Recruiting
Phase 2
Phase 3

PORT-77

Sponsor:

Portal Therapeutics, Inc.

Code:

NCT07820085

Conditions

Erythropoietic Protoporphyria (EPP)

X-Linked Protoporphyria (XLP)

EPP2

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

Interventions

PORT-77

Placebo

Study Details

Brief 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

Conditions

Erythropoietic Protoporphyria (EPP)

X-Linked Protoporphyria (XLP)

EPP2

Study ID

NCT07820085

Start date

Sep, 2026

Status verified date

Sep, 2026

Completion date

Sep, 2029

Anticipated

Primary completion date

Mar, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

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 <18 years: body weight ≥32 kg
2. For participants aged ≥18 years: BMI ≥18.5 kg/m2
  • AST and ALT <3 × ULN and total bilirubin <2 × ULN (unless documented Gilbert syndrome) at Screening
  • Willing and able to provide informed consent and/or assent for the study.
  • Willing and able to comply with study visits, study procedures, and contraception guidance
  • 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.

Exclusion Criteria:

  • 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
  • Active infection with hepatitis B or C
  • Unable to swallow tablets or has a disease that significantly affects gastrointestinal function
  • Any other disease, condition, or circumstance that, at the discretion of the Investigator or Sponsor, would interfere with the evaluation of PORT-77 or study participation, or would make study participation not in the best interest of the participant
  • History of drug or alcohol abuse within the last 12 months, or current or planned use of prohibited or illegal substances
  • Has taken any medication, vitamin, or supplement that alters sensitivity to light exposure (eg, afamelanotide, melanotan, beta carotene, dersimelagon) within 90 days of Day 1; or has taken bitopertin within 120 days of Day 1; or has taken iron within 30 days of Day 1
  • Drugs or supplements that may impact or be impacted by PORT-77
  • Concurrent or anticipated participation in an interventional clinical trial during the study period.
  • Received another investigational therapy within 5 half-lives, if the half-life is known, or within 30 days, if the half-life is unknown, prior to Day 1.
  • Known hypersensitivity to PORT-77 or excipients
  • Female who has a positive pregnancy test at Screening or Day 1 or is breastfeeding

Study Design

Enrollment

225 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

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

Interventions

PORT-77

Oral tablets

Placebo

Oral tablets

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 ]
  • Adverse Events [ Time Frame: Part 1 - Day 1 through Day 84; Part 2 - through Day 182; Part 3 - through Month 12 ]
  • Average daily time in sunlight without pain [ Time Frame: Part 2 - Day 155 through 182 ]

Central Contacts and Locations

Central contacts

Portal Therapeutics, Inc.

877-660-6479Portal.clinical.trials@gondolabio.com

Locations

Investigative Site

Recruiting

Huntington Beach, California, United States, 92647

Investigative Site

Recruiting

Cincinnati, Ohio, United States, 43215

More Information

Sponsor

Portal Therapeutics, Inc.

Last update posted

Sep 15, 2026

Last verified

Sep, 2026

Keywords

  • PORT-77
  • EPP
  • X-linked protoporphyria
  • Erythropoietic Protoporphyria
  • XLP
  • EPP2

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-16. This information was provided to ClinicalTrials.gov by Portal Therapeutics, Inc. on 2026-09-15.