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

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

Eligibility

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

Conditions4

Interventions1

Locations2 sites

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