GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria (NCT06971900) | Clinical Trial Compass
By InvitationPhase 2
GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria
United States14 participantsStarted 2025-04-04
Plain-language summary
A Phase 2a study of PORT-77 in adults with erythropoietic protoporphyria (EPP)
Who can participate
Age range
18 Years – 80 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Major Inclusion Criteria:
* BMI ≥18.0 and ≤35.0 kg/m2 and weight ≥50 kg
* Known diagnosis of EPP and is currently symptomatic even if receiving treatment for EPP, including afamelanotide.
* Willing and able to avoid exposure to sunlight when outside of the clinical research site.
* Willing and able to follow protocol-specified contraception guidance.
* Able to read and understand English
* Able to understand the study procedures as described in the ICF and is willing and able to comply with the study requirements.
Major Exclusion Criteria:
* Is mentally or legally incapacitated
* History or presence of any illness or clinically significant medical or psychiatric condition or disease that, in the opinion of the PI or designee, might confound the results of the study or pose an additional risk to the participant by their participation in the study.
* History of gastrointestinal condition, including surgeries, which may affect absorption after oral administration.
* History of cancer, with the exception of cutaneous non-melanoma skin cancer (basal or squamous cell carcinoma).
* Participation in another clinical study within 28 days or within 5 half-lives (if known), prior to screening
* Unable to refrain from or anticipates the use of medications/supplements known to confound PORT-77 beginning 28 days (or 5 half-lives, whichever is longer) prior to the first dose of study drug and throughout the study.
* Estimated glomerular filtration rate \<80 mL/min/1.73 m2 using the CKD-E…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.