Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
NCT ID: NCT05005975
Last Updated: 2025-12-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
301 participants
INTERVENTIONAL
2021-08-10
2027-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Dersimelagon 200mg
MT-7117
MT-7117
Interventions
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MT-7117
MT-7117
Other Intervention Names
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Eligibility Criteria
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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 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
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.
* 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.
12 Years
75 Years
ALL
No
Sponsors
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Tanabe Pharma America, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Head of Medical Science
Role: STUDY_DIRECTOR
Tanabe Pharma America, Inc.
Locations
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Marvel Clinical Research, LLC
Huntington Beach, California, United States
University of California at San Francisco - CSF Porphyria Center
San Francisco, California, United States
University Of Miami School Of Medicine, Center For Liver Diseases
Miami, Florida, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
MetroBoston Clinical Partners, LLC
Brighton, Massachusetts, United States
Henry Ford Health System
Detroit, Michigan, United States
Kansas City Research Institute
Kansas City, Missouri, United States
Icahn School of Medicine at Mount Sinai (ISSMS)-The Mount Sinai Hospital (MSH)
New York, New York, United States
Wake Forest University Baptist Health
Winston-Salem, North Carolina, United States
Cleveland Clinic - Taussig Cancer Institute
Cleveland, Ohio, United States
Remington-Davis Clinical Research
Columbus, Ohio, United States
Thomas Jefferson University
Philadelphia, Pennsylvania, United States
Einstein Medical Center, Philadelphia
Philadelphia, Pennsylvania, United States
The University of Texas Medical Branch (UTMB)
Galveston, Texas, United States
University of Washington-Seattle Cancer Care Alliance
Seattle, Washington, United States
Royal Prince Alfred Hospital
Camperdown, New South Wales, Australia
Wesley Medical Research
Brisbane, Queensland, Australia
Royal Melbourne Hospital (RMH)
Parkville, Victoria, Australia
University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski
Sophia, , Belgium
University of Alberta Hospital
Edmonton, Alberta, Canada
Institute for Clinical and Experimental Medicine - IKEM
Prague, , Czechia
Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre
Bordeaux, , France
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier
Colombes, , France
CHU Nantes
Nantes, , France
Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine
Paris, , France
Charite - Universitaetsmedizin Berlin
Berlin, , Germany
Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
Brescia BS, , Italy
Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo
Cuneo CN, , Italy
Ospedalle Galliera
Genova GE, , Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan, , Italy
U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
Modena, , Italy
IFO-San Gallicano IRCCS
Rome, , Italy
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica
Trieste TS, , Italy
Kobe University Hospital
Kobe, Hyōgo, Japan
Sophia Dermatology Clinic
Kanazawa, Ishikawa-ken, Japan
Osaka Medical College Hospital
Takatsuki, Osaka, Japan
Tokyo Saiseikai Central Hospital
Minato-ku, Tokyo, Japan
Toyama University Hospital
Sugitani, Toyama, Japan
Mazda Hospital of Mazda Motor Corporation
Hiroshima, , Japan
Hamamatsu University Hospital
Shizuoka, , Japan
Erasmus MC, Universitair Medisch Centrum Rotterdam
Rotterdam, , Netherlands
Haukeland University Hospital
Bergen, , Norway
Instytut hematologii i Transfuzjologii
Warsaw, , Poland
Hospital Clínic de Barcelona
Barcelona, , Spain
Hospital Universitario
Madrid, , Spain
Hospital General Universitario De Valencia
Valencia, , Spain
Karolinska University Hospital
Stockholm, , Sweden
Salford Royal NHS Foundation Trust
Manchester, MN, United Kingdom
St. John's Institute of Dermatology-Guy's & St Thomas' NHS Foundation Trust
London, , United Kingdom
Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
London, , United Kingdom
Southampton General Hospital - University Hospital Southampton NHS Foundation Trust
Southampton, , United Kingdom
Countries
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Central Contacts
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Clinical Trials Information Desk, To prevent mis-communication,
Role: CONTACT
Other Identifiers
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jRCT2041210146
Identifier Type: REGISTRY
Identifier Source: secondary_id
2021-001831-17
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
MT-7117-A-301
Identifier Type: -
Identifier Source: org_study_id
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