Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
NCT ID: NCT03520036
Last Updated: 2025-12-31
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
102 participants
INTERVENTIONAL
2018-07-05
2019-09-28
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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MT-7117 low dose
MT-7117 low dose
MT-7117 low dose QD, oral, 16 weeks
MT-7117 high dose
MT-7117 high dose
MT-7117 high dose QD, oral, 16 weeks
Placebo
Placebo
Placebo QD, oral, 16 weeks
Interventions
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MT-7117 low dose
MT-7117 low dose QD, oral, 16 weeks
MT-7117 high dose
MT-7117 high dose QD, oral, 16 weeks
Placebo
Placebo QD, oral, 16 weeks
Eligibility Criteria
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Inclusion Criteria
* 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
* 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).
18 Years
75 Years
ALL
No
Sponsors
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Tanabe Pharma America, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Head of Medical Science
Role: STUDY_DIRECTOR
Tanabe Pharma America, Inc.
Locations
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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
Countries
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References
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Balwani M, Bonkovsky HL, Levy C, Anderson KE, Bissell DM, Parker C, Takahashi F, Desnick RJ, Belongie K; Endeavor Investigators. Dersimelagon in Erythropoietic Protoporphyrias. N Engl J Med. 2023 Apr 13;388(15):1376-1385. doi: 10.1056/NEJMoa2208754.
Ogasawara A, Ide R, Inoue S, Tsuda M, Teng R. Assessment of Potential Drug-Drug Interactions for Novel Oral Melanocortin-1 Receptor Agonist Dersimelagon. Pharmacol Res Perspect. 2025 Feb;13(1):e70069. doi: 10.1002/prp2.70069.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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MT-7117-A01
Identifier Type: -
Identifier Source: org_study_id