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

NCT05005975 | Recruiting Phase 3 FDA Drug

• 3 other identifiers: MT-7117-A-301jRCT20412101462021-001831-17
• Study Type: interventional
• Target: 301
• Locations: 15 countries
• Sites: 51

Brief Summary

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

Conditions

EPP; XLP

Outcome Measures

Primary Outcomes (3)

• 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

Study Arms (1)

Dersimelagon 200mg

EXPERIMENTAL

Drug: MT-7117

Interventions

MT-7117 DRUG

MT-7117

Also known as: Dersimelagon

Dersimelagon 200mg

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:

• Additional screening criteria check may apply for qualification:
• \. Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided.
• \. 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.
• \. Subjects are willing and able to travel to the study sites for all scheduled visits.
• \. 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).
• \. 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 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)

You may not qualify if:

• Additional screening criteria check may apply for qualification:
• A subject will NOT be eligible for this study if ANY of the following criteria apply:
• \. History or presence of photodermatoses other than EPP or XLP.
• \. Presence of clinically significant hepatobiliary disease at Screening, determined as clinically significant by the Investigator.
• \. 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 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/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.
• \. 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 or afamelanotide within 3 months before baseline (Visit 2 or Re-entry Visit 2).
• \. 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).
• \. 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.
• \. 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).

Sponsors & Collaborators

• Tanabe Pharma America, Inc.: lead

Study Sites (51)

Marvel Clinical Research, LLC

Huntington Beach, California, 92647, United States

RECRUITING

University of California at San Francisco - CSF Porphyria Center

San Francisco, California, 94143, United States

NOT YET RECRUITING

University Of Miami School Of Medicine, Center For Liver Diseases

Miami, Florida, 33136, United States

NOT YET RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

NOT YET RECRUITING

MetroBoston Clinical Partners, LLC

Brighton, Massachusetts, 02135-3211, United States

RECRUITING

Henry Ford Health System

Detroit, Michigan, 48202, United States

NOT YET RECRUITING

Kansas City Research Institute

Kansas City, Missouri, 64131, United States

RECRUITING

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

New York, New York, 10029, United States

RECRUITING

Wake Forest University Baptist Health

Winston-Salem, North Carolina, 27157, United States

RECRUITING

Cleveland Clinic - Taussig Cancer Institute

Cleveland, Ohio, 44106, United States

NOT YET RECRUITING

Remington-Davis Clinical Research

Columbus, Ohio, 43215, United States

RECRUITING

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

Einstein Medical Center, Philadelphia

Philadelphia, Pennsylvania, 19141, United States

NOT YET RECRUITING

The University of Texas Medical Branch (UTMB)

Galveston, Texas, 77555, United States

RECRUITING

University of Washington-Seattle Cancer Care Alliance

Seattle, Washington, 98109, United States

RECRUITING

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

NOT YET RECRUITING

Wesley Medical Research

Brisbane, Queensland, 4066, Australia

RECRUITING

Royal Melbourne Hospital (RMH)

Parkville, Victoria, 3050, Australia

RECRUITING

University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski

Sophia, 1000, Belgium

NOT YET RECRUITING

University of Alberta Hospital

Edmonton, Alberta, Edmonton AB T6G 2G3, Canada

RECRUITING

Institute for Clinical and Experimental Medicine - IKEM

Prague, 140 21, Czechia

NOT YET RECRUITING

Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre

Bordeaux, 33000, France

NOT YET RECRUITING

Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier

Colombes, 92700, France

NOT YET RECRUITING

CHU Nantes

Nantes, 44000, France

NOT YET RECRUITING

Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine

Paris, 75018, France

NOT YET RECRUITING

Charite - Universitaetsmedizin Berlin

Berlin, 10117, Germany

RECRUITING

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

Brescia BS, 25123, Italy

RECRUITING

Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo

Cuneo CN, 12100, Italy

NOT YET RECRUITING

Ospedalle Galliera

Genova GE, 16128, Italy

NOT YET RECRUITING

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano

Milan, 20122, Italy

RECRUITING

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

Modena, 41125, Italy

ACTIVE NOT RECRUITING

IFO-San Gallicano IRCCS

Rome, 5300144, Italy

RECRUITING

Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica

Trieste TS, 34137, Italy

NOT YET RECRUITING

Kobe University Hospital

Kobe, Hyōgo, 650-0017, Japan

RECRUITING

Sophia Dermatology Clinic

Kanazawa, Ishikawa-ken, 921-8035, Japan

COMPLETED

Osaka Medical College Hospital

Takatsuki, Osaka, 569-8686, Japan

RECRUITING

Tokyo Saiseikai Central Hospital

Minato-ku, Tokyo, 108-0073, Japan

NOT YET RECRUITING

Toyama University Hospital

Sugitani, Toyama, 930-0194, Japan

RECRUITING

Mazda Hospital of Mazda Motor Corporation

Hiroshima, 730-8670, Japan

NOT YET RECRUITING

Hamamatsu University Hospital

Shizuoka, 431-3125, Japan

NOT YET RECRUITING

Erasmus MC, Universitair Medisch Centrum Rotterdam

Rotterdam, 3015 GD, Netherlands

NOT YET RECRUITING

Haukeland University Hospital

Bergen, N5021, Norway

RECRUITING

Instytut hematologii i Transfuzjologii

Warsaw, 02-776, Poland

NOT YET RECRUITING

Hospital Clínic de Barcelona

Barcelona, 8036, Spain

RECRUITING

Hospital Universitario

Madrid, 28041, Spain

RECRUITING

Hospital General Universitario De Valencia

Valencia, 46014, Spain

NOT YET RECRUITING

Karolinska University Hospital

Stockholm, 14186, Sweden

RECRUITING

Salford Royal NHS Foundation Trust

Manchester, MN, M6 8HD, United Kingdom

RECRUITING

St. John's Institute of Dermatology-Guy's & St Thomas' NHS Foundation Trust

London, SE1 7EH, United Kingdom

RECRUITING

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

London, SE1 9RT, United Kingdom

ACTIVE NOT RECRUITING

Southampton General Hospital - University Hospital Southampton NHS Foundation Trust

Southampton, SO16 6YD, United Kingdom

NOT YET RECRUITING

Study Officials

• Head of Medical Science

  Tanabe Pharma America, Inc.

  STUDY DIRECTOR

Central Study Contacts

Clinical Trials Information Desk, To prevent mis-communication,

CONTACT

please e-mail; information.US@mb.tanabe-pharma.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2021
• First Posted: August 16, 2021
• Study Start: August 10, 2021
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: December 11, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

PL (1); US (15); BE (1); DE (1); CZ (1); IT (7); GB (4); AU (3); CA (1); NL (1); FR (4); NO (1); ES (3); SE (1); JP (7)