Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria

Completed · Phase 2 · Has a placebo group

Conditions studied: Erythropoietic Protoporphyria (EPP)

In brief

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.

Key facts

Study ID: NCT03520036
Run by: Tanabe Pharma America, Inc.
People needed: 102
Starts: 2018-07-05
Expected to finish: 2019-09-28
Last updated by the study team: 2025-12-31

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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).

You may not qualify if…

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).

Where it is running

ACTCA, A Member of the Alliance, Inc. — Los Angeles, California, United States
University of California at San Francisco — San Francisco, California, United States
University of Miami Miller School of Medicine — Miami, Florida, United States
Metro Boston Clinical Partners, LLC — Brighton, Massachusetts, United States
Ichan School of Medicine at Mount Sinai — New York, New York, United States
Wake Forest Baptist Medical Center — Winston-Salem, North Carolina, United States
Remington-Davis, Inc — Columbus, Ohio, United States
University of Texas Medical Branch Porphyria Center — Galveston, Texas, United States
University of Utah — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

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