PATHWAY Clinical Research Study for EPP & XLP
PATHWAY is a clinical research study evaluating an investigational medication called PORT-77 for people living with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP).
The study is designed to learn how well PORT-77 works, understand possible side effects, and evaluate its potential impact on people living with EPP or XLP. Individuals with EPP or XLP may be eligible to participate.
Participants will be randomly assigned to receive either PORT-77 or a placebo (a pill with no active medication). Neither participants nor the study team will know which treatment is being received during this part of the study. After completing the blinded portion of the study, participants may have the opportunity to continue into an open-label extension where all participants receive PORT-77 for up to 12 months.
Participation includes study-related visits with healthcare professionals experienced in treating EPP and XLP. By taking part, participants may also help advance research that could support the development of future investigational therapies for these conditions. Participation is voluntary, and participants may withdraw at any time.
Who may be eligible?
Are 12 years of age or older
Have a confirmed diagnosis of EPP or XLP
Have symptoms consistent with EPP/XLP
Meet other study eligibility criteria, as determined by the study team
What does participation involve?
Participants will complete a screening visit and attend scheduled study visits throughout the study. These visits may include routine clinical assessments, symptom tracking, and other research-related evaluations. A physician or study team member will determine whether the study is a good fit for you.
Study Locations
The PATHWAY study is being conducted at sites across North America, South America, the United Kingdom, the European Union, Turkey, and Australia. To be connected with a study site please complete this interest form.
The study is sponsored by GondolaBio, a clinical-stage biopharmaceutical company developing potential therapies for people living with genetic diseases.
