Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
Condition: Erythropoietic Protoporphyria (EPP)
MeSH terms: Protoporphyria, Erythropoietic
Intervention: MT-7117 low dose; MT-7117 high dose; Placebo
Intervention description: MT-7117 low dose QD, oral, 16 weeks; MT-7117 high dose QD, oral, 16 weeks; Placebo QD, oral, 16 weeks
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 102.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No

Summary:

      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.
    

Eligibility criteria:

        Additional screening criteria check may apply for qualification.

        Inclusion Criteria:

          -  1. Subjects provided written informed consent to participate.

          -  2. Male and female subjects with a confirmed diagnosis of EPP based on medical
             history, aged 18 years to 75 years, inclusive, at Screening.

          -  3. Subjects are willing and able to travel to the study sites for all scheduled
             visits.

          -  4. 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).

        Exclusion Criteria:

          -  1. History or presence of photodermatoses other than EPP.

          -  2. Subjects who are unwilling or unable to go outside during daylight hours (e.g.,
             between 1 hour post sunrise and 1 hour pre-sunset) during the study.

          -  3. Presence of clinically significant hepatobiliary disease based on LFT values at
             Screening.

          -  4. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin
             >1.5 × ULN at Screening.

          -  5. Subjects with or having a history (in the last 2 years) of excessive alcohol intake
             in the opinion of the Investigator.

          -  6. History or presence of melanoma and/or atypical nevus at Screening.

          -  7. History of familial melanoma (defined as having 2 or more first-degree relatives,
             such as parents, sibling and/or child).

          -  8. History or presence of pre-malignant skin lesion squamous cell carcinoma, basal
             cell carcinoma, or other malignant skin lesions.

          -  9. History or presence of psychiatric disease judged to be clinically significant by
             the Investigator and which may interfere with the study evaluation and/or safety of
             the subjects.

          -  10. Presence of clinically significant acute or chronic renal disease based upon the
             subject's medical records including hemodialysis; and a serum creatinine level of
             greater than 1.2 mg/dL or a glomerular filtration rate (GFR) <60 ml/min.

          -  11. Presence of any clinically significant disease or laboratory abnormality which, in
             the opinion of the Investigator, can interfere with the study objectives and/or safety
             of the subjects.

          -  12. Pregnancy or lactation.

          -  13. Females of child bearing potential and male subjects with partners of
             child-bearing potential unwilling to use adequate contraception measures as described
             in the protocol.

          -  14. Treatment with phototherapy within 3 months before Randomization (Visit 2).

          -  15. Treatment with afamelanotide within 3 months before Randomization (Visit 2).

          -  16. Treatment with cimetidine within 4 weeks before Randomization (Visit 2).

          -  17. Treatment with antioxidant agents at doses which, in the opinion of the
             Investigator, may affect study endpoints (including but not limited to beta-carotene,
             cysteine, pyridoxine) within 4 weeks before Randomization (Visit 2).

          -  18. Chronic treatment with prescription-based analgesic agents including but not
             limited to opioids and opioid derivatives such as morphine, hydrocodone, oxycodone or
             their combination with other analgesics or non-steroidal anti-inflammatory drug
             (NSAID, as Percocet and Vicodin-like prescription drugs) within 4 weeks before
             Randomization (Visit 2).

          -  19. Treatment with any drugs or supplements which, in the opinion of the Investigator,
             can interfere with the objectives of the study or safety of the subjects.

          -  20. Previous exposure to MT 7117.

          -  21. Previous treatment with any investigational agent within 12 weeks before Screening
             OR 5 half-lives of the investigational product (whichever is longer).