The primary objective of this study is to investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP aged 12-75.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
184
Change From Baseline in Average 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 at Week 26 (Visit 7)
Time frame: From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)
Patient Global Impression of Change (PGIC) at Week 26
PGIC: Scale from 1 to 7, where 7 is worse.
Time frame: Week 26
Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.
The Likert scale used ranges from 0 to 10, where 0 indicates the lowest pain rating and 10 indicates the highest pain rating. Likewise, 0 indicates to best outcome and 10 indicates the worst outcome. The sum of the number of pain events with pain rating of 1 to 10 for the day is used as the number of sunlight-induced pain events in the day. The sum of the number of the pain events with pain rating of 1 to 10 in each day during the 26-week Double-blind Treatment Period is calculated as this endpoint.
Time frame: During the 26-week double-blind treatment period
Change From Baseline for Total Score in the Domain of Pain Intensity in the PROMIS-57 at Week 26
Pain intensity: 0 to 10, where 10 is worst pain imaginable.
Time frame: Baseline (Week 0) and Week 26
The Percentage of Subjects Who Are Responders
The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post-sunrise and 1 hour presunset defined by within-subject meaningful change of 66 minutes increase from baseline to Week 26
Time frame: Week 26
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Marvel Clinical Research, LLC
Huntington Beach, California, United States
University Of Miami School Of Medicine, Center For Liver Diseases
Miami, Florida, United States
MetroBoston Clinical Partners, LLC
Brighton, Massachusetts, United States
Kansas City Research Institute
Kansas City, Missouri, United States
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
New York, New York, United States
Wake Forest University Baptist Health
Winston-Salem, North Carolina, United States
Remington-Davis Clinical Research
Columbus, Ohio, United States
Thomas Jefferson University
Philadelphia, Pennsylvania, United States
The University of Texas Medical Branch (UTMB)
Galveston, Texas, United States
University of Washington-Seattle Cancer Care Alliance
Seattle, Washington, United States
...and 21 more locations
Change From Baseline for Total Score in the Domain of Physical Function in the PROMIS-57 at Week 26
Physical function: 1-5, where 5 is without any difficulty.
Time frame: Baseline (Week 0) and Week 26