NCT07820085 A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
| NCT ID | NCT07820085 |
| Status | Recruiting |
| Phase | Phase 2, Phase 3 |
| Sponsor | Portal Therapeutics, Inc. |
| Condition | Erythropoietic Protoporphyria (EPP) |
| Study Type | INTERVENTIONAL |
| Enrollment | 225 participants |
| Start Date | 2026-09 |
| Primary Completion | 2029-03 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 225 participants in total. It began in 2026-09 with a primary completion date of 2029-03.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
Eligibility Criteria
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 ≥85% 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 ≥32 kg 2. For participants aged ≥18 years: BMI ≥18.5 kg/m2 * AST and ALT \<3 × ULN and total bilirubin \<2 × 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
Contact & Investigator
Chief Medical Officer
STUDY DIRECTOR
Portal Therapeutics, Inc.
Frequently Asked Questions
Who can join the NCT07820085 clinical trial?
This trial is open to participants of all sexes, aged 12 Years or older, studying Erythropoietic Protoporphyria (EPP). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07820085 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT07820085 currently recruiting?
Yes, NCT07820085 is actively recruiting participants. Contact the research team at Portal.clinical.trials@gondolabio.com for enrollment information.
Where is the NCT07820085 trial being conducted?
This trial is being conducted at Huntington Beach, United States, Cincinnati, United States.
Who is sponsoring the NCT07820085 clinical trial?
NCT07820085 is sponsored by Portal Therapeutics, Inc.. The principal investigator is Chief Medical Officer at Portal Therapeutics, Inc.. The trial plans to enroll 225 participants.