A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria
Status: Active_not_recruiting
Location: See all (35) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 75
Healthy Volunteers: f
View:
• Subjects provided written informed consent to participate. For minor subjects, both minor's assent and parental consent will be required.
• Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history.
• 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 and any risks involved in participation, and willing to cooperate and comply with the protocol restrictions and requirements (including travel and receiving direct sunlight exposure as much as possible).
• 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 child-bearing potential currently using/willing to use 2 effective methods of contraception.
⁃ Additional screening criteria check may apply for qualification.
Locations
United States
California
Marvel Clinical Research, LLC
Huntington Beach
University of California at San Francisco
San Francisco
Florida
University Of Miami School Of Medicine, Center For Liver Diseases
Miami
Massachusetts
MGH
Boston
MetroBoston Clinical Partners, LLC
Brighton
Michigan
Henry Ford Health System
Detroit
Missouri
Kansas City Research Institute
Kansas City
North Carolina
Wake Forest University Baptist Health
Winston-salem
New York
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
New York
Ohio
Remington-Davis Clinical Research
Columbus
Pennsylvania
Einstein Medical Center (EMC)
Philadelphia
Texas
The University of Texas Medical Branch (UTMB)
Galveston
Washington
University of Washington
Seattle
Other Locations
Australia
Royal Melbourne Hospital (RMH)
Parkville
Bulgaria
University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski - Porphyria Center
Sofia
France
Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre
Bordeaux
CHU Nantes
Nantes
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier
Paris
Hôpital Bichat - Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine
Paris
Italy
Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
Brescia
Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo
Cuneo
Ospedalle Galliera
Genova
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan
U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
Modena
IFO-San Gallicano IRCCS
Rome
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica
Trieste
Japan
Mazda Hospital
Aki-gun
Hamamatsu University Hospital
Hamamatsu
Tokyo Saiseikai Central Hospital
Tokyo
Netherlands
Universitair Medisch Centrum Rotterdam
Rotterdam
Poland
Instytut Hematologii I Transfuzjologii
Warsaw
Spain
Hospital Universitario 12 de Octubre
Madrid
Hospital General Universitario De Valencia
Valencia
United Kingdom
Guy's Hospital
London
Southampton General Hospital - University Hospital Southampton NHS Foundation Trust