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: Active, not recruiting
• Conditions: EPP; XLP
• Intervention / Treatment: Drug: MT-7117

• Study Type: Interventional
• Enrollment (Estimated): 151
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Queensland
    • Brisbane, Queensland, Australia, 4066
      • Wesley Medical Research
  • Victoria
    • Parkville, Victoria, Australia, 3050
      • Royal Melbourne Hospital (RMH)
• Canada
  • Alberta
    • Edmonton, Alberta, Canada, Edmonton AB T6G 2G3
      • University of Alberta Hospital
• Germany
  • Berlin, Germany, 10117
    • Charite - Universitaetsmedizin Berlin
• Italy
  • Brescia BS, Italy, 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, 5300144
    • IFO-San Gallicano IRCCS
• Japan
  • Hyogo
    • Kobe, Hyogo, Japan, 650-0017
      • Kobe University Hospital
  • Ishikawa
    • Kanazawa, Ishikawa, Japan, 921-8035
      • Sophia Dermatology Clinic
  • Osaka
    • Takatsuki, Osaka, Japan, 569-8686
      • Osaka Medical College Hospital
  • Tokyo
    • Minato-ku, Tokyo, Japan, 108-0073
      • Tokyo Saiseikai Central Hospital
  • Toyama
    • Sugitani, Toyama, Japan, 930-0194
      • Toyama University Hospital
• Norway
  • Bergen, Norway, N5021
    • Haukeland University Hospital
• Spain
  • Barcelona, Spain, 8036
    • Hospital Clinic de Barcelona
  • Madrid, Spain, 28041
    • Hospital Universitario
• Sweden
  • Stockholm, Sweden, 14186
    • Karolinska University Hospital
• United Kingdom
  • London, United Kingdom, SE1 9RT
    • Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
  • MN
    • Manchester, MN, United Kingdom, M6 8HD
      • Salford Royal NHS Foundation Trust
• United States
  • California
    • Huntington Beach, California, United States, 92647
      • Marvel Clinical Research, LLC
  • Massachusetts
    • Brighton, Massachusetts, United States, 02135-3211
      • 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 (ISSMS)-The Mount Sinai Hospital (MSH)
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27157
      • 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, 98109
      • 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:

• 1. Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided.
• 2. Subjects who complete MT-7117-G01 (complete through Week 58 [Visit 12])
• 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 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).
• 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 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:

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 or history of any hepatobiliary disease at Screening, determined as clinically significant by the Investigator after the discussion with the Sponsor Medical Monitor.
• 3. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
• 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 melanoma and/or lesions suspicious for melanoma at Screening.
• 7. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
• 8. 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.
• 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; 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.
• 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. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
• 13. Treatment with phototherapy or afamelanotide within 3 months before baseline (Visit 2).
• 14. 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).
• 15. 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 baseline (Visit 2). Acute use of scheduled narcotics greater than 3 months prior to baseline, over-the-counter medications (OTCs), such as non-steroidal anti-inflammatory drugs (NSAIDs) or aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of baseline (Visit 2) are not excluded.
• 16. 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.
• 17. 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).

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 30 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 30 further months
Number of patients with Nevi appearance		up to 30 further months

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): August 10, 2021
• Primary Completion (Estimated): June 1, 2025
• Study Completion (Estimated): June 1, 2025

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): March 13, 2024
• Last Update Submitted That Met QC Criteria: March 12, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Metabolic Diseases; Skin Diseases; Liver Diseases; Genetic Diseases, Inborn; 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