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

NCT04402489 | Completed Phase 3 Results FDA Drug

• 3 other identifiers: MT-7117-G01jRCT20802253552019-004226-16
• Study Type: interventional
• Target: 184
• Locations: 10 countries
• Sites: 31

Brief Summary

The primary objective of this study is to investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP aged 12-75.

Conditions

EPP; XLP

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Average Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight Exposure Between 1 Hour Post Sunrise and 1 Hour Pre-sunset at Week 26 (Visit 7)

  From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)

Secondary Outcomes (5)

• Patient Global Impression of Change (PGIC) at Week 26

  Week 26

• Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.

  During the 26-week double-blind treatment period

• Change From Baseline for Total Score in the Domain of Pain Intensity in the PROMIS-57 at Week 26

  Baseline (Week 0) and Week 26

• The Percentage of Subjects Who Are Responders

  Week 26

• Change From Baseline for Total Score in the Domain of Physical Function in the PROMIS-57 at Week 26

  Baseline (Week 0) and Week 26

Study Arms (3)

Placebo Comparator

PLACEBO COMPARATOR

Oral tablet of placebo once a day.

Drug: Placebo

MT-7117 Low Dose

EXPERIMENTAL

Oral tablet of MT-7117 Low Dose once a day.

Drug: MT-7117 Low Dose

MT-7117 High Dose

EXPERIMENTAL

Oral tablet of MT-7117 High Dose once a day.

Drug: MT-7117 High Dose

Interventions

Placebo DRUG

Placebo

Placebo Comparator

MT-7117 Low Dose DRUG

MT-7117 Low Dose

Also known as: Dersimelagon

MT-7117 Low Dose

MT-7117 High Dose DRUG

MT-7117 High Dose

Also known as: Dersimelagon

MT-7117 High Dose

Eligibility Criteria

• Age: 12 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects provided written informed consent to participate. For minor subjects, both minor assent and parental consent will be provided.
• 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.
• Subjects have a body weight of ≥30 kg.
• Subjects are willing and able to travel to the study sites for all scheduled visits.
• 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).
• 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.
• 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.

You may not qualify if:

• History or presence of photodermatoses other than EPP or XLP.
• 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.
• Presence of clinically significant hepatobiliary disease based on LFT values at Screening.
• Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin \>1.5 × ULN at Screening.
• Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
• History of melanoma.
• Presence of melanoma and/or lesions suspicious for melanoma at Screening.
• History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
• 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.
• 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.
• 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.
• 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.
• Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
• Treatment with phototherapy within 3 months before Randomization (Visit 2).

Sponsors & Collaborators

• Tanabe Pharma America, Inc.: lead

Study Sites (31)

Marvel Clinical Research, LLC

Huntington Beach, California, 92647, United States

Location

University Of Miami School Of Medicine, Center For Liver Diseases

Miami, Florida, 33136, United States

Location

MetroBoston Clinical Partners, LLC

Brighton, Massachusetts, 02135, United States

Location

Kansas City Research Institute

Kansas City, Missouri, 64131, United States

Location

Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)

New York, New York, 10029, United States

Location

Wake Forest University Baptist Health

Winston-Salem, North Carolina, 27109, United States

Location

Remington-Davis Clinical Research

Columbus, Ohio, 43215, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

The University of Texas Medical Branch (UTMB)

Galveston, Texas, 77555, United States

Location

University of Washington-Seattle Cancer Care Alliance

Seattle, Washington, 19023, United States

Location

The Wesley Hospital

Brisbane, Queensland, 4066, Australia

Location

Royal Melbourne Hospital (RMH)

Melbourne, Victoria, 3050, Australia

Location

University of Alberta

Edmonton, Alberta, AB T6G 2R3, Canada

Location

Westfaelische Wilhelms-Universitaet Muenster

Münster, Northrhein Westalien, 48149, Germany

Location

Charite - Universitaetsmedizin Berlin

Berlin, 10117, Germany

Location

Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia

Brescia, 1 25123, Italy

Location

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano

Milan, 20122, Italy

Location

U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena

Modena, 41125, Italy

Location

I.F.O Hospital Centro Porfirie e Malattie Rare

Rome, 144 Rome RM, Italy

Location

Kobe University Hospital

Kobe, Hyōgo, Japan

Location

Sophia Dermatology Clinic

Kanazawa, Ishikawa-ken, 921-8035, Japan

Location

Investigator site

Sayama, Osaka, Japan

Location

Osaka Medical College Hospital

Takatsuki, Osaka, Japan

Location

Tokyo Saiseikai Central Hospital

Minato-ku, Tokyo, Japan

Location

Toyama University Hospital

Sugitani, Toyama, 930-0194, Japan

Location

Haukeland University Hospital

Bergen, Norway

Location

Hospital Clínic de Barcelona

Barcelona, 08036, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Karolinska University Hospital

Stockholm, 171 64, Sweden

Location

University of Manchester

Salford, Manchester, M13 9PL, United Kingdom

Location

Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust

London, SE1 7EH, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 39887900 DERIVED

Results Point of Contact

• Title: Clinical Trials, Information Desk
• Organization: Tanabe Pharma America, Inc.

information.US@mb.tanabe-pharma.com

Please E-mail

Study Officials

• Head of Medical Science

  Tanabe Pharma America, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 20, 2020
• First Posted: May 26, 2020
• Study Start: June 1, 2020
• Primary Completion: December 14, 2021
• Study Completion: July 26, 2022
• Last Updated: December 30, 2025
• Results First Posted: March 21, 2025

Record last verified: 2025-12

Locations

GB (2); ES (2); NO (1); DE (2); IT (4); JP (6); AU (2); US (10); CA (1); SE (1)