Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
NCT ID: NCT04402489
Last Updated: 2025-12-30
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
184 participants
INTERVENTIONAL
2020-06-01
2022-07-26
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
TRIPLE
Study Groups
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Placebo Comparator
Oral tablet of placebo once a day.
Placebo
Placebo
MT-7117 Low Dose
Oral tablet of MT-7117 Low Dose once a day.
MT-7117 Low Dose
MT-7117 Low Dose
MT-7117 High Dose
Oral tablet of MT-7117 High Dose once a day.
MT-7117 High Dose
MT-7117 High Dose
Interventions
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Placebo
Placebo
MT-7117 Low Dose
MT-7117 Low Dose
MT-7117 High Dose
MT-7117 High Dose
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history, aged 12 years to 75 years, inclusive, at Screening.
3. Subjects have a body weight of ≥30 kg.
4. Subjects are willing and able to travel to the study sites for all scheduled visits.
5. 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).
6. 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.
7. Female subjects of childbearing potential and male subjects with partner of child-bearing potential currently using/willing to use 2 effective methods of contraception including barrier method as described in the protocol.
Exclusion Criteria
2. Subjects who are unwilling or unable to go outside during daylight hours most days (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 of melanoma.
7. Presence of melanoma and/or lesions suspicious for melanoma at Screening.
8. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
9. Presence of squamous cell carcinoma, basal cell carcinoma, or other malignant skin lesions.
Any suspicious lesions or nevi will be evaluated. If the suspicious lesion or nevi cannot be resolved through biopsy or excision, the subject will be excluded from the study.
10. 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.
11. Presence of clinically significant acute or chronic renal disease based upon the subject's medical records including hemodialysis; an estimated glomerular filtration rate (eGFR) \<60 mL/min as calculated by the Chronic Kidney Disease-Epidemiology Collaboration (CKDEPI) creatinine equation (2009) for adults and by the Schwartz creatinine equation for adolescents (2009). Modification of Diet in Renal Disease (MDRD) can be used for adults per local recommendations.
12. 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.
13. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
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 within 4 weeks before Randomization (Visit 2), at doses which, in the opinion of the Investigator, may affect study endpoints (including but not limited to beta-carotene, cysteine, pyridoxine).
18. Chronic treatment with any scheduled analgesic agents including, but not limited to, opioids and opioid derivatives such as morphine, hydrocodone, oxycodone, fentanyl, or their combination with other unscheduled analgesics or non-steroidal anti-inflammatory drug (Percocet and Vicodin-like prescription drugs) within 4 weeks before Randomization (Visit 2).
Acute use of scheduled narcotics greater than 3 months prior to randomization, OTCs, such as NSAIDs or aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of screening are not excluded.
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 (this does not include placebo treated subjects).
21. Previous treatment with any investigational agent within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).
12 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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Marvel Clinical Research, LLC
Huntington Beach, California, United States
University Of Miami School Of Medicine, Center For Liver Diseases
Miami, Florida, United States
MetroBoston Clinical Partners, LLC
Brighton, Massachusetts, United States
Kansas City Research Institute
Kansas City, Missouri, United States
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
New York, New York, United States
Wake Forest University Baptist Health
Winston-Salem, North Carolina, United States
Remington-Davis Clinical Research
Columbus, Ohio, United States
Thomas Jefferson University
Philadelphia, Pennsylvania, United States
The University of Texas Medical Branch (UTMB)
Galveston, Texas, United States
University of Washington-Seattle Cancer Care Alliance
Seattle, Washington, United States
The Wesley Hospital
Brisbane, Queensland, Australia
Royal Melbourne Hospital (RMH)
Melbourne, Victoria, Australia
University of Alberta
Edmonton, Alberta, Canada
Westfaelische Wilhelms-Universitaet Muenster
Münster, Northrhein Westalien, Germany
Charite - Universitaetsmedizin Berlin
Berlin, , Germany
Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
Brescia, , Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan, , Italy
U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
Modena, , Italy
I.F.O Hospital Centro Porfirie e Malattie Rare
Rome, , Italy
Kobe University Hospital
Kobe, Hyōgo, Japan
Sophia Dermatology Clinic
Kanazawa, Ishikawa-ken, Japan
Investigator site
Sayama, Osaka, Japan
Osaka Medical College Hospital
Takatsuki, Osaka, Japan
Tokyo Saiseikai Central Hospital
Minato-ku, Tokyo, Japan
Toyama University Hospital
Sugitani, Toyama, Japan
Haukeland University Hospital
Bergen, , Norway
Hospital Clínic de Barcelona
Barcelona, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Karolinska University Hospital
Stockholm, , Sweden
University of Manchester
Salford, Manchester, United Kingdom
Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
London, , United Kingdom
Countries
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References
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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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jRCT2080225355
Identifier Type: REGISTRY
Identifier Source: secondary_id
2019-004226-16
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
MT-7117-G01
Identifier Type: -
Identifier Source: org_study_id