Clinical trial • Phase III • Rare Disease|Dermatology
DERSIMELAGON PHOSPHATE for Erythropoietic protoporphyria (EPP)|X-linked protoporphyria (XLP)
Phase III trial of DERSIMELAGON PHOSPHATE for Erythropoietic protoporphyria (EPP)|X-linked protoporphyria (XLP). open-label. 112 participants.
Overview
- Trial Therapeutic Area
- Rare Disease|Dermatology
- Trial Disease
- Erythropoietic protoporphyria (EPP)|X-linked protoporphyria (XLP)
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 09-07-2024
- First CTIS Authorization Date
- 18-07-2024
Trial design
open-label Phase III trial in Sweden, Italy, Spain and others.
- Open Label
- Yes
- Target Sample Size
- 112
Eligibility
Recruits 112 paediatric patients.
- Pregnancy Exclusion
- Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
- Vulnerable Population
- Adolescents are included (age 12 years and older) and require adolescent assent plus legal representative (parent/guardian) consent. The trial materials include age-specific consent/assent forms (adult, parent/guardian, and assent forms). Subjects who are legally institutionalized or under judicial protection are excluded.
Inclusion criteria
- {"criterion_text":"- A subject will be eligible for enrollment in the study if ALL of the following criteria apply:"}
- {"criterion_text":"- Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and legal representative's consent will be provided."}
- {"criterion_text":"- Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history, aged 12 years to 75 years, inclusive, at Screening of studies MT-7117-G01 or MT-7117-A-302 and 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."}
- {"criterion_text":"- Subjects have a body weight of ≥ xx kg. (Please refer to protocol for full details)"}
- {"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 is willing to cooperate and comply with the protocol restrictions and requirements (including travel)."}
- {"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 must agree to use 2 effective methods of contraception including barrier method (especially for female subjects, one method must be highly effective method) as described in Section 4.6.1 of the Protocol."}
Exclusion criteria
- {"criterion_text":"- A subject will NOT be eligible for this study if ANY of the following criteria apply:"}
- {"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 phototherapy or afamelanotide within 3 months, before baseline (Visit 2 or Re-entry Visit 2)."}
- {"criterion_text":"- 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)."}
- {"criterion_text":"- 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."}
- {"criterion_text":"- 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."}
- {"criterion_text":"- Previous treatment with any investigational agent other than MT-7117 within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer)."}
- {"criterion_text":"- 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)."}
- {"criterion_text":"- 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."}
- {"criterion_text":"- Subjects who are legally institutionalized, or subjects under judicial protection."}
- {"criterion_text":"- History or presence of photodermatoses other than EPP or XLP."}
- {"criterion_text":"- Subjects with an immediate family member (i.e. spouse, parent/legal guardian, sibling, or a child) who is a member of study site staff or a member of the Sponsor’s study team."}
- {"criterion_text":"- Use of the following drugs (including but not limited to) within 1 week of baseline (Visit 2 or Re-entry Visit 2): a. 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. b. 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."}
- {"criterion_text":"- Please refer to the protocol for the full list of exclusion criteria."}
- {"criterion_text":"- Presence or history of any hepatobiliary disease at Screening, determined as clinically significant by the Investigator."}
- {"criterion_text":"- 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."}
- {"criterion_text":"- Subjects with or having a history (in the last 2 years) of excessive alcohol intake 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 based upon the subject’s medical records including haemodialysis; an estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m2 as calculated by the CKD-EPI/Creatinine Equation for Glomerular Filtration Rate creatinine equation (2009) for adults and by the Schwartz creatinine equation for adolescents (2009) (Section 16.2, Appendix 2). MDRD/ Modification of Diet in Renal Disease can be used for adults per local recommendations."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Treatment-emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]).","definition_or_measurement_approach":"Collection and reporting of treatment-emergent adverse events, including classification of serious adverse events and adverse events of special interest per safety monitoring procedures."}
- {"endpoint_text":"- Physical examination.","definition_or_measurement_approach":"Standard clinical physical examinations performed at scheduled visits."}
- {"endpoint_text":"- Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature).","definition_or_measurement_approach":"Measurement of blood pressure, respiratory rate, pulse rate, and body temperature at scheduled timepoints."}
- {"endpoint_text":"- Clinical laboratory examinations (hematology, coagulation, biochemistry, and urinalysis), including liver function markers (aspartate aminotransferase [AST], alanine aminotransferase [ALT], gamma glutamyl transpeptidase [GGT], alkaline phosphatase [ALP], direct and total bilirubin).","definition_or_measurement_approach":"Laboratory testing of hematology, coagulation, biochemistry, urinalysis, and specified liver function markers according to central/local laboratory procedures."}
- {"endpoint_text":"- 12-lead electrocardiogram (ECG) parameters.","definition_or_measurement_approach":"Acquisition and analysis of 12-lead ECGs; parameters reviewed per protocol-defined intervals and analysis methods."}
- {"endpoint_text":"- Nevi appearance (assessed by a dermatologist or other qualified site staff). Any nevi undergoing change of clinical concern during active treatment will be biopsied for follow-up and evaluated by the central pathology lab.","definition_or_measurement_approach":"Dermatologic assessment of nevi; biopsy of any clinically concerning changes and central pathology review for follow-up."}
Recruitment
- Planned Sample Size
- 112
- Recruitment Window Months
- 68
- Consent Approach
- Written informed consent is required from participants. For adolescent subjects, both adolescent assent and legal representative (parent/guardian) consent are required. Age-specific documents are provided (adult ICFs, parent/guardian ICFs, assent forms for adolescents). Country-specific language versions of consent/assent documents are available (examples in the submission include English and country-language variants).
Geography
- Total Number Of Sites
- 21
- Total Number Of Participants
- 90
Sweden
- Earliest CTIS Part Ii Submission Date
- 17-06-2024
- Latest Decision Or Authorization Date
- 22-07-2024
- Processing Time Days
- 35
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Karolinska University Hospital
- Department Name
- Department of Upper GI Diseases, C177
- Principal Investigator Name
- Staffan Wahlin
- Principal Investigator Email
- staffan.wahlin@regionstockholm.se
- Contact Person Name
- Staffan Wahlin
- Contact Person Email
- staffan.wahlin@regionstockholm.se
Italy
- Earliest CTIS Part Ii Submission Date
- 17-06-2024
- Latest Decision Or Authorization Date
- 24-07-2024
- Processing Time Days
- 37
- Number Of Sites
- 7
- Number Of Participants
- 19
Sites
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- UOC Medicina Generale “UOS Attività diurne Malattie Rare Infermieristiche”
- 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
- 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
- Site Name
- Azienda Ospedaliera Santa Croce E Carle
- Department Name
- 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
- Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
- Department Name
- U.O. Dermatologia
- Principal Investigator Name
- Mariateresa Rossi
- Principal Investigator Email
- dottoressarossimt@gmail.com
- Contact Person Name
- Mariateresa Rossi
- Contact Person Email
- dottoressarossimt@gmail.com
- Site Name
- I.F.O. Istituti Fisioterapici Ospitalieri
- Department Name
- UOSD Porfirie e Malattie Rare Istituto Dermatologico San Gallicano-
- 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
- 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 Ospedaliero Universitaria Di Modena
- Department Name
- U.O.C. Medicina Interna
- Principal Investigator Name
- Paolo Ventura
- Principal Investigator Email
- paoloven@unimore.it
- Contact Person Name
- Paolo Ventura
- Contact Person Email
- paoloven@unimore.it
Spain
- Earliest CTIS Part Ii Submission Date
- 17-06-2024
- Latest Decision Or Authorization Date
- 18-07-2024
- Processing Time Days
- 31
- Number Of Sites
- 3
- Number Of Participants
- 8
Sites
- Site Name
- Hospital Clinic De Barcelona
- Principal Investigator Name
- Diana Alejandra Sandoval Clavijo
- Principal Investigator Email
- info@hospitalclinic.com
- Contact Person Name
- Diana Alejandra Sandoval Clavijo
- Contact Person Email
- info@hospitalclinic.com
- Site Name
- Hospital General Universitario De Valencia
- Principal Investigator Name
- Amparo Pérez Ferriols
- Principal Investigator Email
- perez_ampferr@gva.es
- Contact Person Name
- Amparo Pérez Ferriols
- Contact Person Email
- perez_ampferr@gva.es
- Site Name
- Hospital Universitario 12 De Octubre
- Principal Investigator Name
- Francisco Javier Castelbon Fernandez
- Principal Investigator Email
- atepac.hdoc@salud.madrid.org
- Contact Person Name
- Francisco Javier Castelbon Fernandez
- Contact Person Email
- atepac.hdoc@salud.madrid.org
Norway
- Earliest CTIS Part Ii Submission Date
- 17-06-2024
- Latest Decision Or Authorization Date
- 25-07-2024
- Processing Time Days
- 38
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Helse Bergen HF
- Department Name
- Norwegian Porphyria Centre (NAPOS)
- Principal Investigator Name
- Aasne Karine Aarsand
- Principal Investigator Email
- porfyri@helse-bergen.no
- Contact Person Name
- Aasne Karine Aarsand
- Contact Person Email
- porfyri@helse-bergen.no
Bulgaria
- Earliest CTIS Part Ii Submission Date
- 19-05-2025
- Latest Decision Or Authorization Date
- 11-06-2025
- Processing Time Days
- 23
- Number Of Sites
- 1
- Number Of Participants
- 2
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
Netherlands
- Earliest CTIS Part Ii Submission Date
- 06-06-2025
- Latest Decision Or Authorization Date
- 19-06-2025
- Processing Time Days
- 13
- Number Of Sites
- 1
- Number Of Participants
- 17
Sites
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- Department of Internal Medicine
- Principal Investigator Name
- Nicole Chantal Peltenburg
- Principal Investigator Email
- n.c.peltenburg@erasmusmc.nl
- Contact Person Name
- Nicole Chantal Peltenburg
- Contact Person Email
- n.c.peltenburg@erasmusmc.nl
Czechia
- Earliest CTIS Part Ii Submission Date
- 29-05-2025
- Latest Decision Or Authorization Date
- 11-07-2025
- Processing Time Days
- 43
- 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
Germany
- Earliest CTIS Part Ii Submission Date
- 17-06-2024
- Latest Decision Or Authorization Date
- 26-07-2024
- Processing Time Days
- 39
- Number Of Sites
- 1
- Number Of Participants
- 6
Sites
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Institut für Allergieforschung
- Principal Investigator Name
- Manuel Pedro Fernandes Lobo Pereira
- Principal Investigator Email
- manuel.pereira@charite.de
- Contact Person Name
- Manuel Pedro Fernandes Lobo Pereira
- Contact Person Email
- manuel.pereira@charite.de
Poland
- Earliest CTIS Part Ii Submission Date
- 13-06-2025
- Latest Decision Or Authorization Date
- 07-07-2025
- Processing Time Days
- 24
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Instytut Hematologii I Transfuzjologii
- Department Name
- Klinika Zaburzen Hemostazy i Chorob Wewnetrznych
- Principal Investigator Name
- Jerzy Windyga
- Principal Investigator Email
- onkocwbk@ihit.waw.pl
- Contact Person Name
- Jerzy Windyga
- Contact Person Email
- onkocwbk@ihit.waw.pl
France
- Earliest CTIS Part Ii Submission Date
- 22-05-2025
- Latest Decision Or Authorization Date
- 13-06-2025
- Processing Time Days
- 22
- Number Of Sites
- 4
- Number Of Participants
- 26
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
- Assistance Publique Hopitaux De Paris
- Department Name
- Department of Biochemistry and molecular genetics
- 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
- 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
- Department of Biochemistry and molecular genetics
- Principal Investigator Name
- Laurent Gouya
- Principal Investigator Email
- Laurent.gouya@aphp.fr
- Contact Person Name
- Laurent Gouya
- Contact Person Email
- Laurent.gouya@aphp.fr
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
- Operational and study management tasks; clinical chemistry, clinical haematology, serology/endocrinology, analytical chemistry, central lab functions (as listed in sponsor duties)
- Name
- Accellacare Limited
- Responsibilities
- Home health visits
- Name
- Signant Health Global LLC
- Responsibilities
- IVRS / treatment randomisation and related eClinical services
- Name
- Medidata Solutions Inc.
- Responsibilities
- Clinical data platform services
Third parties
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Sponsor duties codes: 1,11,12,2,4,6; includes clinical chemistry, clinical haematology, serology/endocrinology, analytical chemistry, central lab","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"University Of Texas Medical Branch At Galveston","duties_or_roles":"porphyrin testing","organisation_type":"Educational Institution"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Sponsor duties code: 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Japan","full_name":"Konica Minolta Realm Inc.","duties_or_roles":"supplier of Evaluation equipment (spectrophotometer & laptop)","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Accellacare Limited","duties_or_roles":"home health visits","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"IVRS - treatment randomisation; sponsor duty code: 3","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG analysis / review","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Scarritt Group Inc.","duties_or_roles":"patient travel and reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Eurofins Central Laboratory LLC","duties_or_roles":"skin biopsies","organisation_type":"Laboratory/Research/Testing facility"}
Investigational products
- Investigational Product Name
- MT-7117 Formulation Code C
- Active Substance
- DERSIMELAGON PHOSPHATE
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL USE
- Orphan Designation
- Yes
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