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

Portal Therapeutics

Status and phase: Invitation-only; Phase 2

Conditions: Erythropoietic Protoporphyria (EPP)

Treatments: Drug: PORT-77; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT06971900

PORT-77-201

Details and patient eligibility

About

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

Enrollment: 14 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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-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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Double Blind

---

14 participants in 2 patient groups, including a placebo group

PORT-77

Experimental group

Description:

Active

Treatment:

Drug: PORT-77

Placebo

Placebo Comparator group

Description:

Placebo

Treatment:

Drug: Placebo