A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
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
Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
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
Primary outcome measure(s)
Plasma metal-free PPIX concentration change — Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12 Characterize the dose-response of PORT-77 on plasma PPIX levels compared to placebo
Adverse Events — Part 1 - Day 1 through Day 84; Part 2 - through Day 182; Part 3 - through Month 12 Evaluate safety and tolerability of each dose of PORT-77 over the course of the study
Average daily time in sunlight without pain — Part 2 - Day 155 through 182 Assess the effect of different doses of PORT-77 on average daily time in sunlight without pain compared to placebo
Trial sites (2)
Facility
City
Region
Status
Investigative Site
Huntington Beach
California
Recruiting
Investigative Site
Cincinnati
Ohio
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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