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

NCT06971900 | Enrolling By Invitation Phase 2 FDA Drug

• 1 other identifier: PORT-77-201
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Erythropoietic Protoporphyria (EPP)

Keywords

PORT-77; EPP; erythropoietic protoporphyria

Outcome Measures

Primary Outcomes (1)

• Plasma PPIX level

  Change over the course of the study in plasma PPIX concentrations

  9 Days

Secondary Outcomes (4)

• Evaluate safety and tolerability of 2 different dose regimens of PORT-77

  9 Days

• Cmax

  Predose and 1, 2, 3, 4, 6, 8, 12 hours post dose

• Tmax

  Predose and 1, 2, 3, 4, 6, 8, 12 hours post dose

• AUC0-last

  Predose and 1, 2, 3, 4, 6, 8, 12 hours post dose

Study Arms (2)

PORT-77

EXPERIMENTAL

Active

Drug: PORT-77

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Interventions

PORT-77 DRUG

Active oral dose form

PORT-77

Placebo DRUG

Matching inactive oral dose form

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Portal Therapeutics, Inc.: lead
• Celerion: collaborator

Study Sites (1)

Celerion

Tempe, Arizona, 85283, United States

Location

MeSH Terms

Conditions

Protoporphyria, Erythropoietic

Condition Hierarchy (Ancestors)

Porphyrias, Hepatic; Liver Diseases; Digestive System Diseases; Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Porphyrias; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Chief Medical Officer

  Portal Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 3, 2025
• First Posted: May 14, 2025
• Study Start: April 4, 2025
• Primary Completion: January 1, 2026
• Study Completion: January 1, 2026
• Last Updated: May 14, 2025

Record last verified: 2025-04

Locations

US (1)