Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Pā¦ (NCT04402489) | Clinical Trial Compass
CompletedPhase 3
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
United States184 participantsStarted 2020-06-01
Plain-language summary
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.
Who can participate
Age range12 Years ā 75 Years
SexALL
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Inclusion criteria
ā. Subjects provided written informed consent to participate. For minor subjects, both minor assent and parental consent will be provided.
ā. Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history, aged 12 years to 75 years, inclusive, at Screening.
ā. Subjects have a body weight of ā„30 kg.
ā. Subjects are willing and able to travel to the study sites for all scheduled visits.
ā. In the Investigator's opinion, subject is able to understand the nature of the study and any risks involved in participation, and willing to cooperate and comply with the protocol restrictions and requirements (including travel).
ā. Female subjects who are non-lactating and have a negative urine pregnancy test at baseline visit prior to receiving the first dose of study drug.
ā. Female subjects of childbearing potential and male subjects with partner of child-bearing potential currently using/willing to use 2 effective methods of contraception including barrier method as described in the protocol.
Exclusion criteria
ā. History or presence of photodermatoses other than EPP or XLP.
ā. Subjects who are unwilling or unable to go outside during daylight hours most days (e.g., between 1 hour post sunrise and 1 hour pre-sunset) during the study.
ā. Presence of clinically significant hepatobiliary disease based on LFT values at Screening.
What they're measuring
1
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)
Timeframe: From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)