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225 Participants NeededMy employer runs this trial

PORT-77 for Erythropoietic Protoporphyria

(PATHWAY Trial)

Recruiting at 1 trial location
PT
Overseen ByPortal Therapeutics, Inc.
Age: Any Age
Sex: Any
Trial Phase: Phase 2 & 3
Sponsor: Portal Therapeutics, Inc.
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

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

Who Is on the Research Team?

CM

Chief Medical Officer

Principal Investigator

Portal Therapeutics, Inc.

Are You a Good Fit for This Trial?

This trial is for people diagnosed with Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP), rare conditions that cause painful reactions to sunlight. Participants must meet specific medical criteria set by the study team.

Inclusion Criteria

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
I regularly feel warning signs, but no pain, soon after sun exposure before skin reactions.
Demonstrates ≥85% compliance with daily symptom diary during run-in period
See 7 more

Exclusion Criteria

Concurrent or anticipated participation in an interventional clinical trial during the study period
I am not taking any drugs or supplements that interact with PORT-77.
I have taken an experimental treatment within the last 30 days.
See 11 more

What Are the Treatments Tested in This Trial?

Interventions

  • PORT-77

Trial Overview

The study compares an oral medication called PORT-77 to a placebo in people with EPP or XLP. It aims to find the best dose, see if PORT-77 lowers harmful blood levels and increases pain-free time in sunlight, and monitor safety over both short and long term.

How Is the Trial Designed?

6

Treatment groups

Experimental Treatment

Placebo Group

Group I: Phase 3 (Treatment Group 1)Experimental Treatment1 Intervention
Group II: Phase 3 (Open-Label Extension)Experimental Treatment1 Intervention
Group III: Phase 2b (Treatment Group 2)Experimental Treatment1 Intervention
Group IV: Phase 2b (Treatment Group 1)Experimental Treatment1 Intervention
Group V: Phase 2b (Treatment Group 3)Placebo Group1 Intervention
Group VI: Phase 3 (Treatment Group 2)Placebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Portal Therapeutics, Inc.

Lead Sponsor

Trials
1
Recruited
110+

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