NCT07820085 · Portal Therapeutics, Inc.
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
(PATHWAY)
What this study is about
PATHWAY is a Phase 2b/3 clinical study with an where both patients and doctors know the treatment given Extension (OLE). It tests PORT-77 in patients with EPP, EPP2, and XLP.
View original scientific description
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.
Interventions
DRUG
PORT-77
Oral tablets
DRUG
Placebo
Oral tablets
Primary outcome measures
Plasma metal-free PPIX concentration change
Time frame: 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
Time frame: 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
Time frame: 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
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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:
- For participants aged 12 to \<18 years: body weight ≥32 kg
- 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
Where
- Huntington Beach, California
- Cincinnati, Ohio
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Sep 15, 2026 · Source of record for eligibility and locations