EPP, XLP
Conditions
Brief summary
To evaluate the long-term safety and tolerability of oral dersimelagon.
Interventions
DRUGMT-7117
MT-7117
Sponsors
Tanabe Pharma America, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
Additional screening criteria check may apply for qualification: * 1\. Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided. * 2\. Subjects who have completed: MT-7117-G01 (completed through Week 58 \[Visit 12\]) or, MT-7117-A-302 (completed through Week 58 \[Visit 10\]) or, MT-7117-A-301 (completed EOT - Week 104 or Week 130) according to protocol amendment 1 or 2. * 3\. Subjects are willing and able to travel to the study sites for all scheduled visits. * 4\. In the Investigator's opinion, subject can understand the nature of the study and any risks involved in participation, and is willing to cooperate and comply with the protocol restrictions and requirements (including travel). * 5\. 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. * 6\. Female subjects of childbearing potential and male subjects with partner of childbearing potential must agree to use 2 effective methods of contraception including barrier method (especially for female subjects, one method must be highly effective method)
Exclusion criteria
Additional screening criteria check may apply for qualification: A subject will NOT be eligible for this study if ANY of the following criteria apply: * 1\. History or presence of photodermatoses other than EPP or XLP. * 2\. Presence of clinically significant hepatobiliary disease at Screening, determined as clinically significant by the Investigator. * 3\. Subjects with AST, ALT, ALP ≥ 3.0 × upper limit of normal (ULN) or TB \> 1.5 × ULN at Screening. The TB level of \> 1.5 × ULN listed in this
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]). | up to 66 further months | Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature), Clinical laboratory examinations (hematology, coagulation, biochemistry, urinalysis, and others), 12-lead electrocardiogram (ECG) parameters (Mean Heart Rate, PR Interval, QRS Duration, QT interval, QTcB and QTcF) will be assessed. |
| Number of patients with abnormal Physical examination data | up to 66 further months | Physical examination consists of assessment of abdominal, respiratory, cardiovascular, general appearance, and others. |
| Number of patients with Nevi appearance | up to 66 further months | — |
Countries
Australia, Belgium, Canada, Czechia, France, Germany, Italy, Japan, Netherlands, Norway, Poland, Spain, Sweden, United Kingdom, United States
Contacts
Primary ContactClinical Trials Information Desk, To prevent mis-communication,
Outcome results
None listed