The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older. The main questions it aims to answer are: * Whether bitopertin increases pain-free sunlight exposure after 6 months of treatment in participants with EPP or XLP. * How PPIX concentration levels change from before bitopertin treatment to after 6 months of treatment. Researchers will compare bitopertin to a placebo look-alike substance that contains no drug. Participants will complete daily questionnaires and attend study visits for assessments.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
183
Oral dose, once a day for 24 weeks
Oral dose, once a day for 24 weeks
Marvel Clinical Research
Huntington Beach, California, United States
University of California San Francisco
San Francisco, California, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
MetroBoston Clinical Partners
Boston, Massachusetts, United States
Average monthly total time in sunlight on days without pain from a phototoxic reaction between 10:00 to 18:00 (10:00 AM to 6:00 PM) after 6 months (24 weeks) of treatment
Time frame: 24 weeks
Percent change from baseline in whole-blood metal-free PPIX levels at 6 months
Time frame: 24 weeks
Safety and tolerability, as assessed by adverse events (AEs) and laboratory results, over the 6-month treatment period
Time frame: 24 weeks
Occurrence of phototoxic reactions over the 6-month treatment period
Time frame: 24 weeks
Cumulative total time in sunlight on days without pain from a phototoxic reaction between 10:00 to 18:00 (10:00 AM to 6:00 PM) over the 6-month (24-week) treatment period
Time frame: 24 weeks
Change from baseline in 2-week average daily sunlight exposure time (minutes) to first prodromal symptom (eg, burning, tingling, itching, or stinging) associated with sunlight exposure between 1 hour post-sunrise and 1 hour pre-sunset at 6 months
Time frame: 24 weeks
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Henry Ford Health System
Detroit, Michigan, United States
Mount Sinai Hospital
New York, New York, United States
Wake Forest University
Winston-Salem, North Carolina, United States
Remington-Davis Clinical Research
Columbus, Ohio, United States
University of Texas Medical Branch
Galveston, Texas, United States
...and 17 more locations