Principal Investigator: Charles Parker
Keywords: erythropoietic protoporphyria , hematology Department: Hematology
IRB Number: 00112517
Specialty: Hematology/BMT, Hematology/BMT
Sub Specialties: Porphyria
Recruitment Status: Not yet recruiting

Contact Information

Janet Hood

Brief Summary

The goal of the study is to determine whether the study drug has an effect on increasing the symptom-free time in the sun and/or reducing the duration or intensity of symptoms associated with sun exposure.

Primary Objective

  • To investigate the efficacy and safety of MT-7117 on sunlight exposure duration and tolerance in subjects with EPP.

Secondary Objectives

  • To investigate the effect on time to onset and severity of prodromal symptoms in subjects with EPP
  • To investigate the effect on melanin density in subjects with EPP.
  • To assess the effect of treatment with MT-7117 on quality of life in subjects with EPP.
  • To investigate PK of MT-7117 in subjects with EPP.

Exploratory Objectives

  • To evaluate skin samples obtained by means of a pharmacodynamics skin biopsy for exploratory biomarkers related to pathogenesis of EPP, inflammatory response, and the mode of action of MT-7117.
  • To evaluate pharmacogenetics (PGx) including MC1R with single-nucleotide polymorphisms (SNPs), if applicable.
  • To evaluate porphyrin and protoporphyrin levels

Inclusion Criteria

A subject will be eligible for enrollment in the study if ALL of the following criteria apply:

  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

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.
  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 medical history or 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 squamous cell carcinoma, basal cell carcinoma, or other pre-malignant or 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 centrally acting analgesic agents including but not limited to opioids and opioid derivatives 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).
  22. Use of following drugs (including but not limited to) within 1 week of Randomization (Visit 2):

a. Drugs known to be predominantly metabolized by CYP3A4. (e.g., aprepitant, cyclosporine, pimozide, quinidine, and tacrolimus), for which elevated plasma concentrations are associated with significant medical events.
b. Drugs that are known substrates of P-gp, BCRP, organic anion transporter 3, OATP1B1, or OATP1B3 (e.g., rosuvastatin, pitavastatin, atorvastatin, telmisartan, valsartan, olmesartan; for which elevated plasma concentrations are associated with significant medical events)
c. Drugs known to inhibit P-gp, UGT, OATP1B1 or OATP1B3. (e.g. probenecid, valproic acid, amiodarone, captopril, clarithromycin, felodipine, verapamil, rifampin, gemfibrozil)
d. Drugs known to increase the gastric pH (e.g., omeprazole, lansoprazole, sodium hydrogen carbonate, aluminum hydroxide)