A Phase 2/3 interventional study of PORT-77 and Placebo in Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP) and EPP2, sponsored by Portal Therapeutics, Inc.. Recruiting at 2 sites in United States. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-09-15.
Sponsored by Portal Therapeutics, Inc. · Phase 2/3, Interventional, and Treatment
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Among 18 studies in Protoporphyria, Erythropoietic with a site in United States — 4 open to participants now, 4 under way, 2 of them phase 3. Protoporphyria, Erythropoietic · United States.
Among the 18 studies in Protoporphyria, Erythropoietic with a site in United States, this one is the 2nd largest by planned enrollment of the 13 interventional ones that give a count, is one of 2 that started in 2026, is one of 3 in phase 2/3, and was the 18th registered.
29 studies on the registry are indexed under Protoporphyria, Erythropoietic; 5 are open to participants now.
Browse Protoporphyria, Erythropoietic studies →4 of the 18 studies in Protoporphyria, Erythropoietic with a site in United States are open now. Recruiting first, the newest first:
Portal Therapeutics, Inc. · Sites in United States, Belgium, Canada and 6 more countries
Ages 12+
The American Porphyrias Expert Collaborative · Sites in United States
Ages 1 min+
Disc Medicine, Inc · Sites in United States, Australia, Belgium and 8 more countries
Ages 12+
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 3 Protoporphyria, Erythropoietic studies recruiting now, 3 set no upper age limit and none stop at 75 or younger; 3 take children under 18; none take healthy volunteers.
This study: 12 Years and older · all sexes · no healthy volunteers.
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18 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 2 8 · Phase 3 6
First in 2010; this study’s year is marked. 2 started in 2026.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 225 is above the median of 62 across 20 interventional studies indexed under Protoporphyria, Erythropoietic.
Median enrollment 77 across the 13 interventional studies, 150 across the 4 observational. Of the 13 that completed or were terminated, 9 (69%) have results posted.
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11 of 18 have an industry lead sponsor. The 10 most frequent:
Portal Therapeutics, Inc. is the lead sponsor of 4 studies on the registry; 2 are open to participants now.
Portal Therapeutics, Inc. has 3 studies filed under Protoporphyria, Erythropoietic — fewer than ten, so there is no comparison to draw.
1 of the studies in Protoporphyria, Erythropoietic with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07567131Portal Therapeutics, Inc.Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP) | 2027-03-01 |
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Among the 18 studies in Protoporphyria, Erythropoietic with a site in United States, from their registry updates:
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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
Part 2: Phase 3
Part 3: Open-Label Extension (OLE)
PATHWAY is a Phase 2b/3, multiregional, multicenter clinical study of PORT-77 in adults and adolescents with EPP or XLP. The study consists of 3 parts: Part 1 (Phase 2b), Part 2 (Phase 3), and Part 3 (OLE).
Part 1 and Part 2 are randomized, quadruple-blinded, placebo-controlled studies. Part 1 is a Phase 2b study evaluating the safety, tolerability, and preliminary efficacy of 2 dose levels of PORT-77. Part 2 is a Phase 3 study designed to confirm the efficacy of the selected PORT-77 dose and to further characterize safety in a larger cohort. All participants who complete Part 1 or Part 2 of the study will have the opportunity to enroll in Part 3 (OLE).
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Body weight or at Screening as follows:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
PORT-77 Dose 1
Drug: PORT-77
PORT-77 Dose 2
Drug: PORT-77
Placebo
Drug: Placebo
PORT-77
Drug: PORT-77
Placebo
Drug: Placebo
PORT-77
Drug: PORT-77
Oral tablets
Oral tablets
Plasma metal-free PPIX concentration change
Characterize the dose-response of PORT-77 on plasma PPIX levels compared to placebo
Time frame: Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Adverse Events
Evaluate safety and tolerability of each dose of PORT-77 over the course of the study
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
Assess the effect of different doses of PORT-77 on average daily time in sunlight without pain compared to placebo
Time frame: Part 2 - Day 155 through 182
Average daily time in sunlight without pain
Assess the effect of different doses of PORT-77 on average daily time in sunlight without pain compared to placebo
Time frame: Part 1 - Days 57 through 84
Change from baseline in daily time in sunlight before first prodromal symptom
Assess the effect of different doses of PORT-77 on average daily time in sunlight before first prodromal symptom compared to placebo
Time frame: Part 1 - Day 1 through Day 84; Part 2 - through Day 182
Cumulative total time in sunlight without pain
Assess the effect of different doses of PORT-77 on cumulative total time in sunlight without pain compared to placebo
Time frame: Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Number and severity of phototoxic reactions
Assess the effect of different doses of PORT-77 on the rate and severity of phototoxic reactions compared to placebo
Time frame: Part 1 - Day 1 through 84; Part 2 - through Day 182; through Month 12
Change from baseline in validated QoL scores - PGI-C
Evaluate the effect of PORT-77 on the Patient Global Impressions - Change in clinical status (PGI-C) in adults compared to placebo. The PGI-C measures Change in clinical status. Higher numerical scores (1-7) in PGI-C indicate worsening of clinical status.
Time frame: Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Change from baseline in validated QoL scores - PGI-S
Evaluate the effect of PORT-77 on the Patient Global Impressions - Severity of clinical status. The PGI-S measures Severity of clinical status. Higher numerical scores in PGI-S (1-5) indicate greater symptom severity.
Time frame: Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Change from baseline in validated QoL scores - SF36
Evaluate the effect of PORT-77 on the Short Form 36 Health Survey (SF-36) in adults compared to placebo. The SF-36 covers 36 individual items that evaluate functional health and well-being. It produces eight health domain scores and two summary component scores (Physical Component Summary and Mental Component Summary) where higher values (0-100) represent better health.
Time frame: Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Change from baseline in validated QoL scores - PedsQL
Evaluate the effect of PORT-77 on the Pediatric Quality of Life Inventory (PedsQL) in adolescents compared to placebo. The (PedsQL) evaluates four main domains: physical functioning, emotional functioning, social functioning, and school functioning. It uses a 0 to 100 scale where higher scores show a better health-related quality of life.
Time frame: Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Area under the curve (AUCtau) of PORT-77
Model derived area under the curve of PORT-77 during dosing interval at steady state
Time frame: Part 1 - Day 1 through 84; Part 2 - through Day 182
Cmax of PORT-77
Model derived maximal concentration after dosing of PORT-77 at steady state
Time frame: Part 1 - Day 1 through 84; Part 2 - through Day 182
Ctrough of PORT-77
Model derived trough concentration after dosing of PORT-77 at steady state
Time frame: Part 1 - Day 1 through 84; Part 2 - through Day 182
Plan to share: Undecided
No publications or documents are linked to this record.
Start is estimated for Sep 2026; primary completion for Mar 2029, 30 months later.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: PORT-77
Protoporphyria, Erythropoietic →
Portal Therapeutics, Inc.