This is a Phase 2, multi-center, double-blind, placebo-controlled, parallel group study of bitopertin to evaluate the safety, tolerability, efficacy, and PPIX concentration change in participants with EPP. Participants may roll over to an open label extension portion after completing the double-blind treatment period.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
75
University of Alabama Hospital
Birmingham, Alabama, United States
University of California San Francisco
San Francisco, California, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
Percent Change From Baseline in Whole Blood Metal-free PPIX Levels
Percent change from baseline in PPIX concentration was analyzed using a mixed model repeated measures analysis in the ITT population.
Time frame: 121 days
Total Hours of Sunlight Exposure to Skin on Days With no Pain From 1000 to 1800 Hours (10:00am to 6:00pm)
Cumulative total hours of sunlight exposure on days with no pain from 1000 to 1800 hours from baseline to Day 121 (EOS) was analyzed using analysis of variance in the ITT population.
Time frame: 121 days
Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight Exposure Between 1 Hour Post-sunrise and 1 Hour Pre-sunset
Participants exposed their skin to sunlight once a week and measured the time it takes to experience a prodrome. The time (minutes) to first prodromal symptom (e.g., burning, tingling, itching, or stinging) associated with sunlight exposure was recorded in a diary. This sun exposure challenge was performed weekly. The average time (minutes) to first prodromal symptom (e.g., burning, tingling, itching, or stinging) associated with sunlight exposure was averaged over two-week intervals through Study Day 121.
Time frame: 121 days
Total Pain Intensity
The maximum total daily pain intensity scores of phototoxic reactions over the entire treatment period (D1-D121). The maximum pain score of a phototoxic reaction was measured on a Likert Scale (0-10). Total scores range from 0-1210. A score of "0" is the best outcome; and higher scores are a worse outcome. The maximum pain values on a scale of 0-10 in a day were summed across 121 days.
Time frame: Sum of Day 1 to Day 121
Incidence of Treatment-emergent Adverse Events
Incidence of treatment-emergent adverse events
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Massachusetts General Hospital
Boston, Massachusetts, United States
Mount Sinai Hospital
New York, New York, United States
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, United States
Einstein Medical Center
Philadelphia, Pennsylvania, United States
University of Texas
Galveston, Texas, United States
Fred Hutchinson Cancer Center
Seattle, Washington, United States
Time frame: 121 days
Erythrocyte Total PPIX Concentrations
Percent change from baseline in erythrocyte total PPIX concentration was summarized by analysis visit in the ITT population.
Time frame: 121 days
Plasma Total PPIX Concentrations
Percent change from baseline in plasma total PPIX concentration was summarized by analysis visit in the ITT population.
Time frame: 121 days
Whole Blood Total PPIX Concentrations
Percent change from baseline in whole blood total PPIX concentration was summarized by analysis visit in the ITT population.
Time frame: 121 days
Plasma Bitopertin Concentrations
Day 29 plasma bitopertin concentrations, 4 hours post-dose
Time frame: Day 29