106,"armGroupLabels":107,"otherNames":54},"Placebo","Normal saline for subcutaneous injection",[85],[109],{"name":110,"affiliation":5,"role":111},"Michael Imperiale, MD","STUDY_DIRECTOR",[113],{"name":114,"role":115,"phone":116,"phoneExt":54,"email":117},"Travere Call Center","CONTACT","1-877-659-5518","medinfo@travere.com",[119,133,142,153,162,171,180,189,193,204,213,224],{"facility":120,"status":121,"city":122,"state":123,"zip":124,"country":125,"countryCode":126,"cosmosGeoPoint":127,"geoPoint":132,"contacts":54},"Travere Investigational Site","COMPLETED","Aurora","Colorado","80045","United States","US",{"type":128,"coordinates":129},"Point",[130,131],-104.83192,39.72943,{"lat":131,"lon":130},{"facility":120,"status":121,"city":134,"state":135,"zip":136,"country":125,"countryCode":126,"cosmosGeoPoint":137,"geoPoint":141,"contacts":54},"Miami","Florida","33136",{"type":128,"coordinates":138},[139,140],-80.19366,25.77427,{"lat":140,"lon":139},{"facility":143,"status":144,"city":145,"state":146,"zip":147,"country":125,"countryCode":126,"cosmosGeoPoint":148,"geoPoint":152,"contacts":54},"Ann & Robert H. Lurie Children's Hospital of Chicago","NOT_YET_RECRUITING","Chicago","Illinois","60611",{"type":128,"coordinates":149},[150,151],-87.65005,41.85003,{"lat":151,"lon":150},{"facility":120,"status":121,"city":154,"state":155,"zip":156,"country":125,"countryCode":126,"cosmosGeoPoint":157,"geoPoint":161,"contacts":54},"Indianapolis","Indiana","46202",{"type":128,"coordinates":158},[159,160],-86.15804,39.76838,{"lat":160,"lon":159},{"facility":120,"status":121,"city":163,"state":164,"zip":165,"country":125,"countryCode":126,"cosmosGeoPoint":166,"geoPoint":170,"contacts":54},"Portland","Maine","04102",{"type":128,"coordinates":167},[168,169],-70.2589,43.65737,{"lat":169,"lon":168},{"facility":120,"status":121,"city":172,"state":173,"zip":174,"country":125,"countryCode":126,"cosmosGeoPoint":175,"geoPoint":179,"contacts":54},"Boston","Massachusetts","02115",{"type":128,"coordinates":176},[177,178],-71.05977,42.35843,{"lat":178,"lon":177},{"facility":181,"status":144,"city":182,"state":182,"zip":183,"country":125,"countryCode":126,"cosmosGeoPoint":184,"geoPoint":188,"contacts":54},"The Mount Sinai Hospital","New York","10029",{"type":128,"coordinates":185},[186,187],-74.00597,40.71427,{"lat":187,"lon":186},{"facility":120,"status":121,"city":182,"state":182,"zip":183,"country":125,"countryCode":126,"cosmosGeoPoint":190,"geoPoint":192,"contacts":54},{"type":128,"coordinates":191},[186,187],{"lat":187,"lon":186},{"facility":194,"status":195,"city":196,"state":197,"zip":198,"country":125,"countryCode":126,"cosmosGeoPoint":199,"geoPoint":203,"contacts":54},"Science 37 - Virtual Site","RECRUITING","Morrisville","North Carolina","27560",{"type":128,"coordinates":200},[201,202],-78.82556,35.82348,{"lat":202,"lon":201},{"facility":120,"status":121,"city":205,"state":206,"zip":207,"country":125,"countryCode":126,"cosmosGeoPoint":208,"geoPoint":212,"contacts":54},"Philadelphia","Pennsylvania","19104",{"type":128,"coordinates":209},[210,211],-75.16362,39.95238,{"lat":211,"lon":210},{"facility":214,"status":144,"city":215,"state":54,"zip":216,"country":217,"countryCode":218,"cosmosGeoPoint":219,"geoPoint":223,"contacts":54},"Hospital Necker-Enfants Malades, Neurologie Pediatrique","Paris","75015","France","FR",{"type":128,"coordinates":220},[221,222],2.3488,48.85341,{"lat":222,"lon":221},{"facility":225,"status":144,"city":226,"state":54,"zip":54,"country":227,"countryCode":228,"cosmosGeoPoint":229,"geoPoint":233,"contacts":54},"Sidra Medicine","Doha","Qatar","QA",{"type":128,"coordinates":230},[231,232],51.53096,25.28545,{"lat":232,"lon":231},{"type":235,"investigatorFullName":54,"investigatorTitle":54,"investigatorAffiliation":54,"oldNameTitle":54,"oldOrganization":54},"SPONSOR","100316977","pegtibatinase-as-a-treatment-for-patients-with-classical-homocystinuria-hcu-also-known-as-the-compose-study-100316977",false,"NCT03406611","Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)","A Phase 1\u002F2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effects on Clinical Outcomes of Pegtibatinase (TVT-058) Administered Subcutaneously in Subjects With Cystathionine Beta Synthase-Deficient Homocystinuria (COMPOSE)","Inclusion Criteria:\n\n* Age\n\n  * Cohort 7 (currently enrolling): ≥5 to \\\u003C12 years of age.\n  * Completed Cohorts 1-6: ≥12 to 65 years of age.\n* Diagnosis of classical homocystinuria (HCU)\n\n  * Cohort 7 (currently enrolling): Diagnosis based on clinical, biochemical, and\u002For molecular genetic testing.\n  * Completed Cohorts 1-6: Genetically confirmed cystathionine beta-synthase (CBS)-deficient HCU.\n* Plasma total homocysteine (tHcy)\n\n  * Cohort 7 (currently enrolling): Plasma tHcy ≥50 μM at Screening.\n  * Completed Cohorts 1-6: Plasma tHcy ≥50 μM at Screening and documented historical plasma tHcy ≥80 μM.\n* Willing and able (or parent\u002Flegal guardian willing and able) to provide informed consent\u002Fassent and comply with study procedures.\n* Willing to maintain a generally stable standard-of-care treatment regimen, including dietary management and HCU-related therapies, unless changes are medically necessary.\n* Participants of childbearing potential must have a negative pregnancy test before study treatment and agree to use protocol-specified contraception, if applicable.\n\nExclusion Criteria:\n\nCohort 7 only:\n\n* Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or a disorder of cobalamin metabolism.\n* History of a major thrombotic event within the previous 6 months.\n* Body weight \\\u003C15 kg.\n\nAll Cohorts:\n\n* Previous treatment with pegtibatinase or pegtarviliase)\n* Participation in a pegtibatinase clinical study.\n* Receipt of another investigational drug or investigational medical device within 30 days before Screening or planned use during study participation.\n* Use of injectable polyethylene glycol (PEG)-containing medications (other than pegtibatinase or PEG-containing vaccines) within 3 months before Screening or during study participation.\n* Known hypersensitivity to pegtibatinase or a history of severe hypersensitivity to a PEG-containing product.\n* Active HIV, hepatitis B, or hepatitis C infection.\n* History of organ transplantation or immunosuppressive therapy.\n* Clinically significant medical conditions that could interfere with study participation or participant safety.\n* Pregnant or breastfeeding, or planning to become pregnant during study participation.\n* Major surgery planned during the study period.\n* Any condition that could prevent the participant from complying with study procedures or completing the study.","ALL","5 Years","65 Years",{"count":247,"type":248},39,"ESTIMATED","INTERVENTIONAL",[251,252],"PHASE1","PHASE2","Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or \"genetic condition\"). It is caused by changes in the cystathionine beta-synthase (or \"CBS\") gene and prevents an enzyme from working correctly in the body. This enzyme breaks down a substance called homocysteine (from dietary methionine found in protein) and keeps both homocysteine and methionine at normal levels. When this enzyme is not working, homocysteine and methionine build up in the blood, which spreads into different tissues of the body and stops these body tissues from working normally.\n\nPeople with HCU can experience problems with vision, bones, blood vessels, and cognitive function (the ability to think, learn, and remember). Treatments available for HCU, such as a low protein diet and betaine (Cystadane®), help reduce homocysteine levels. The diet is a low methionine diet and a methionine-free protein supplement (a product that provides extra protein to help meet daily protein needs). These treatments are either not sufficient or are hard to take for many patients.\n\nPegtibatinase was developed by scientists to be a version of the CBS enzyme that can be given to people with HCU. Researchers believe that giving pegtibatinase to people with HCU already getting medical treatment (or \"standard of care\") may reduce their homocysteine levels.\n\nThis study is split into 7 different groups getting different amounts of drug. The first 6 groups have already finished the study.\n\nGroup 7 plans to enroll participants from the US (virtual and in-person), France, and Qatar.",[255],"Homocystinuria","2026-08-05",{"date":258,"type":259},"2026-08-10","ACTUAL",{"date":261,"type":259},"2019-01-22",{"date":263,"type":248},"2027-07",{"name":5,"class":6},12]