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

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria

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.

Study Overview

• Status: Completed
• Conditions: EPP; XLP
• Intervention / Treatment: Drug: Placebo; Drug: MT-7117 Low Dose; Drug: MT-7117 High Dose

• Study Type: Interventional
• Enrollment (Actual): 184
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Queensland
    • Brisbane, Queensland, Australia, 4066
      • The Wesley Hospital
  • Victoria
    • Melbourne, Victoria, Australia, 3050
      • Royal Melbourne Hospital (RMH)
• Canada
  • Alberta
    • Edmonton, Alberta, Canada, AB T6G 2R3
      • University of Alberta
• Germany
  • Berlin, Germany, 10117
    • Charite - Universitaetsmedizin Berlin
  • Northrhein Westalien
    • Münster, Northrhein Westalien, Germany, 48149
      • Westfaelische Wilhelms-Universitaet Muenster
• Italy
  • Brescia, Italy, 1 25123
    • Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
  • Milan, Italy, 20122
    • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
  • Modena, Italy, 41125
    • U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
  • Rome, Italy, 144 Rome RM
    • I.F.O Hospital Centro Porfirie e Malattie Rare
• Japan
  • Hyōgo
    • Kobe, Hyōgo, Japan
      • Kobe University Hospital
  • Ishikawa-ken
    • Kanazawa, Ishikawa-ken, Japan, 921-8035
      • Sophia Dermatology Clinic
  • Osaka
    • Sayama, Osaka, Japan
      • Investigator Site
    • Takatsuki, Osaka, Japan
      • Osaka Medical College Hospital
  • Tokyo
    • Minato-ku, Tokyo, Japan
      • Tokyo Saiseikai Central Hospital
  • Toyama
    • Sugitani, Toyama, Japan, 930-0194
      • Toyama University Hospital
• Norway
  • Bergen, Norway
    • Haukeland University Hospital
• Spain
  • Barcelona, Spain, 08036
    • Hospital Clinic De Barcelona
  • Madrid, Spain, 28041
    • Hospital Universitario 12 de Octubre
• Sweden
  • Stockholm, Sweden, 171 64
    • Karolinska University Hospital
• United Kingdom
  • London, United Kingdom, SE1 7EH
    • Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
  • Manchester
    • Salford, Manchester, United Kingdom, M13 9PL
      • University of Manchester
• United States
  • California
    • Huntington Beach, California, United States, 92647
      • Marvel Clinical Research, LLC
  • Florida
    • Miami, Florida, United States, 33136
      • University Of Miami School Of Medicine, Center For Liver Diseases
  • Massachusetts
    • Brighton, Massachusetts, United States, 02135
      • MetroBoston Clinical Partners, LLC
  • Missouri
    • Kansas City, Missouri, United States, 64131
      • Kansas City Research Institute
  • New York
    • New York, New York, United States, 10029
      • Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27109
      • Wake Forest University Baptist Health
  • Ohio
    • Columbus, Ohio, United States, 43215
      • Remington-Davis Clinical Research
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Thomas Jefferson University
  • Texas
    • Galveston, Texas, United States, 77555
      • The University of Texas Medical Branch (UTMB)
  • Washington
    • Seattle, Washington, United States, 19023
      • University of Washington-Seattle Cancer Care Alliance

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Additional screening criteria check may apply for qualification.

Inclusion Criteria:

1. Subjects provided written informed consent to participate. For minor subjects, both minor assent and parental consent will be provided.
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:

1. History or presence of photodermatoses other than EPP or XLP.
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).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo Comparator; Oral tablet of placebo once a day.	Drug: Placebo; Placebo
Experimental: MT-7117 Low Dose; Oral tablet of MT-7117 Low Dose once a day.	Drug: MT-7117 Low Dose; MT-7117 Low Dose; Other Names: Dersimelagon
Experimental: MT-7117 High Dose; Oral tablet of MT-7117 High Dose once a day.	Drug: MT-7117 High Dose; MT-7117 High Dose; Other Names: Dersimelagon

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient Global Impression of Change (PGIC) at Week 26	PGIC: Scale from 1 to 7, where 7 is worse.	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.	The Likert scale used ranges from 0 to 10, where 0 indicates the lowest pain rating and 10 indicates the highest pain rating. Likewise, 0 indicates to best outcome and 10 indicates the worst outcome. The sum of the number of pain events with pain rating of 1 to 10 for the day is used as the number of sunlight-induced pain events in the day. The sum of the number of the pain events with pain rating of 1 to 10 in each day during the 26-week Double-blind Treatment Period is calculated as this endpoint.	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	Pain intensity: 0 to 10, where 10 is worst pain imaginable.	Baseline (Week 0) and Week 26
The Percentage of Subjects Who Are Responders	The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post-sunrise and 1 hour presunset defined by within-subject meaningful change of 66 minutes increase from baseline to Week 26	Week 26
Change From Baseline for Total Score in the Domain of Physical Function in the PROMIS-57 at Week 26	Physical function: 1-5, where 5 is without any difficulty.	Baseline (Week 0) and Week 26

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline for total score in the domain of pain intensity in the PROMIS-57.	Pain intensity: 0 to 10, where 10 is worst pain imaginable.	Baseline (Week 0) and Week 26
The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset defined by within-subject meaningful change.		Week 26
Change from baseline for total score in the domain of physical function in the PROMIS-57.	Physical function: 1-5, where 5 is without any difficulty.	Baseline (Week 0) and Week 26

Collaborators and Investigators

• Sponsor: Tanabe Pharma America, Inc.
• Investigators: Study Director: Head of Medical Science, Tanabe Pharma America, Inc.

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2020
• Primary Completion (Actual): December 14, 2021
• Study Completion (Actual): July 26, 2022

Study Registration Dates

• First Submitted: May 20, 2020
• First Submitted That Met QC Criteria: May 20, 2020
• First Posted (Actual): May 26, 2020

Study Record Updates

• Last Update Posted (Actual): December 30, 2025
• Last Update Submitted That Met QC Criteria: December 12, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Metabolic Diseases; Digestive System Diseases; Liver Diseases; Skin Diseases; Skin Diseases, Genetic; Porphyrias, Hepatic; Porphyrias; Protoporphyria, Erythropoietic
• Other Study ID Numbers: MT-7117-G01; jRCT2080225355 (Registry Identifier: Japan Registry of Clinical Trials (jRCT)); 2019-004226-16 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No