GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria

Phase 2

• Conditions: Erythropoietic Protoporphyria (EPP)
• Interventions: Drug: PORT-77; Drug: Placebo

• Registration Number: NCT06971900
• Lead Sponsor: Portal Therapeutics, Inc.

Brief Summary

A Phase 2a study of PORT-77 in adults with erythropoietic protoporphyria (EPP)

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-04
• Study First Submitted: 2025-04-03
• Study Start: 2025-04-04
• Study First Submitted QC: 2025-05-07
• Last Update Submitted: 2025-05-07
• Study First Posted: 2025-05-14
• Last Update Posted: 2025-05-14
• Primary Completion: 2026-01-01
• Study Completion: 2026-01

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 14

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-EPI equation at screening
• Hepatic impairment, with alanine aminotransferase, aspartate aminotransferase, or total bilirubin >1.5 x ULN at screening
• Female participant with a positive pregnancy test at screening or who is breastfeeding.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
PORT-77	PORT-77	Active
Placebo	Placebo	Placebo

Primary Outcome Measures

Name	Time	Method
Plasma PPIX level	9 Days	Change over the course of the study in plasma PPIX concentrations

Secondary Outcome Measures

Name	Time	Method
Evaluate safety and tolerability of 2 different dose regimens of PORT-77	9 Days	Safety and tolerability over the course of the study, as assessed by adverse events (AEs) and laboratory results
Cmax	Predose and 1, 2, 3, 4, 6, 8, 12 hours post dose	Maximum measured concentration of PORT-77 in plasma (Cmax).
AUC0-last	Predose and 1, 2, 3, 4, 6, 8, 12 hours post dose	Area under the concentration-time curve of of PORT-77 in plasma over the time interval from 0 to the time of the last quantifiable data point.
Tmax	Predose and 1, 2, 3, 4, 6, 8, 12 hours post dose	Time from dosing to maximum measured concentration of PORT-77 in plasma

Trial Locations

Locations (1)

Celerion; 🇺🇸 Tempe, Arizona, United States