Actively Recruiting
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
Led by Tanabe Pharma America, Inc. · Updated on 2025-12-11
301
Participants Needed
51
Research Sites
329 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
To evaluate the long-term safety and tolerability of oral dersimelagon.
CONDITIONS
Official Title
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided.
- 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.
- Subjects are willing and able to travel to the study sites for all scheduled visits.
- Investigator believes subject can understand the study and risks, and will cooperate with protocol requirements.
- Female subjects who are non-lactating and have a negative urine pregnancy test at baseline before first dose.
- Female subjects of childbearing potential and male subjects with partner of childbearing potential must agree to use two effective contraception methods, including one highly effective method for females.
You will not qualify if you...
- History or presence of photodermatoses other than EPP or XLP.
- Clinically significant hepatobiliary disease at Screening.
- AST, ALT, ALP ≥ 3.0 times upper limit of normal (ULN) or total bilirubin > 1.5 times ULN at Screening, except for Gilbert's syndrome cases as per Sponsor consultation.
- History (last 2 years) of excessive alcohol intake.
- History of melanoma.
- Presence of squamous cell carcinoma, basal cell carcinoma, or other malignant skin lesions; unresolved suspicious lesions or nevi.
- Clinically significant psychiatric disease interfering with study.
- Clinically significant acute or chronic renal disease including hemodialysis; eGFR <60 mL/min/1.73m2.
- Any significant disease or lab abnormality that may interfere with study or safety.
- Female subjects who are pregnant, lactating, or intending pregnancy during the study.
- Treatment with phototherapy or afamelanotide within 3 months before baseline.
- Treatment with cimetidine or antioxidant agents affecting endpoints within 4 weeks before baseline.
- Chronic treatment with opioids, ketamine, or cannabis derivatives within 4 weeks before baseline.
- Use of drugs or supplements interfering with study objectives or safety.
- Previous investigational agent treatment other than dersimelagon within 12 weeks before Screening or 5 half-lives.
- History of hypersensitivity to active ingredient or excipients.
- Unable to swallow tablets or with significant gastrointestinal conditions.
- Use of certain drugs metabolized by CYP3A4 or substrates of transport proteins within 1 week of baseline that could cause safety concerns.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 51 locations
1
Marvel Clinical Research, LLC
Huntington Beach, California, United States, 92647
Actively Recruiting
2
University of California at San Francisco - CSF Porphyria Center
San Francisco, California, United States, 94143
Not Yet Recruiting
3
University Of Miami School Of Medicine, Center For Liver Diseases
Miami, Florida, United States, 33136
Not Yet Recruiting
4
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Not Yet Recruiting
5
MetroBoston Clinical Partners, LLC
Brighton, Massachusetts, United States, 02135-3211
Actively Recruiting
6
Henry Ford Health System
Detroit, Michigan, United States, 48202
Not Yet Recruiting
7
Kansas City Research Institute
Kansas City, Missouri, United States, 64131
Actively Recruiting
8
Icahn School of Medicine at Mount Sinai (ISSMS)-The Mount Sinai Hospital (MSH)
New York, New York, United States, 10029
Actively Recruiting
9
Wake Forest University Baptist Health
Winston-Salem, North Carolina, United States, 27157
Actively Recruiting
10
Cleveland Clinic - Taussig Cancer Institute
Cleveland, Ohio, United States, 44106
Not Yet Recruiting
11
Remington-Davis Clinical Research
Columbus, Ohio, United States, 43215
Actively Recruiting
12
Thomas Jefferson University
Philadelphia, Pennsylvania, United States, 19107
Actively Recruiting
13
Einstein Medical Center, Philadelphia
Philadelphia, Pennsylvania, United States, 19141
Not Yet Recruiting
14
The University of Texas Medical Branch (UTMB)
Galveston, Texas, United States, 77555
Actively Recruiting
15
University of Washington-Seattle Cancer Care Alliance
Seattle, Washington, United States, 98109
Actively Recruiting
16
Royal Prince Alfred Hospital
Camperdown, New South Wales, Australia, 2050
Not Yet Recruiting
17
Wesley Medical Research
Brisbane, Queensland, Australia, 4066
Actively Recruiting
18
Royal Melbourne Hospital (RMH)
Parkville, Victoria, Australia, 3050
Actively Recruiting
19
University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski
Sophia, Belgium, 1000
Not Yet Recruiting
20
University of Alberta Hospital
Edmonton, Alberta, Canada, Edmonton AB T6G 2G3
Actively Recruiting
21
Institute for Clinical and Experimental Medicine - IKEM
Prague, Czechia, 140 21
Not Yet Recruiting
22
Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre
Bordeaux, France, 33000
Not Yet Recruiting
23
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier
Colombes, France, 92700
Not Yet Recruiting
24
CHU Nantes
Nantes, France, 44000
Not Yet Recruiting
25
Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine
Paris, France, 75018
Not Yet Recruiting
26
Charite - Universitaetsmedizin Berlin
Berlin, Germany, 10117
Actively Recruiting
27
Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
Brescia BS, Italy, 25123
Actively Recruiting
28
Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo
Cuneo CN, Italy, 12100
Not Yet Recruiting
29
Ospedalle Galliera
Genova GE, Italy, 16128
Not Yet Recruiting
30
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan, Italy, 20122
Actively Recruiting
31
U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
Modena, Italy, 41125
Active, Not Recruiting
32
IFO-San Gallicano IRCCS
Rome, Italy, 5300144
Actively Recruiting
33
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica
Trieste TS, Italy, 34137
Not Yet Recruiting
34
Kobe University Hospital
Kobe, Hyōgo, Japan, 650-0017
Actively Recruiting
35
Sophia Dermatology Clinic
Kanazawa, Ishikawa-ken, Japan, 921-8035
Completed
36
Osaka Medical College Hospital
Takatsuki, Osaka, Japan, 569-8686
Actively Recruiting
37
Tokyo Saiseikai Central Hospital
Minato-ku, Tokyo, Japan, 108-0073
Not Yet Recruiting
38
Toyama University Hospital
Sugitani, Toyama, Japan, 930-0194
Actively Recruiting
39
Mazda Hospital of Mazda Motor Corporation
Hiroshima, Japan, 730-8670
Not Yet Recruiting
40
Hamamatsu University Hospital
Shizuoka, Japan, 431-3125
Not Yet Recruiting
41
Erasmus MC, Universitair Medisch Centrum Rotterdam
Rotterdam, Netherlands, 3015 GD
Not Yet Recruiting
42
Haukeland University Hospital
Bergen, Norway, N5021
Actively Recruiting
43
Instytut hematologii i Transfuzjologii
Warsaw, Poland, 02-776
Not Yet Recruiting
44
Hospital Clínic de Barcelona
Barcelona, Spain, 8036
Actively Recruiting
45
Hospital Universitario
Madrid, Spain, 28041
Actively Recruiting
46
Hospital General Universitario De Valencia
Valencia, Spain, 46014
Not Yet Recruiting
47
Karolinska University Hospital
Stockholm, Sweden, 14186
Actively Recruiting
48
Salford Royal NHS Foundation Trust
Manchester, MN, United Kingdom, M6 8HD
Actively Recruiting
49
St. John's Institute of Dermatology-Guy's & St Thomas' NHS Foundation Trust
London, United Kingdom, SE1 7EH
Actively Recruiting
50
Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
London, United Kingdom, SE1 9RT
Active, Not Recruiting
51
Southampton General Hospital - University Hospital Southampton NHS Foundation Trust
Southampton, United Kingdom, SO16 6YD
Not Yet Recruiting
Research Team
C
Clinical Trials Information Desk, To prevent mis-communication,
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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