Clinical trial • Phase III • Dermatology
DERSIMELAGON PHOSPHATE for Erythropoietic protoporphyria | X-linked protoporphyria
Phase III trial of DERSIMELAGON PHOSPHATE for Erythropoietic protoporphyria | X-linked protoporphyria.
Overview
- Trial Therapeutic Area
- Dermatology
- Trial Disease
- Erythropoietic protoporphyria | X-linked protoporphyria
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 14-11-2023
- First CTIS Authorization Date
- 11-03-2024
Trial design
Randomised, mt-7117 placebo (matching placebo arm). dose and schedule not specified in provided documents.-controlled Phase III trial in Czechia, France, Spain and others.
- Randomised
- Yes
- Comparator
- MT-7117 Placebo (matching placebo arm). Dose and schedule not specified in provided documents.
- Target Sample Size
- 52
- Trial Duration For Participant
- 364
Eligibility
Recruits 52 paediatric patients.
- Pregnancy Exclusion
- Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
- Vulnerable Population
- Vulnerable population: minors/adolescents are included (subjects aged 12 years and older). For minor subjects, both the minor’s assent and the legal representative’s (parent/guardian) consent are required; written informed consent is required from participants or their legal representatives. Assent and parent/guardian consent forms are included among study documents.
Inclusion criteria
- {"criterion_text":"- Subjects provided written informed consent to participate. For minor subjects, both minor’s assent and legal representative´s consent will be required."}
- {"criterion_text":"- Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history."}
- {"criterion_text":"- Subjects aged 12 years to 75 years, inclusive, at Screening."}
- {"criterion_text":"- Subjects are willing and able to travel to the study sites for all scheduled visits."}
- {"criterion_text":"- 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)."}
- {"criterion_text":"- 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."}
- {"criterion_text":"- Female subjects of childbearing potential and male subjects with partner of childbearing potential currently using/willing to use 2 effective methods of contraception"}
Exclusion criteria
- {"criterion_text":"- History or presence of photodermatoses other than EPP or XLP."}
- {"criterion_text":"- Previous treatment with any investigational agent such as bitopertin within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer)."}
- {"criterion_text":"- Subjects who are unwilling or unable to go outside in sunlight during daylight hours most days (e.g., between 1-hour post-sunrise and 1 hour pre-sunset) during the study."}
- {"criterion_text":"- Use of sunscreens with zinc oxide."}
- {"criterion_text":"- Presence or history of any hepatobiliary disease, including drug-induced liver injury at screening, determined as clinically significant by the Investigator after the discussion with the Sponsor Medical Monitor."}
- {"criterion_text":"- 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."}
- {"criterion_text":"- History (in the last 2 years) or presence of alcohol abuse, or abuse of illicit drugs in the opinion of the Investigator."}
- {"criterion_text":"- History of melanoma."}
- {"criterion_text":"- 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."}
- {"criterion_text":"- 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."}
- {"criterion_text":"- Presence of clinically significant acute or chronic renal disease or subjects with an estimated glomerular filtration rate (eGFR) <60 mL/min as calculated by the Chronic Kidney Disease-Epidemiology 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 local recommendations."}
- {"criterion_text":"- 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."}
- {"criterion_text":"- Female subjects who are pregnant, lactating, or intending to become pregnant during the study."}
- {"criterion_text":"- Treatment with any of the following medications or therapy within each period before Randomization (Visit 2): - Afamelanotide within 3 months; - Phototherapy within 3 months; - Cimetidine within 4 weeks; - Antioxidant agents within 4 weeks; - Chronic treatment with any scheduled analgesic agents."}
- {"criterion_text":"- Dermatological treatments 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 at screening such as, for example, tanning agents."}
- {"criterion_text":"- Subjects who participated in any previous MT-7117 clinical studies."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Change from baseline in average daily sunlight exposure time (in minutes) to first prodromal symptom (burning, tingling, itching, or stinging) associated with sunlight exposure between 1-hour post-sunrise and 1 hour pre-sunset at Week 16.","definition_or_measurement_approach":"Change from baseline in average daily sunlight exposure time (minutes) until first prodromal symptom (defined as burning, tingling, itching, or stinging) occurring between 1 hour after sunrise and 1 hour before sunset; assessed at Week 16."}
Secondary endpoints
- {"endpoint_text":"- Patient Global Impression of Change (PGIC) at Week 16.","definition_or_measurement_approach":"Assessed using the Patient Global Impression of Change scale at Week 16."}
- {"endpoint_text":"- Total number of sunlight-induced pain events defined as prodromal symptoms (burning, tingling, itching, or stinging) with pain rating of 1-10 on the Likert scale during the 16-week double-blind treatment period.","definition_or_measurement_approach":"Count of sunlight-induced pain events (prodromal symptoms) with pain rated 1-10 on a Likert scale during the 16-week double-blind treatment period."}
- {"endpoint_text":"- Total number of sunlight-induced non-prodrome, phototoxic reactions during the 16-week double-blind treatment period.","definition_or_measurement_approach":"Count of sunlight-induced non-prodrome phototoxic reactions occurring during the 16-week double-blind treatment period."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 52
- Recruitment Window Months
- 26
- Consent Approach
- Written informed consent is required for all subjects. For minors (adolescents), both the minor's assent and the legal representative's (parent/guardian) consent are required. Age-specific consent/assent documents are included (e.g., 'Assent 12-16', 'Parent' and 'Adult Main' ICFs). Consent/assent documents are available in at least English and Bulgarian as evidenced by document filenames (e.g., L1_SIS-ICF_Adult Main_ENG_FP, L1_SIS-ICF_Adult Main_BGN_FP, L1_SIS-ICF Assent 12-16_FP).
Methods
- K2_HCP Letter (HCP outreach) - document present indicating use of healthcare professional letters to inform clinicians about the study.
- K2_Patient Letter / K2_Recruitment Brochure (Patient-facing materials) - patient letters and brochures available to inform potential participants.
- K2_Splash Campaign_Marketing Materials / K2_Splash Campaign_Privacy Notice (Splash campaign / advertising) - campaign/marketing materials and privacy notices listed among recruitment documents.
- K2_Recruitment_Advertising_Statement (Advertising statement) - advertising materials for recruitment referenced in documents.
- K1_Recruitment procedure / K1_Recruit-ICF process (Recruitment procedure) - procedural documents describing recruitment flow and informed consent handling.
Geography
- Total Number Of Sites
- 17
- Total Number Of Participants
- 98
Czechia
- Earliest CTIS Part Ii Submission Date
- 20-02-2024
- Latest Decision Or Authorization Date
- 17-06-2024
- Processing Time Days
- 118
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Institute For Clinical And Experimental Medicine
- Department Name
- Clinics of Hepatogastroenterology
- Principal Investigator Name
- Jan Sperl
- Principal Investigator Email
- jan.sperl@ikem.cz
- Contact Person Name
- Jan Sperl
- Contact Person Email
- jan.sperl@ikem.cz
France
- Earliest CTIS Part Ii Submission Date
- 22-02-2024
- Latest Decision Or Authorization Date
- 07-06-2024
- Processing Time Days
- 106
- Number Of Sites
- 4
- Number Of Participants
- 25
Sites
- Site Name
- Centre Hospitalier Universitaire De Bordeaux
- Department Name
- Médecine Interne et immonologie
- Principal Investigator Name
- Patrick Mercie
- Principal Investigator Email
- Patrick.mercie@chu-bordeaux.fr
- Contact Person Name
- Patrick Mercie
- Contact Person Email
- Patrick.mercie@chu-bordeaux.fr
- Site Name
- Centre Hospitalier Universitaire De Nantes
- Department Name
- Dermatologie
- Principal Investigator Name
- Marie Le Moigne
- Principal Investigator Email
- marie.lemoigne@chu-nantes.fr
- Contact Person Name
- Marie Le Moigne
- Contact Person Email
- marie.lemoigne@chu-nantes.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Centre d’investigation clinique
- Principal Investigator Name
- Laurent Gouya
- Principal Investigator Email
- Laurent.gouya@aphp.fr
- Contact Person Name
- Laurent Gouya
- Contact Person Email
- Laurent.gouya@aphp.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- CRMR Porphyries
- Principal Investigator Name
- Laurent Gouya
- Principal Investigator Email
- Laurent.gouya@aphp.fr
- Contact Person Name
- Laurent Gouya
- Contact Person Email
- Laurent.gouya@aphp.fr
Spain
- Earliest CTIS Part Ii Submission Date
- 22-02-2024
- Latest Decision Or Authorization Date
- 26-07-2024
- Processing Time Days
- 155
- Number Of Sites
- 2
- Number Of Participants
- 8
Sites
- Site Name
- Hospital General Universitario De Valencia
- Department Name
- Dermatology
- Principal Investigator Name
- Amparo Perez Ferriols
- Principal Investigator Email
- Perez_ampferr@gva.es
- Contact Person Name
- Amparo Perez Ferriols
- Contact Person Email
- Perez_ampferr@gva.es
- Site Name
- Hospital Universitario 12 De Octubre
- Department Name
- Internal Medicine
- Principal Investigator Name
- Francisco Javier Castelbón Fernández
- Principal Investigator Email
- fjcastelbon@yahoo.es
- Contact Person Name
- Francisco Javier Castelbón Fernández
- Contact Person Email
- fjcastelbon@yahoo.es
Poland
- Earliest CTIS Part Ii Submission Date
- 19-02-2024
- Latest Decision Or Authorization Date
- 18-06-2024
- Processing Time Days
- 120
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Instytut Hematologii I Transfuzjologii
- Department Name
- Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych
- Principal Investigator Name
- Jerzy Windyga
- Principal Investigator Email
- sekretariatkzh@ihit.waw.pl
- Contact Person Name
- Jerzy Windyga
- Contact Person Email
- sekretariatkzh@ihit.waw.pl
Netherlands
- Earliest CTIS Part Ii Submission Date
- 23-02-2024
- Latest Decision Or Authorization Date
- 21-01-2025
- Processing Time Days
- 333
- Number Of Sites
- 1
- Number Of Participants
- 20
Sites
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- Department of Internal Medicine
- Principal Investigator Name
- Nicole Peltenburg
- Principal Investigator Email
- e.padberg@erasmusmc.nl
- Contact Person Name
- Nicole Peltenburg
- Contact Person Email
- e.padberg@erasmusmc.nl
Bulgaria
- Earliest CTIS Part Ii Submission Date
- 15-03-2024
- Latest Decision Or Authorization Date
- 17-09-2025
- Processing Time Days
- 551
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- University Multiprofile Hospital For Active Treatment St. Ivan Rilski EAD
- Department Name
- Gastroenterology clinic
- Principal Investigator Name
- Aneta Ivanova
- Principal Investigator Email
- aneta.ivanova@rilski.com
- Contact Person Name
- Aneta Ivanova
- Contact Person Email
- aneta.ivanova@rilski.com
Italy
- Earliest CTIS Part Ii Submission Date
- 19-02-2024
- Latest Decision Or Authorization Date
- 29-04-2026
- Processing Time Days
- 800
- Number Of Sites
- 7
- Number Of Participants
- 30
Sites
- Site Name
- Azienda Ospedaliera Santa Croce E Carle
- Department Name
- Struttura complessa interaziendale di Immunologia e medicina Trasfusionale
- Principal Investigator Name
- Paola Maria Manzini
- Principal Investigator Email
- manzini.p@ospedale.cuneo.it
- Contact Person Name
- Paola Maria Manzini
- Contact Person Email
- manzini.p@ospedale.cuneo.it
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- U.O. Medicina Generale
- Principal Investigator Name
- Giovanna Graziadei
- Principal Investigator Email
- giovanna.graziadei@policlinico.mi.it
- Contact Person Name
- Giovanna Graziadei
- Contact Person Email
- giovanna.graziadei@policlinico.mi.it
- Site Name
- Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo
- Department Name
- Clinica Pediatrica
- Principal Investigator Name
- Serena Pastore
- Principal Investigator Email
- serena.pastore@burlo.trieste.it
- Contact Person Name
- Serena Pastore
- Contact Person Email
- serena.pastore@burlo.trieste.it
- Site Name
- Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
- Department Name
- U.O. Dermatologia
- Principal Investigator Name
- Mariateresa Rossi
- Principal Investigator Email
- mariateresa.rossi@alice.it
- Contact Person Name
- Mariateresa Rossi
- Contact Person Email
- mariateresa.rossi@alice.it
- Site Name
- I.F.O. Istituti Fisioterapici Ospitalieri
- Department Name
- U.O.S.D. Porfirie e Malattie Rare
- Principal Investigator Name
- Luca Barbieri
- Principal Investigator Email
- luca.barbieri@ifo.it
- Contact Person Name
- Luca Barbieri
- Contact Person Email
- luca.barbieri@ifo.it
- Site Name
- Azienda Ospedaliero Universitaria Di Modena
- Department Name
- U.O.C. Medicina Interna
- Principal Investigator Name
- Andrea Ricci
- Principal Investigator Email
- andrea.ricci@unimore.it
- Contact Person Name
- Andrea Ricci
- Contact Person Email
- andrea.ricci@unimore.it
- Site Name
- Ente Ospedaliero Ospedali Galliera Di Genova
- Department Name
- Microcitemia and congenital anemia and Iron, dismetabolism centre
- Principal Investigator Name
- Manuela Ballocco
- Principal Investigator Email
- manuela.balocco@galliera.it
- Contact Person Name
- Manuela Ballocco
- Contact Person Email
- manuela.balocco@galliera.it
Sponsor
Primary sponsor
- Full Name
- Tanabe Pharma America Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Icon Clinical Research Limited
- Responsibilities
- codes: 1,12,13,15 (Lab kits),2,4,5,6
- Name
- Cmic Inc.
- Responsibilities
- code: 4
Third parties
- {"country":"United States","full_name":"University Of Texas Medical Branch At Galveston","duties_or_roles":"code: 4","organisation_type":"Educational Institution"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"code: 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Dcl Pathology LLC","duties_or_roles":"code: 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Scarritt Group Inc.","duties_or_roles":"code: 15 (Investigator Meetings and Travel concierge)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Japan","full_name":"LSI Medience Corp.","duties_or_roles":"code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Ireland","full_name":"Signant Health Global Solutions Limited","duties_or_roles":"code: 3","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"code: 15 (Central ECG reader)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Umotif Limited","duties_or_roles":"code: 15 (ePRO eDiary)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"codes: 1,12,13,15 (Lab kits),2,4,5,6","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Cmic Inc.","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
- {"country":"Japan","full_name":"Konica Minolta Realm Inc.","duties_or_roles":"code: 15 (Melanin density evaluation)","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- MT-7117 Formulation Code C
- Active Substance
- DERSIMELAGON PHOSPHATE
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL
- Orphan Designation
- Yes
- Investigational Product Name
- MT-7117 Placebo
- Modality
- Other
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