[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100655993":3},{"organization":4,"outcomesModule":7,"designInfo":58,"detailedDescription":69,"studyPopulation":57,"armGroups":70,"interventions":97,"overallOfficials":104,"centralContacts":108,"locations":113,"responsibleParty":142,"collaborators":57,"id":144,"slug":145,"hasResults":146,"nctId":147,"briefTitle":148,"officialTitle":149,"acronym":150,"eligibilityCriteria":151,"healthyVolunteers":146,"sex":152,"minAge":153,"maxAge":57,"enrollmentInfo":154,"targetDuration":57,"studyType":157,"phases":158,"briefSummary":161,"conditions":162,"keywords":166,"overallStatus":116,"whyStopped":57,"lastUpdateSubmitDate":171,"lastUpdatePostDateStruct":172,"startDateStruct":175,"completionDateStruct":177,"leadSponsor":179,"locationsCount":180},{"fullName":5,"class":6},"Portal Therapeutics, Inc.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":21,"otherOutcomes":57},[9,13,17],{"measure":10,"description":11,"timeFrame":12},"Plasma metal-free PPIX concentration change","Characterize the dose-response of PORT-77 on plasma PPIX levels compared to placebo","Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12",{"measure":14,"description":15,"timeFrame":16},"Adverse Events","Evaluate safety and tolerability of each dose of PORT-77 over the course of the study","Part 1 - Day 1 through Day 84; Part 2 - through Day 182; Part 3 - through Month 12",{"measure":18,"description":19,"timeFrame":20},"Average daily time in sunlight without pain","Assess the effect of different doses of PORT-77 on average daily time in sunlight without pain compared to placebo","Part 2 - Day 155 through 182",[22,24,28,31,35,38,41,44,47,51,54],{"measure":18,"description":19,"timeFrame":23},"Part 1 - Days 57 through 84",{"measure":25,"description":26,"timeFrame":27},"Change from baseline in daily time in sunlight before first prodromal symptom","Assess the effect of different doses of PORT-77 on average daily time in sunlight before first prodromal symptom compared to placebo","Part 1 - Day 1 through Day 84; Part 2 - through Day 182",{"measure":29,"description":30,"timeFrame":12},"Cumulative total time in sunlight without pain","Assess the effect of different doses of PORT-77 on cumulative total time in sunlight without pain compared to placebo",{"measure":32,"description":33,"timeFrame":34},"Number and severity of phototoxic reactions","Assess the effect of different doses of PORT-77 on the rate and severity of phototoxic reactions compared to placebo","Part 1 - Day 1 through 84; Part 2 - through Day 182; through Month 12",{"measure":36,"description":37,"timeFrame":12},"Change from baseline in validated QoL scores - PGI-C","Evaluate the effect of PORT-77 on the Patient Global Impressions - Change in clinical status (PGI-C) in adults compared to placebo.\n\nThe PGI-C measures Change in clinical status. Higher numerical scores (1-7) in PGI-C indicate worsening of clinical status.",{"measure":39,"description":40,"timeFrame":12},"Change from baseline in validated QoL scores - PGI-S","Evaluate the effect of PORT-77 on the Patient Global Impressions - Severity of clinical status.\n\nThe PGI-S measures Severity of clinical status. Higher numerical scores in PGI-S (1-5) indicate greater symptom severity.",{"measure":42,"description":43,"timeFrame":12},"Change from baseline in validated QoL scores - SF36","Evaluate the effect of PORT-77 on the Short Form 36 Health Survey (SF-36) in adults compared to placebo.\n\nThe SF-36 covers 36 individual items that evaluate functional health and well-being. It produces eight health domain scores and two summary component scores (Physical Component Summary and Mental Component Summary) where higher values (0-100) represent better health.",{"measure":45,"description":46,"timeFrame":12},"Change from baseline in validated QoL scores - PedsQL","Evaluate the effect of PORT-77 on the Pediatric Quality of Life Inventory (PedsQL) in adolescents compared to placebo.\n\nThe (PedsQL) evaluates four main domains: physical functioning, emotional functioning, social functioning, and school functioning. It uses a 0 to 100 scale where higher scores show a better health-related quality of life.",{"measure":48,"description":49,"timeFrame":50},"Area under the curve (AUCtau) of PORT-77","Model derived area under the curve of PORT-77 during dosing interval at steady state","Part 1 - Day 1 through 84; Part 2 - through Day 182",{"measure":52,"description":53,"timeFrame":50},"Cmax of PORT-77","Model derived maximal concentration after dosing of PORT-77 at steady state",{"measure":55,"description":56,"timeFrame":50},"Ctrough of PORT-77","Model derived trough concentration after dosing of PORT-77 at steady state",null,{"allocation":59,"interventionModel":60,"interventionModelDescription":57,"primaryPurpose":61,"observationalModel":57,"timePerspective":57,"maskingInfo":62},"RANDOMIZED","PARALLEL","TREATMENT",{"masking":63,"maskingDescription":57,"whoMasked":64},"QUADRUPLE",[65,66,67,68],"PARTICIPANT","CARE_PROVIDER","INVESTIGATOR","OUTCOMES_ASSESSOR","PATHWAY is a Phase 2b\u002F3, multiregional, multicenter clinical study of PORT-77 in adults and adolescents with EPP or XLP. The study consists of 3 parts: Part 1 (Phase 2b), Part 2 (Phase 3), and Part 3 (OLE).\n\nPart 1 and Part 2 are randomized, quadruple-blinded, placebo-controlled studies. Part 1 is a Phase 2b study evaluating the safety, tolerability, and preliminary efficacy of 2 dose levels of PORT-77. Part 2 is a Phase 3 study designed to confirm the efficacy of the selected PORT-77 dose and to further characterize safety in a larger cohort. All participants who complete Part 1 or Part 2 of the study will have the opportunity to enroll in Part 3 (OLE).",[71,77,81,87,91,94],{"label":72,"type":73,"description":74,"interventionNames":75},"Phase 2b (Treatment Group 1)","EXPERIMENTAL","PORT-77 Dose 1",[76],"Drug: PORT-77",{"label":78,"type":73,"description":79,"interventionNames":80},"Phase 2b (Treatment Group 2)","PORT-77 Dose 2",[76],{"label":82,"type":83,"description":84,"interventionNames":85},"Phase 2b (Treatment Group 3)","PLACEBO_COMPARATOR","Placebo",[86],"Drug: Placebo",{"label":88,"type":73,"description":89,"interventionNames":90},"Phase 3 (Treatment Group 1)","PORT-77",[76],{"label":92,"type":83,"description":84,"interventionNames":93},"Phase 3 (Treatment Group 2)",[86],{"label":95,"type":73,"description":89,"interventionNames":96},"Phase 3 (Open-Label Extension)",[76],[98,102],{"type":99,"name":89,"description":100,"armGroupLabels":101,"otherNames":57},"DRUG","Oral tablets",[72,78,95,88],{"type":99,"name":84,"description":100,"armGroupLabels":103,"otherNames":57},[82,92],[105],{"name":106,"affiliation":5,"role":107},"Chief Medical Officer","STUDY_DIRECTOR",[109],{"name":5,"role":110,"phone":111,"phoneExt":57,"email":112},"CONTACT","877-660-6479","Portal.clinical.trials@gondolabio.com",[114,131],{"facility":115,"status":116,"city":117,"state":118,"zip":119,"country":120,"countryCode":121,"cosmosGeoPoint":122,"geoPoint":127,"contacts":128},"Investigative Site","RECRUITING","Huntington Beach","California","92647","United States","US",{"type":123,"coordinates":124},"Point",[125,126],-117.99923,33.6603,{"lat":126,"lon":125},[129],{"name":130,"role":110,"phone":57,"phoneExt":57,"email":57},"Site Contact",{"facility":115,"status":116,"city":132,"state":133,"zip":134,"country":120,"countryCode":121,"cosmosGeoPoint":135,"geoPoint":139,"contacts":140},"Cincinnati","Ohio","43215",{"type":123,"coordinates":136},[137,138],-84.51439,39.12711,{"lat":138,"lon":137},[141],{"name":130,"role":110,"phone":57,"phoneExt":57,"email":57},{"type":143,"investigatorFullName":57,"investigatorTitle":57,"investigatorAffiliation":57,"oldNameTitle":57,"oldOrganization":57},"SPONSOR","100655993","phase-2-a-study-to-investigate-port-77-oral-dosing-compared-with-placebo-in-participants-with-erythropoietic-protoporphyria-epp-or-x-linked-protoporphyria-xlp-100655993",false,"NCT07820085","A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)","A Phase 2b\u002F3, Quadruple-Blinded, Randomized, Placebo-Controlled Study of PORT-77 Administered Orally to Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria and an Open-Label Extension","PATHWAY","Inclusion Criteria:\n\n* Aged 12 years or older.\n* Clinical history of EPP, XLP, or EPP2 supported by genetic confirmation or historical laboratory test results\n* History of consistent, non-painful prodrome within approximately 45 minutes of sunlight exposure and prior to phototoxic attacks\n* Demonstrates ≥85% compliance with daily symptom diary during run-in period.\n* Body weight or BMI at Screening as follows:\n\n  1. For participants aged 12 to \\\u003C18 years: body weight ≥32 kg\n  2. For participants aged ≥18 years: BMI ≥18.5 kg\u002Fm2\n* AST and ALT \\\u003C3 × ULN and total bilirubin \\\u003C2 × ULN (unless documented Gilbert syndrome) at Screening\n* Willing and able to provide informed consent and\u002For assent for the study.\n* Willing and able to comply with study visits, study procedures, and contraception guidance\n* Intends to remain in the same approximate geographic latitude for the duration of the placebo-controlled period, with no more than 14 days spent outside this region.\n\nExclusion Criteria:\n\n* Diagnosis of another porphyria or another photodermatosis that may confound the evaluation of PORT-77\n* Any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory assessments\n* Major surgery within 8 weeks before Screening, incomplete recovery from any previous surgery, or major surgery planned to occur during the study\n* History of, or anticipated need for, liver transplantation or history of bone marrow transplantation\n* Active infection with hepatitis B or C\n* Unable to swallow tablets or has a disease that significantly affects gastrointestinal function\n* Any other disease, condition, or circumstance that, at the discretion of the Investigator or Sponsor, would interfere with the evaluation of PORT-77 or study participation, or would make study participation not in the best interest of the participant\n* History of drug or alcohol abuse within the last 12 months, or current or planned use of prohibited or illegal substances\n* Has taken any medication, vitamin, or supplement that alters sensitivity to light exposure (eg, afamelanotide, melanotan, beta carotene, dersimelagon) within 90 days of Day 1; or has taken bitopertin within 120 days of Day 1; or has taken iron within 30 days of Day 1\n* Drugs or supplements that may impact or be impacted by PORT-77\n* Concurrent or anticipated participation in an interventional clinical trial during the study period.\n* Received another investigational therapy within 5 half-lives, if the half-life is known, or within 30 days, if the half-life is unknown, prior to Day 1.\n* Known hypersensitivity to PORT-77 or excipients\n* Female who has a positive pregnancy test at Screening or Day 1 or is breastfeeding","ALL","12 Years",{"count":155,"type":156},225,"ESTIMATED","INTERVENTIONAL",[159,160],"PHASE2","PHASE3","PATHWAY is a Phase 2b\u002F3 clinical study with an Open-Label Extension (OLE). It tests PORT-77 in patients with EPP, EPP2, and XLP.\n\nThe study has three parts:\n\nPart 1: Phase 2b\n\n* Find the right dose of PORT-77 to lower PPIX levels in the blood\n* Test if PORT-77 lowers PPIX levels better than placebo\n* Check the safety of each dose\n\nPart 2: Phase 3\n\n* Test if PORT-77 lowers PPIX levels better than placebo\n* Test if PORT-77 increases the amount of time patients spend in sunlight each day without pain compared to placebo\n* Check the safety of PORT-77\n\nPart 3: Open-Label Extension (OLE)\n\n* Test the long-term effect of PORT-77 on PPIX levels and time in sun\n* Monitor long-term safety",[163,164,165],"Erythropoietic Protoporphyria (EPP)","X-Linked Protoporphyria (XLP)","EPP2",[89,167,168,169,170,165],"EPP","X-linked protoporphyria","Erythropoietic Protoporphyria","XLP","2026-09-10",{"date":173,"type":174},"2026-09-15","ACTUAL",{"date":176,"type":156},"2026-09",{"date":178,"type":156},"2029-09",{"name":5,"class":6},2]