Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

To evaluate the long-term safety and tolerability of oral dersimelagon.

Study Overview

• Status: Recruiting
• Conditions: EPP; XLP
• Intervention / Treatment: Drug: MT-7117

• Study Type: Interventional
• Enrollment (Estimated): 301
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Clinical Trials Information Desk, To prevent mis-communication,; Phone Number: please e-mail; Email: information.US@mb.tanabe-pharma.com

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Not yet recruiting
      • Royal Prince Alfred Hospital
  • Queensland
    • Brisbane, Queensland, Australia, 4066
      • Recruiting
      • Wesley Medical Research
  • Victoria
    • Parkville, Victoria, Australia, 3050
      • Recruiting
      • Royal Melbourne Hospital (RMH)
• Belgium
  • Sophia, Belgium, 1000
    • Not yet recruiting
    • University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski
• Canada
  • Alberta
    • Edmonton, Alberta, Canada, Edmonton AB T6G 2G3
      • Recruiting
      • University of Alberta Hospital
• Czechia
  • Prague, Czechia, 140 21
    • Not yet recruiting
    • Institute for Clinical and Experimental Medicine - IKEM
• France
  • Bordeaux, France, 33000
    • Not yet recruiting
    • Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre
  • Colombes, France, 92700
    • Not yet recruiting
    • Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier
  • Nantes, France, 44000
    • Not yet recruiting
    • CHU Nantes
  • Paris, France, 75018
    • Not yet recruiting
    • Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine
• Germany
  • Berlin, Germany, 10117
    • Recruiting
    • Charité - Universitaetsmedizin Berlin
• Italy
  • Brescia BS, Italy, 25123
    • Recruiting
    • Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
  • Cuneo CN, Italy, 12100
    • Not yet recruiting
    • Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo
  • Genova GE, Italy, 16128
    • Not yet recruiting
    • Ospedalle Galliera
  • Milan, Italy, 20122
    • Recruiting
    • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
  • Modena, Italy, 41125
    • Active, not recruiting
    • U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
  • Rome, Italy, 5300144
    • Recruiting
    • IFO-San Gallicano IRCCS
  • Trieste TS, Italy, 34137
    • Not yet recruiting
    • Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica
• Japan
  • Hiroshima, Japan, 730-8670
    • Not yet recruiting
    • Mazda Hospital of Mazda Motor Corporation
  • Shizuoka, Japan, 431-3125
    • Not yet recruiting
    • Hamamatsu University Hospital
  • Hyōgo
    • Kobe, Hyōgo, Japan, 650-0017
      • Recruiting
      • Kobe University Hospital
  • Ishikawa-ken
    • Kanazawa, Ishikawa-ken, Japan, 921-8035
      • Completed
      • Sophia Dermatology Clinic
  • Osaka
    • Takatsuki, Osaka, Japan, 569-8686
      • Recruiting
      • Osaka Medical College Hospital
  • Tokyo
    • Minato-ku, Tokyo, Japan, 108-0073
      • Not yet recruiting
      • Tokyo Saiseikai Central Hospital
  • Toyama
    • Sugitani, Toyama, Japan, 930-0194
      • Recruiting
      • Toyama University Hospital
• Netherlands
  • Rotterdam, Netherlands, 3015 GD
    • Not yet recruiting
    • Erasmus MC, Universitair Medisch Centrum Rotterdam
• Norway
  • Bergen, Norway, N5021
    • Recruiting
    • Haukeland University Hospital
• Poland
  • Warsaw, Poland, 02-776
    • Not yet recruiting
    • Instytut Hematologii i Transfuzjologii
• Spain
  • Barcelona, Spain, 8036
    • Recruiting
    • Hospital Clinic de Barcelona
  • Madrid, Spain, 28041
    • Recruiting
    • Hospital Universitario
  • Valencia, Spain, 46014
    • Not yet recruiting
    • Hospital General Universitario de Valencia
• Sweden
  • Stockholm, Sweden, 14186
    • Recruiting
    • Karolinska University Hospital
• United Kingdom
  • London, United Kingdom, SE1 7EH
    • Recruiting
    • St. John's Institute of Dermatology-Guy's & St Thomas' NHS Foundation Trust
  • London, United Kingdom, SE1 9RT
    • Active, not recruiting
    • Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
  • Southampton, United Kingdom, SO16 6YD
    • Not yet recruiting
    • Southampton General Hospital - University Hospital Southampton NHS Foundation Trust
  • MN
    • Manchester, MN, United Kingdom, M6 8HD
      • Recruiting
      • Salford Royal NHS Foundation Trust
• United States
  • California
    • Huntington Beach, California, United States, 92647
      • Recruiting
      • Marvel Clinical Research, LLC
    • San Francisco, California, United States, 94143
      • Not yet recruiting
      • University of California at San Francisco - CSF Porphyria Center
  • Florida
    • Miami, Florida, United States, 33136
      • Not yet recruiting
      • University Of Miami School Of Medicine, Center For Liver Diseases
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Not yet recruiting
      • Massachusetts General Hospital
    • Brighton, Massachusetts, United States, 02135-3211
      • Recruiting
      • MetroBoston Clinical Partners, LLC
  • Michigan
    • Detroit, Michigan, United States, 48202
      • Not yet recruiting
      • Henry Ford Health System
  • Missouri
    • Kansas City, Missouri, United States, 64131
      • Recruiting
      • Kansas City Research Institute
  • New York
    • New York, New York, United States, 10029
      • Recruiting
      • Icahn School of Medicine at Mount Sinai (ISSMS)-The Mount Sinai Hospital (MSH)
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27157
      • Recruiting
      • Wake Forest University Baptist Health
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • Not yet recruiting
      • Cleveland Clinic - Taussig Cancer Institute
    • Columbus, Ohio, United States, 43215
      • Recruiting
      • Remington-Davis Clinical Research
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Recruiting
      • Thomas Jefferson University
    • Philadelphia, Pennsylvania, United States, 19141
      • Not yet recruiting
      • Einstein Medical Center, Philadelphia
  • Texas
    • Galveston, Texas, United States, 77555
      • Recruiting
      • The University of Texas Medical Branch (UTMB)
  • Washington
    • Seattle, Washington, United States, 98109
      • Recruiting
      • 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

Inclusion Criteria:

Additional screening criteria check may apply for qualification:

• 1. Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided.
• 2. Subjects who have completed: MT-7117-G01 (completed through Week 58 [Visit 12]) or, MT-7117-A-302 (completed through Week 58 [Visit 10]) or, MT-7117-A-301 (completed EOT - Week 104 or Week 130) according to protocol amendment 1 or 2.
• 3. Subjects are willing and able to travel to the study sites for all scheduled visits.
• 4. In the Investigator's opinion, subject can understand the nature of the study and any risks involved in participation, and is willing to cooperate and comply with the protocol restrictions and requirements (including travel).
• 5. 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.
• 6. Female subjects of childbearing potential and male subjects with partner of childbearing potential must agree to use 2 effective methods of contraception including barrier method (especially for female subjects, one method must be highly effective method)

Exclusion Criteria:

Additional screening criteria check may apply for qualification:

A subject will NOT be eligible for this study if ANY of the following criteria apply:

• 1. History or presence of photodermatoses other than EPP or XLP.
• 2. Presence of clinically significant hepatobiliary disease at Screening, determined as clinically significant by the Investigator.
• 3. Subjects with AST, ALT, ALP ≥ 3.0 × upper limit of normal (ULN) or TB > 1.5 × ULN at Screening. The TB level of > 1.5 × ULN listed in this exclusion criteria may not be applicable to subjects with a documented medical history of Gilbert's syndrome. Please consult with the Sponsor for eligibility of subjects with elevated levels due to Gilbert's syndrome.
• 4. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
• 5. History of melanoma.
• 6. 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.
• 7. 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.
• 8. 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/1.73m2 as calculated by the CKD-EPI creatinine equation (2009) for adults and by the Schwartz creatinine equation for adolescents (2009). MDRD can be used for adults per local recommendations.
• 9. 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.
• 10. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
• 11. Treatment with phototherapy or afamelanotide within 3 months before baseline (Visit 2 or Re-entry Visit 2).
• 12. Treatment with cimetidine or 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 baseline (Visit 2 or Re-entry Visit 2).
• 13. Chronic treatment with opioids, ketamine, or medical formulations or derivatives of cannabis within 4 weeks before baseline (Visit 2). Note: This exclusion criterion may not be applicable to subjects at Re-entry Visits. Acute use of scheduled analgesics more than 3 months before baseline (Visit 2) is allowed.
• 14. 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.
• 15. Previous treatment with any investigational agent other than dersimelagon within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).
• 16. History of any hypersensitivity to the active ingredient and/or excipients (lactose monohydrate, hydroxypropyl cellulose, carmellose calcium, magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, iron oxide red, and iron oxide black).
• 17. Subjects who are unable to swallow tablets or have diseases significantly affecting the gastrointestinal function such as malabsorption syndrome, resection of the stomach or small bowel, bariatric surgery procedures, symptomatic inflammatory bowel disease, or partial or complete bowel obstruction.

• 18. Using the following drugs (including but not limited to) within 1 week of baseline (Visit 2 or Re-entry Visit 2):

  1. Drugs known to be predominantly metabolized by cytochrome P450 (CYP) 3A4 with a narrow therapeutic index for which elevated plasma concentrations are associated with clinical safety concern or significant medical events.
  2. Drugs that are known substrates of P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), organic anion transporting polypeptide (OATP)1B1, or OATP1B3 for which elevated plasma concentrations are associated with significant medical events.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dersimelagon 200mg	Drug: MT-7117; MT-7117; Other Names: Dersimelagon

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]).	Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature), Clinical laboratory examinations (hematology, coagulation, biochemistry, urinalysis, and others), 12-lead electrocardiogram (ECG) parameters (Mean Heart Rate, PR Interval, QRS Duration, QT interval, QTcB and QTcF) will be assessed.	up to 66 further months
Number of patients with abnormal Physical examination data	Physical examination consists of assessment of abdominal, respiratory, cardiovascular, general appearance, and others.	up to 66 further months
Number of patients with Nevi appearance		up to 66 further months

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): August 10, 2021
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: July 28, 2021
• First Submitted That Met QC Criteria: August 6, 2021
• First Posted (Actual): August 16, 2021

Study Record Updates

• Last Update Posted (Actual): December 11, 2025
• Last Update Submitted That Met QC Criteria: December 4, 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-A-301; jRCT2041210146 (Registry Identifier: Japan Registry of Clinical Trials (jRCT)); 2021-001831-17 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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