Clinical Research Directory
Browse clinical research sites, groups, and studies.
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
Sponsor: Portal Therapeutics, Inc.
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
Official title: A Phase 2b/3, 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
Key Details
Gender
All
Age Range
12 Years - Any
Study Type
INTERVENTIONAL
Enrollment
225
Start Date
2026-09
Completion Date
2029-09
Last Updated
2026-09-15
Healthy Volunteers
No
Interventions
PORT-77
Oral tablets
Placebo
Oral tablets
Locations (2)
Investigative Site
Huntington Beach, California, United States
Investigative Site
Cincinnati, Ohio, United States