The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration without symptoms and tolerance in subjects with EPP.
This is a Phase 2, randomized, double-blind, placebo controlled study to assess the efficacy, tolerability, and safety of MT-7117 in subjects with EPP. The study consists of a 2 week screening period, a 16 week double-blind treatment period, and a 6 week follow-up period at Week 22. The total participation period is approximately 24 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
102
MT-7117 low dose QD, oral, 16 weeks
MT-7117 high dose QD, oral, 16 weeks
Placebo QD, oral, 16 weeks
ACTCA, A Member of the Alliance, Inc.
Los Angeles, California, United States
University of California at San Francisco
San Francisco, California, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
Change From Baseline in Average Daily Time (Minutes) to First Prodromal Symptom Associated With Sunlight Exposure Between Hour Post Sunrise and 1 Hour Pre-Sunset at Week 16.
Duration in minutes, of sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset. The average Duration in minutes, of sunlight exposure before the first prodromal symptom between 1 hour post sunrise and 1 hour pre-sunset. The average duration means that average of daily durations in 14-day windows before Day 1 (or week 16 Day) applied.
Time frame: Baseline (Week 0) and Week 16
Change From Baseline in Average Daily Duration (Minutes) of Sunlight Exposure Between 1 Hour Post Sunrise and 1 Hour Pre-Sunset Without Prodromal Symptoms at Week 16
Change from baseline to week 16 in Average Daily Duration (Minutes) of Sunlight Exposure sums any sunlight exposure time excluding any overlapped time with prodromal symptoms, including if the patients go out multiple times on the same day after the prodromal symptom had previously ended.
Time frame: Baseline (Week 0), and Week 16
Change From Baseline in Average Daily Mean Duration (Minutes) of Sunlight Exposure Between 1 Hour Post Sunrise and 1 Hour Pre-Sunset Without Prodromal Symptoms at Week 16
Change from baseline to week 16 in Average Daily Mean Duration (Minutes) of Sunlight Exposure without prodromal symptoms divided by the number of sunlight exposures periods applicable that day.
Time frame: Baseline (Week 0), and Week 16
Total Number of Sunlight Exposure Episodes With Prodromal Symptoms During 16-Week Double-Blind Treatment Period
Time frame: Week 16
Change From Baseline in Average Daily Mean Intensity of Prodromal Symptoms During 16-week Double-blind Treatment Period in 11-point Likert Scale
The Intensity of Prodromal Symptoms is measured by 11-point Likert scale ranges from 0 (no symptom) to 10 (greatest severity of symptom).
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Metro Boston Clinical Partners, LLC
Brighton, Massachusetts, United States
Ichan School of Medicine at Mount Sinai
New York, New York, United States
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, United States
Remington-Davis, Inc
Columbus, Ohio, United States
University of Texas Medical Branch Porphyria Center
Galveston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Time frame: Baseline (Week 0), and Week 16
Change From Baseline in Average Daily Duration (Minutes) of Prodromal Symptoms at 16-Week Double-Blind Treatment Period
Time frame: Baseline (Week 0), and Week 16
Change From Baseline in Pigmentation as Measured by Melanin Density for Average of 6 Skin Segments at Week 8 and Week 16.
Pigmentation will be assessed in melanin density which are numeric scores measured by spectrophotometer on 6 skin segments (forehead, left cheek, right inside upper arm, left medial forearm, right-hand side of abdomen, and left-hand side of buttock).
Time frame: Baseline (Week 0), Week 8, and Week 16
Percent Change From Baseline in Pigmentation as Measured by Melanin Density for Average of 6 Skin Segments at Week 8 and Week 16.
Time frame: Baseline (Week 0), Week 8, and Week 16
Total Number of Pain Events During 16-Week Double-Blind Treatment Period
Time frame: Week 16