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

Conditions

• EPP2
• Erythropoietic Protoporphyria (EPP)
• X-Linked Protoporphyria (XLP)

Eligibility Criteria

Sex

ALL

Age

12 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• PORT-77 — DRUG
  Oral tablets
• Placebo — DRUG
  Oral tablets

Study Details

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

Key Dates

First listed

Sep 15, 2026

Start date

Sep 30, 2026

Status verified

Sep 2026

Primary completion

Mar 31, 2029

Completion

Sep 30, 2029

Study Design

Enrollment

225 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Phase 2b (Treatment Group 1)
  PORT-77 Dose 1
• Experimental: Phase 2b (Treatment Group 2)
  PORT-77 Dose 2
• Placebo Comparator: Phase 2b (Treatment Group 3)
  Placebo
• Experimental: Phase 3 (Treatment Group 1)
  PORT-77
• Placebo Comparator: Phase 3 (Treatment Group 2)
  Placebo
• Experimental: Phase 3 (Open-Label Extension)
  PORT-77

Primary Outcome Measure

Plasma metal-free PPIX concentration change [ Time Frame: Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12 ]

Central Contacts

• Portal Therapeutics, Inc.
  877-660-6479
  [email protected]

Locations (2)

Find similar trials in Huntington Beach, CA

Related Studies

• Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)
  Recruiting · Portal Therapeutics, Inc. · Boston, Massachusetts