Actively Recruiting
Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)
Led by Portal Therapeutics, Inc. · Updated on 2026-05-05
50
Participants Needed
19
Research Sites
48 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this study is to collect information about the effects of EPP/XLP in adults and adolescents. This is an observational study in which participants will not receive any treatment. Study details include: * The study duration will be up to 6 months for each participant. * After Screening, participants will have a Baseline (Day 1) visit and return for visits every 4 weeks through 24 weeks. * Assessments to evaluate EPP/XLP will include clinical laboratory tests, physical exams, vital signs measurement, blood draws for PPIX concentration, reporting of EPP/XLP-related symptoms (including completion of a daily diary), light dosimetry.
CONDITIONS
Official Title
Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 12 years and older
- Confirmed diagnosis of EPP or XLP by genetic testing or elevated free PPIX levels with clinical/family history
- Currently has symptoms of EPP or XLP
- History of consistent, non-painful prodrome within about 45 minutes of sunlight exposure before phototoxic attacks
- Willing and able to wear a light dosimetry device during the study
- Willing and able to complete a daily diary of EPP/XLP symptoms
- Willing and able to keep skin sites covered with opaque material when outside or exposed to triggering light starting 2 days before sunlight exposure testing
- Willing and able to provide informed consent or assent
- Willing and able to comply with study visits and procedures
You will not qualify if you...
- Diagnosis of another porphyria or photodermatosis that may interfere with EPP/XLP evaluation
- Having a significant disease or condition that may interfere with study participation or evaluation
- Taking or having taken within 60 days any medication, vitamin, or supplement that changes sensitivity to light exposure (e.g., afamelanotide, melanotan, beta carotene, dersimelagon, bitopertin)
- Concurrent or planned participation in another interventional clinical trial during this study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 19 locations
1
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
2
Mount Sinai School of Medicine
New York, New York, United States, 10029
Actively Recruiting
3
Wake Forest University Baptist Health - Dept of Gastroenterology
Wake Forest, North Carolina, United States, 27587
Actively Recruiting
4
Temple University
Philadelphia, Pennsylvania, United States, 19121
Not Yet Recruiting
5
University of Texas Health - Ertan Digestive Disease Center
Houston, Texas, United States, 77030
Actively Recruiting
6
UZ Leuven
Leuven, Belgium
Not Yet Recruiting
7
University of Alberta Hospital
Edmonton, Canada
Not Yet Recruiting
8
Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre
Bordeaux, France
Not Yet Recruiting
9
AP-HP Hopital Bichat - Claude Bernard
Paris, France
Not Yet Recruiting
10
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
Milan, Italy
Not Yet Recruiting
11
Azienda Ospedaliero-Universitaria di Modena
Modena, Italy
Not Yet Recruiting
12
Haukeland University Hospital
Bergen, Norway
Not Yet Recruiting
13
Hospital Universitario 12 de Octubre
Madrid, Spain
Actively Recruiting
14
Consorcio Hospital General Universitario de Valencia
Valencia, Spain
Not Yet Recruiting
15
Gazi University Faculty of Medicine
Ankara, Turkey (Türkiye)
Not Yet Recruiting
16
University Hospital of Wales
Cardiff, United Kingdom
Actively Recruiting
17
Ninewells Hospital
Dundee, United Kingdom
Actively Recruiting
18
Guy's Hospital
London, United Kingdom
Actively Recruiting
19
Salford Royal Hospital
Manchester, United Kingdom
Actively Recruiting
Research Team
G
GondolaBio Inquiries
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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