Phase
Condition
N/ATreatment
Dersimelagon
Placebo
Clinical Study ID
Ages 12-75 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Subjects provided written informed consent to participate. For minor subjects, bothminor's assent and parental consent will be required.
Male and female subjects with a confirmed diagnosis of EPP or XLP based on medicalhistory.
Subjects aged 12 years to 75 years, inclusive, at Screening.
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 andany risks involved in participation, and willing to cooperate and comply with theprotocol restrictions and requirements (including travel and receiving directsunlight exposure as much as possible).
Female subjects who are non-lactating and have a negative urine pregnancy test atbaseline visit prior to receiving the first dose of study drug.
Female subjects of childbearing potential and male subjects with partner ofchild-bearing potential currently using/willing to use 2 effective methods ofcontraception.
Additional screening criteria check may apply for qualification.
Exclusion
Exclusion Criteria:
History or presence of photodermatoses other than EPP or XLP.
Subjects who are unwilling or unable to go outside in sunlight during daylight hoursmost days (e.g., between 1-hour post-sunrise and 1 hour pre-sunset) during thestudy.
Presence or history of any hepatobiliary disease, including druginduced liver injuryat screening, determined as clinically significant by the Investigator after thediscussion with the Sponsor Medical Monitor.
Subjects with aspartate aminotransferase (AST), alanine aminotransferase (ALT),alkaline phosphatase (ALP) ≥ 2.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
History (in the last 2 years) or presence of alcohol abuse, or abuse of illicitdrugs in the opinion of the Investigator.
History of melanoma.
Presence of squamous cell carcinoma, basal cell carcinoma, or other malignant skinlesions. Any suspicious lesions or nevi will be evaluated. If the suspicious lesionor nevi cannot be resolved through biopsy or excision, the subject will be excludedfrom the study.
History or presence of psychiatric disease judged to be clinically significant bythe Investigator and which may interfere with the study evaluation and/or safety ofthe subjects.
Presence of clinically significant acute or chronic renal disease or subjects withan estimated glomerular filtration rate (eGFR) <60 mL/min as calculated by theChronic Kidney DiseaseEpidemiology Collaboration (CKD-EPI) creatinine equation (2021) for adults and by the Schwartz creatinine equation for adolescents (2009).Modification of Diet in Renal Disease can be used for adults per localrecommendations.
Presence of any clinically significant disease or laboratory abnormality which, inthe opinion of the Investigator, can interfere with the study objectives and/orsafety of the subjects.
Female subjects who are pregnant, lactating, or intending to become pregnant duringthe study.
Treatment with any of the following medications or therapy within each period beforeRandomization (Visit 2);
Afamelanotide within 3 months
Phototherapy within 3 months
Cimetidine within 4 weeks
Antioxidant agents within 4 weeks, at doses which, in the opinion of theInvestigator, may affect study endpoints (including but not limited tobeta-carotene, cysteine, pyridoxine).
Chronic treatment with any scheduled analgesic agents including, but notlimited to, opioids and opioid derivatives such as morphine, hydrocodone,oxycodone, fentanyl, or their combination with other unscheduled analgesics ornon-steroidal anti-inflammatory drug (Percocet and Vicodin-like prescriptiondrugs) within 4 weeks. Note: Acute use of scheduled narcotics more than 3 months prior to randomization areallowed. Non-steroidal anti-inflammatory drug, aspirin for analgesia, or priortemporary use of scheduled agents within 3 months of screening is allowed.
Dermatological treatments with any drugs or supplements which, in the opinion of theInvestigator, can interfere with the objectives of the study or safety of thesubjects at screening, such as, for example, tanning agents.
Subjects who participated in any previous MT-7117 clinical studies.
Previous treatment with any investigational agent such as bitopertin, within 12weeks before Screening or 5 half-lives of the investigational product (whichever islonger).
Use of sunscreens with zinc oxide. Note: Sunscreens without zinc oxide are allowed,however their use, in frequency, quantity and body surface area should be maintainedrelatively stable throughout the duration of the study.
History of any hypersensitivity to the active ingredient and/or excipients (lactosemonohydrate, hydroxypropylcellulose, carmellose calcium, magnesium stearate,hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, ironoxide red, and iron oxide black). (EU ONLY)
Subjects who are unable to swallow tablets or have diseases significantly affectingthe gastrointestinal function such as malabsorption syndrome, resection of thestomach or small bowel, bariatric surgery procedures, symptomatic inflammatory boweldisease, or partial or complete bowel obstruction.(EU ONLY)
History of any hypersensitivity to the active ingredient and/or excipients containedin MT-7117 IMP (lactose monohydrate, hydroxypropyl cellulose, carmellose calcium,magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, ironoxide yellow, iron oxide red, and iron oxide black). (UK ONLY)
Additional screening criteria check may apply for qualification.
Study Design
Connect with a study center
Royal Prince Alfred Hospital
Camperdown, New South Wales 02050
AustraliaSite Not Available
Royal Melbourne Hospital (RMH)
Parkville, Victoria 03050
AustraliaSite Not Available
University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski - Porphyria Center
Sofia, 01000
BulgariaSite Not Available
Institute for Clinical and Experimental Medicine - IKEM
Prague, 01958
CzechiaSite Not Available
Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre
Bordeaux, 33000
FranceSite Not Available
CHU Nantes
Nantes, 44000
FranceSite Not Available
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier
Paris, 92701
FranceSite Not Available
Hôpital Bichat - Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine
Paris, 75018
FranceSite Not Available
Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
Brescia, 25123
ItalySite Not Available
Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo
Cuneo, 12100
ItalySite Not Available
Ospedalle Galliera
Genova, 16128
ItalySite Not Available
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan, 20122
ItalySite Not Available
U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
Modena, 41124
ItalySite Not Available
IFO-San Gallicano IRCCS
Rome, 00144
ItalySite Not Available
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica
Trieste, 34137
ItalySite Not Available
Mazda Hospital
Aki-gun, Hiroshima-ken 735-8585
JapanSite Not Available
Hamamatsu University Hospital
Hamamatsu, Shizuoka 431-3194
JapanSite Not Available
Tokyo Saiseikai Central Hospital
Tokyo, 108-0073
JapanSite Not Available
Universitair Medisch Centrum Rotterdam
Rotterdam, 03015
NetherlandsSite Not Available
Instytut Hematologii I Transfuzjologii
Warsaw, 02-776
PolandSite Not Available
Hospital Universitario 12 de Octubre
Madrid, 28041
SpainSite Not Available
Hospital General Universitario De Valencia
Valencia, 46014
SpainSite Not Available
Guy's Hospital
London, SE1 9RT
United KingdomSite Not Available
Southampton General Hospital - University Hospital Southampton NHS Foundation Trust
Southamption, SO16 6YD
United KingdomSite Not Available
Marvel Clinical Research, LLC
Huntington Beach, California 92647
United StatesSite Not Available
University of California at San Francisco
San Francisco, California 94143
United StatesSite Not Available
University Of Miami School Of Medicine, Center For Liver Diseases
Miami, Florida 33136
United StatesSite Not Available
MGH
Boston, Massachusetts 02129
United StatesSite Not Available
MetroBoston Clinical Partners, LLC
Brighton, Massachusetts 02135-3511
United StatesSite Not Available
Henry Ford Health System
Detroit, Michigan 48202
United StatesSite Not Available
Kansas City Research Institute
Kansas City, Missouri 64131
United StatesSite Not Available
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
New York, New York 10029-0311
United StatesSite Not Available
Wake Forest University Baptist Health
Winston-Salem, North Carolina 27103
United StatesSite Not Available
Remington-Davis Clinical Research
Columbus, Ohio 43215
United StatesSite Not Available
Einstein Medical Center (EMC)
Philadelphia, Pennsylvania 19141
United StatesSite Not Available
The University of Texas Medical Branch (UTMB)
Galveston, Texas 77555-0342
United StatesSite Not Available
University of Washington
Seattle, Washington 98195
United StatesSite Not Available
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