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NCT07820085
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
Conditions: Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP), EPP2
Sex: All
Ages: 12 Years – N/A
Healthy volunteers: No
Phase: PHASE2, PHASE3
Enrollment: 225
Sponsor: Portal Therapeutics, Inc.
Location: Investigative Site Huntington Beach California
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 \
Source: ClinicalTrials.gov (NCT07820085). StuddyBuddy aggregates publicly available trial information.