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NCT05005975PHASE3Recruiting

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

Tanabe Pharma America, Inc.

Start Date

8/10/2021

Completion Date

12/1/2027

Summary

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

Eligibility Criteria

Age Range: 12 years to 75 years

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.

Interventions

DRUG

MT-7117

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Conditions

EPPXLP

Locations

Marvel Clinical Research, LLC

Huntington Beach, California 92647

United States

University of California at San Francisco - CSF Porphyria Center

San Francisco, California 94143

United States

University Of Miami School Of Medicine, Center For Liver Diseases

Miami, Florida 33136

United States

Massachusetts General Hospital

Boston, Massachusetts 02114

United States

MetroBoston Clinical Partners, LLC

Brighton, Massachusetts 02135-3211

United States

Henry Ford Health System

Detroit, Michigan 48202

United States

Kansas City Research Institute

Kansas City, Missouri 64131

United States

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

New York, New York 10029

United States

Wake Forest University Baptist Health

Winston-Salem, North Carolina 27157

United States

Cleveland Clinic - Taussig Cancer Institute

Cleveland, Ohio 44106

United States

Remington-Davis Clinical Research

Columbus, Ohio 43215

United States

Thomas Jefferson University

Philadelphia, Pennsylvania 19107

United States

Einstein Medical Center, Philadelphia

Philadelphia, Pennsylvania 19141

United States

The University of Texas Medical Branch (UTMB)

Galveston, Texas 77555

United States

University of Washington-Seattle Cancer Care Alliance

Seattle, Washington 98109

United States

Royal Prince Alfred Hospital

Camperdown, New South Wales 2050

Australia

Wesley Medical Research

Brisbane, Queensland 4066

Australia

Royal Melbourne Hospital (RMH)

Parkville, Victoria 3050

Australia

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

Sophia, 1000

Belgium

University of Alberta Hospital

Edmonton, Alberta Edmonton AB T6G 2G3

Canada

Institute for Clinical and Experimental Medicine - IKEM

Prague, 140 21

Czechia

Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre

Bordeaux, 33000

France

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

Colombes, 92700

France

CHU Nantes

Nantes, 44000

France

Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine

Paris, 75018

France

Charite - Universitaetsmedizin Berlin

Berlin, 10117

Germany

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

Brescia BS, 25123

Italy

Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo

Cuneo CN, 12100

Italy

Ospedalle Galliera

Genova GE, 16128

Italy

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano

Milan, 20122

Italy

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

Modena, 41125

Italy

IFO-San Gallicano IRCCS

Rome, 5300144

Italy

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

Trieste TS, 34137

Italy

Kobe University Hospital

Kobe, Hyōgo 650-0017

Japan

Sophia Dermatology Clinic

Kanazawa, Ishikawa-ken 921-8035

Japan

Osaka Medical College Hospital

Takatsuki, Osaka 569-8686

Japan

Tokyo Saiseikai Central Hospital

Minato-ku, Tokyo 108-0073

Japan

Toyama University Hospital

Sugitani, Toyama 930-0194

Japan

Mazda Hospital of Mazda Motor Corporation

Hiroshima, 730-8670

Japan

Hamamatsu University Hospital

Shizuoka, 431-3125

Japan

Erasmus MC, Universitair Medisch Centrum Rotterdam

Rotterdam, 3015 GD

Netherlands

Haukeland University Hospital

Bergen, N5021

Norway

Instytut hematologii i Transfuzjologii

Warsaw, 02-776

Poland

Hospital Clínic de Barcelona

Barcelona, 8036

Spain

Hospital Universitario

Madrid, 28041

Spain

Hospital General Universitario De Valencia

Valencia, 46014

Spain

Karolinska University Hospital

Stockholm, 14186

Sweden

Salford Royal NHS Foundation Trust

Manchester, MN M6 8HD

United Kingdom

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

London, SE1 7EH

United Kingdom

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

London, SE1 9RT

United Kingdom

Southampton General Hospital - University Hospital Southampton NHS Foundation Trust

Southampton, SO16 6YD

United Kingdom