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

ConditionsErythropoietic Protoporphyria (EPP)X-Linked Protoporphyria (XLP)EPP2
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age12+
SponsorPortal Therapeutics, Inc.

About 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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • PORT-77

    Drug

    Oral tablets

  • Placebo

    Drug

    Oral tablets

Treatment groups

225 Participants
are divided into 6 treatment groups

6

Treatment groups

See each treatment group below.

Group A: Phase 2b (Treatment Group 1)Experimental treatment 1 intervention
Group B: Phase 2b (Treatment Group 2)Experimental treatment 1 intervention
Group C: Phase 2b (Treatment Group 3)Placebo comparator 1 intervention
Group D: Phase 3 (Treatment Group 1)Experimental treatment 1 intervention
Group E: Phase 3 (Treatment Group 2)Placebo comparator 1 intervention
Group F: Phase 3 (Open-Label Extension)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Plasma metal-free PPIX concentration change

Characterize the dose-response of PORT-77 on plasma PPIX levels compared to placebo

Time frame
Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
2

Adverse Events

Evaluate safety and tolerability of each dose of PORT-77 over the course of the study

Time frame
Part 1 - Day 1 through Day 84; Part 2 - through Day 182; Part 3 - through Month 12
3

Average daily time in sunlight without pain

Assess the effect of different doses of PORT-77 on average daily time in sunlight without pain compared to placebo

Time frame
Part 2 - Day 155 through 182

Secondary outcomes

1

Average daily time in sunlight without pain

Assess the effect of different doses of PORT-77 on average daily time in sunlight without pain compared to placebo

Time frame
Part 1 - Days 57 through 84
2

Change from baseline in daily time in sunlight before first prodromal symptom

Assess the effect of different doses of PORT-77 on average daily time in sunlight before first prodromal symptom compared to placebo

Time frame
Part 1 - Day 1 through Day 84; Part 2 - through Day 182
3

Cumulative total time in sunlight without pain

Assess the effect of different doses of PORT-77 on cumulative total time in sunlight without pain compared to placebo

Time frame
Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
4

Number and severity of phototoxic reactions

Assess the effect of different doses of PORT-77 on the rate and severity of phototoxic reactions compared to placebo

Time frame
Part 1 - Day 1 through 84; Part 2 - through Day 182; through Month 12

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Portal Therapeutics, Inc.

Lead sponsor

Source ClinicalTrials.gov
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