AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

โ† Trials
๐Ÿงช

A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)

RecruitingPhase 2, Phase 3Ages 12 Years - anyAllPortal Therapeutics, Inc.
๐Ÿ’ฌ

In plain words

What the trial is, and whether it could suit you

PATHWAY is a Phase 2b/3 clinical study with an Open-Label Extension (OLE). It tests PORT-77 in patients with EPP, EPP2, and XLP. The study has three parts: Part 1: Phase 2b * Find the right dose of PORT-77 to lower PPIX levels in the blood * Test if PORT-77 lowers PPIX levels better than placebo * Check the safety of each dose Part 2: Phase 3 * Test if PORT-77 lowers PPIX levels better than placebo * Test if PORT-77 increases the amount of time patients spend in sunlight each day without pain compared to placebo * Check the safety of PORT-77 Part 3: Open-Label Extension (OLE) * Test the long-term effect of PORT-77 on PPIX levels and time in sun * Monitor long-term safety

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * Aged 12 years or older. * Clinical history of EPP, XLP, or EPP2 supported by genetic confirmation or historical laboratory test results * History of consistent, non-painful prodrome within approximately 45 minutes of sunlight exposure and prior to phototoxic attacks * Demonstrates โ‰ฅ85% compliance with daily symptom diary during run-in period. * Body weight or BMI at Screening as follows: 1. For participants aged 12 to \<18 years: body weight โ‰ฅ32 kg 2. For participants aged โ‰ฅ18 years: BMI โ‰ฅ18.5 kg/m2 * AST and ALT \<3 ร— ULN and total bilirubin \<2 ร— ULN (unless documented Gilbert syndrome) at Screening * Willing and able to provide informed consent and/or assent for the study. * Willing and able to comply with study visits, study procedures, and contraception guidance * 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. Exclusion Criteria: * Diagnosis of another porphyria or another photodermatosis that may confound the evaluation of PORT-77 * Any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory assessments * Major surgery within 8 weeks before Screening, incomplete recovery from any previous surgery, or major surgery planned to occur during the study * History of, or anticipated need for, liver transplantation or history of bone marrow transplantation * Active infection with hepatitis B or C * Unable to swallow tablets or has a disease that significantly affects gastrointestinal function * 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 * History of drug or alcohol abuse within the last 12 months, or current or planned use of prohibited or illegal substances * 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 * Drugs or supplements that may impact or be impacted by PORT-77 * Concurrent or anticipated participation in an interventional clinical trial during the study period. * 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. * Known hypersensitivity to PORT-77 or excipients * Female who has a positive pregnancy test at Screening or Day 1 or is breastfeeding
๐Ÿ’Š

What it tests

  • PORT-77
  • Placebo
๐Ÿ“

Where

2 sites
Investigative Site
Huntington Beach, United States
Investigative Site
Cincinnati, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
๐Ÿ›ก๏ธ

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it โ€” not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use ยท Privacy