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

NCT06971900 | Completed Phase 2 FDA Drug

• 1 other identifier: PORT-77-201
• Study Type: interventional
• Target: 28
• 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 (9)

• Safety and tolerability

  9 Days

• Area Under the Plasma Concentration-Time Curve From Time Zero to Last Quantifiable Concentration (AUC0-last)

  Day 1 through Day 9

• Area Under the Plasma Concentration-Time Curve Over the 12-Hour Dosing Interval at Steady State (AUC0-12)

  Up to Day 8

• Area Under the Plasma Concentration-Time Curve Over the 24-Hour Dosing Interval at Steady State (AUC0-24)

  Up to Day 8

• Maximum Observed Plasma Concentration (Cmax)

  Day 1 through Day 9

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 ≤40.0 kg/m2 and weight ≥50 kg on Day -1.
• Nonsmoker or willing to refrain from the use of nicotine or tobacco-containing products for at least 30 days prior to the first dose of study drug based on participant self-reporting and agrees to refrain from the use of these products throughout the study.
• Known diagnosis of EPP.
• History of consistent, non-painful prodrome prior to phototoxic attacks.
• Experiences prodrome within 40 minutes of uncovered sunlight exposure.
• Willing and able to follow protocol-specified contraception guidance.
• Able to read and understand English and is willing and able to provide document informed consent to participate in the study at the time of the Remote Screening Visit and at the In-Clinic Screening Visit.
• 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 or has significant emotional problems identified or observed during the screening period or expected during the conduct of the study that could lead to difficulty complying with study procedures or interfere with the safe completion of the study.
• 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.
• Current alcohol or drug use disorder, as defined by the DSM-V-TR.
• 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).
• Received another investigational medicine within 5 half-lives, if the half-life is known, or within 28 days, if the half-life is unknown, prior to Day 1.
• Surgical procedure planned within 28 days prior to Day 1.
• Donation of blood or significant blood loss within 60 days prior to Day 1.
• Plasma donation within 7 days prior to Day 1.
• Unable to refrain from or anticipates the use of any of the following beginning 28 days (or 5 half-lives, whichever is longer) prior to the first dose of study drug and throughout the study:
• Substrates of BCRP or MATE1/2-K
• CYP3A4 substrates that cannot be separated by at least 2 hours from PORT-77 dosing
• Moderate or strong P-gp inhibitors or inducers
• Moderate or strong CYP3A4 inhibitors or inducers
• Herbal supplements or food products containing grapefruit juice, star fruit, or Seville oranges

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: SEQUENTIAL
• 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: November 22, 2025
• Study Completion: November 24, 2025
• Last Updated: July 29, 2026

Record last verified: 2026-07

Locations

US (1)