[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100657269":3},{"organization":4,"outcomesModule":7,"designInfo":30,"detailedDescription":24,"studyPopulation":24,"armGroups":36,"interventions":43,"overallOfficials":48,"centralContacts":56,"locations":24,"responsibleParty":66,"collaborators":24,"id":68,"slug":69,"hasResults":70,"nctId":71,"briefTitle":72,"officialTitle":72,"acronym":24,"eligibilityCriteria":73,"healthyVolunteers":70,"sex":74,"minAge":75,"maxAge":76,"enrollmentInfo":77,"targetDuration":24,"studyType":80,"phases":81,"briefSummary":83,"conditions":84,"keywords":24,"overallStatus":87,"whyStopped":24,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":92,"completionDateStruct":94,"leadSponsor":96,"locationsCount":24},{"fullName":5,"class":6},"Thiogenesis Therapeutics, Inc.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":24},[9],{"measure":10,"description":11,"timeFrame":12},"White blood cell (WBC) cystine","The primary endpoint is the change in white blood cell (WBC) cystine levels following treatment with TTI-0102. The study will evaluate whether a weight-based dose of 60 ± 5 mg\u002Fkg\u002Fday maintains WBC cystine levels below 1 nmol ½ cystine\u002Fmg protein over 24 hours.","Comparison of baseline, Day 1-2, Day 3-4, and treatment end (Day 7)",[14,18,20,22,26,28],{"measure":15,"description":16,"timeFrame":17},"Pharmacokinetic parameter: Cmax","cysteamine in plasma","At specified days\u002Ftimepoints during the treatment period: Day1-2, Day 3-4, Day 7",{"measure":19,"description":16,"timeFrame":17},"Pharmacokinetic parameter: Tmax",{"measure":21,"description":16,"timeFrame":17},"Pharmacokinetic parameter: AUC",{"measure":23,"description":24,"timeFrame":25},"Pharmacodynamic biomarker: lactate in plasma",null,"Baseline (Day 0) to treatment end (Day 7)",{"measure":27,"description":24,"timeFrame":25},"Pharmacodynamic biomarker: pyruvate in whole blood",{"measure":29,"description":24,"timeFrame":25},"Pharmacodynamic biomarker: tryptophan in plasma",{"allocation":31,"interventionModel":32,"interventionModelDescription":24,"primaryPurpose":33,"observationalModel":24,"timePerspective":24,"maskingInfo":34},"NA","SINGLE_GROUP","TREATMENT",{"masking":35,"maskingDescription":24,"whoMasked":24},"NONE",[37],{"label":38,"type":39,"description":40,"interventionNames":41},"TTI-0102 (cysteamine-pantetheine disulfide)","EXPERIMENTAL","Given once daily for 7 days",[42],"Drug: TTI-0102 (cysteamine-pantetheine disulfide)",[44],{"type":45,"name":38,"description":46,"armGroupLabels":47,"otherNames":24},"DRUG","TTI-0102 (cysteamine-pantetheine disulfide) - a prodrug of other FDA-approved forms of cysteamine",[38],[49,53],{"name":50,"affiliation":51,"role":52},"Laurence A Greenbaum, MD, PhD, FAAP","Emory University","PRINCIPAL_INVESTIGATOR",{"name":54,"affiliation":5,"role":55},"Patrice P Rioux, MD, PhD","STUDY_DIRECTOR",[57,62],{"name":58,"role":59,"phone":60,"phoneExt":24,"email":61},"Margo Kamel, PhD - Director of Research Projects","CONTACT","404-712-9923","mkamel@emory.edu",{"name":63,"role":59,"phone":64,"phoneExt":24,"email":65},"Kaiyang Zhu - Clinical Research Coordinator","404-727-2375","kzhu42@emory.edu",{"type":67,"investigatorFullName":24,"investigatorTitle":24,"investigatorAffiliation":24,"oldNameTitle":24,"oldOrganization":24},"SPONSOR","100657269","phase-2-pilot-study-of-tti-0102-in-cystinosis-100657269",false,"NCT07837726","Pilot Study of TTI-0102 in Cystinosis","Inclusion Criteria:\n\n* Adults aged 18-65 years with a confirmed diagnosis of cystinosis based on clinical features and\u002For genetic testing, with or without history of kidney transplantation.\n* Currently treated with a stable dose of Cystagon® or Procysbi® for at least 3 months prior to enrollment.\n* Body weight between 50 kg and 80 kg at screening.\n* Able to attend all required study visits and comply with study procedures, including blood draws over a 24-hour period.\n* Able to provide informed consent in English.\n* The first 3 patients must have WBC cystine levels over the last year no more than 50% greater than the upper limit of target level\n\nExclusion Criteria:\n\n* Known hypersensitivity or allergy to cysteamine, pantetheine, TTI-0102, or any excipients in the study drug.\n* Clinically significant uncontrolled medical conditions (e.g., unstable cardiac, hepatic, or renal disease) that, in the investigator's judgment, would increase risk or interfere with study participation.\n* Participation in another interventional clinical trial within 30 days prior to screening.\n* Any condition that, in the investigator's opinion, would make the participant an unsuitable candidate (e.g., inability to comply with procedures, significant cognitive impairment, active substance use disorder).\n* Any patient who is pregnant, plans to become pregnant or is unwilling to use contraceptive measures during study participation.","ALL","18 Years","65 Years",{"count":78,"type":79},6,"ESTIMATED","INTERVENTIONAL",[82],"PHASE2","White blood cell (WBC) cystine levels serve as the primary therapeutic target for monitoring cysteamine treatment in cystinosis, with a goal of \\\u003C1.9 nmol ½ cystine\u002Fmg protein as measured 5-6 hours post-dose when treated with immediate-release formulation, Cystagon® , or 11-12 hours post-dose when treated with delayed-release formulation, Procysbi®, using UCSD granulocyte assay.\n\nAlthough cysteamine dosing in cystinosis has traditionally been individualized based on WBC cystine levels, the FDA-approved Cystagon® prescribing information now includes an approximate recommended maintenance dose of 1.30 g\u002Fm²\u002Fday. When this dosing strategy is plotted against body weight, it aligns with the dosing regimen of TTI-0102 that was found effective in mitochondrial diseases, demonstrating a linear relationship between dose and weight.\n\nThe main objective of this study is to demonstrate that administration of a single dose of 60 ± 5 mg\u002Fkg\u002Fday of TTI-0102 (\\~26 mg\u002Fkg cysteamine base equivalent) allows maintenance of WBC cystine at \\\u003C1.9 nmol ½ cystine\u002Fmg protein over 24 hours as determined by UCSD granulocyte assay.",[85,86],"Nephropathic Cystinosis","Cystinosis","NOT_YET_RECRUITING","2026-09-17",{"date":90,"type":91},"2026-09-23","ACTUAL",{"date":93,"type":79},"2026-10",{"date":95,"type":79},"2027-03",{"name":5,"class":6}]