EPPPhase 3NCT05005975
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
A Phase 3 study testing MT-7117 in people with EPP and XLP, run by Tanabe Pharma America, Inc. at 45 sites in 15 countries.
Trial summary
To evaluate the long-term safety and tolerability of oral dersimelagon. Read more about this study
- Who is it for?
- Ages 12 to 75 years
- What is being tested?
- MT-7117
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 286 participants
- Where
- 45 sitesin 15 countries
- Study dates
- 10 Aug 2021to Dec 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Additional screening criteria check may apply for qualification:
- 1. Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided.
- 2. 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.
- 3. Subjects are willing and able to travel to the study sites for all scheduled visits.
- 4. 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).
- 5. 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.
- 6. 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)
Who cannot join
- Additional screening criteria check may apply for qualification:
- A subject will NOT be eligible for this study if ANY of the following criteria apply:
- 1. History or presence of photodermatoses other than EPP or XLP.
- 2. Presence of clinically significant hepatobiliary disease at Screening, determined as clinically significant by the Investigator.
- 3. 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.
- 4. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
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- 5. History of melanoma.
- 6. 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.
- 7. 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.
- 8. Presence of clinically significant acute or chronic renal disease based upon the subject's medical records including hemodialysis; an estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m2 as calculated by the CKD-EPI creatinine equation (2009) for adults and by the Schwartz creatinine equation for adolescents (2009). MDRD can be used for adults per local recommendations.
- 9. 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.
- 10. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
- 11. Treatment with phototherapy or afamelanotide within 3 months before baseline (Visit 2 or Re-entry Visit 2).
- 12. 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).
- 13. 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.
- 14. 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.
- 15. Previous treatment with any investigational agent other than dersimelagon within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).
- 16. 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).
- 17. 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.
- 18. Using the following drugs (including but not limited to) within 1 week of baseline (Visit 2 or Re-entry Visit 2):
- 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.
- 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.
Trial locations and contacts
45 sites in 15 countries.
45 sites in 15 countries
United States13 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
MetroBoston Clinical Partners, LLC
Brighton, Massachusetts, 02135-3211
Remington-Davis Clinical Research
Columbus, Ohio, 43215
Henry Ford Health System
Detroit, Michigan, 48202
The University of Texas Medical Branch (UTMB)
Galveston, Texas, 77555
Marvel Clinical Research, LLC
Huntington Beach, California, 92647
Kansas City Research Institute
Kansas City, Missouri, 64131
University Of Miami School Of Medicine, Center For Liver Diseases
Miami, Florida, 33136
Icahn School of Medicine at Mount Sinai (ISSMS)-The Mount Sinai Hospital (MSH)
New York, New York, 10029
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
University of California at San Francisco - CSF Porphyria Center
San Francisco, California, 94143
University of Washington-Seattle Cancer Care Alliance
Seattle, Washington, 98109
Wake Forest University Baptist Health
Winston-Salem, North Carolina, 27157
Italy7 sites
Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
Brescia BS, 25123
Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo
Cuneo CN, 12100
Ospedalle Galliera
Genova GE, 16128
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan, 20122
U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
Modena, 41125
I.F.O. Hospital, Centro Porfirie e Malattie Rare
Roma, Rome, 128
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica
Trieste TS, 34137
Japan6 sites
Sophia Dermatology Clinic
Kanazawa, Ishikawa-ken, 921-8035
Kobe University Hospital
Kobe, Hyōgo, 650-0017
Tokyo Saiseikai Central Hospital
Minato-ku, Tokyo, 108-0073
Hamamatsu University Hospital
Shizuoka, 431-3125
Toyama University Hospital
Sugitani, Toyama, 930-0194
Osaka Medical College Hospital
Takatsuki, Osaka, 569-8686
France4 sites
Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre
Bordeaux, 33000
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier
Colombes, 92700
CHU Nantes
Nantes, 44000
Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine
Paris, 75018
Spain3 sites
Hospital Clínic de Barcelona
Barcelona, 8036
Hospital Universitario
Madrid, 28041
Hospital General Universitario De Valencia
Valencia, 46014
Australia2 sites
Wesley Medical Research
Brisbane, Queensland, 4066
Royal Melbourne Hospital (RMH)
Parkville, Victoria, 3050
United Kingdom2 sites
St. John's Institute of Dermatology-Guy's & St Thomas' NHS Foundation Trust
London, SE1 7EH
Salford Royal NHS Foundation Trust
Manchester, MN, M6 8HD
Bulgaria1 site
University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski
Sofia, 1000
Canada1 site
University of Alberta Hospital
Edmonton, Alberta, Edmonton AB T6G 2G3
Czechia1 site
Institute for Clinical and Experimental Medicine - IKEM
Prague, 140 21
Netherlands1 site
Erasmus MC, Universitair Medisch Centrum Rotterdam
Rotterdam, 3015 GD
About this study
Official title: A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
To evaluate the long-term safety and tolerability of oral dersimelagon.
- Being tested
- MT-7117
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MT-7117Drug
Also called Dersimelagon
MT-7117
| Study group | Role | What they receive |
|---|---|---|
| Dersimelagon 200mg | Experimental | Not described Receives: Drug: MT-7117 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]).
Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature), Clinical laboratory examinations (hematology, coagulation, biochemistry, urinalysis, and others), 12-lead electrocardiogram (ECG) parameters (Mean Heart Rate, PR Interval, QRS Duration, QT interval, QTcB and QTcF) will be assessed.
Measured: up to 66 further months
Number of patients with abnormal Physical examination data
Physical examination consists of assessment of abdominal, respiratory, cardiovascular, general appearance, and others.
Measured: up to 66 further months
Number of patients with Nevi appearance
Measured: up to 66 further months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2021
- Study start10 Aug 2021
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for EPPStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 286 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tanabe Pharma America, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Head of Medical Science, Study director, Tanabe Pharma America, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05005975
- Sponsor's protocol ID
- MT-7117-A-301
- Other IDs
- jRCT2041210146, 2021-001831-17
- Status last verified
- Sep 2026
- First submitted
- 28 Jul 2021
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
- ClinicalTrials.gov ID
- NCT05005975
- Acronym / sponsor ID
- MT-7117-A-301
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 286 participants (actual)
- Lead sponsor
- Tanabe Pharma America, Inc.Industry (pharma, biotech, devices)
- Study start
- 10 Aug 2021 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 16 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the long-term safety and tolerability of oral dersimelagon.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dersimelagon 200mg | Experimental | Drug: MT-7117 |
Who may qualify
- Ages
- 12 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 1. Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided.
- 2. 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.
- 3. Subjects are willing and able to travel to the study sites for all scheduled visits.
- 4. 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).
- 5. 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.
Key exclusion criteria (22 listed)
- 1. History or presence of photodermatoses other than EPP or XLP.
- 2. Presence of clinically significant hepatobiliary disease at Screening, determined as clinically significant by the Investigator.
- 3. 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.
- 4. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
- 5. History of melanoma.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MT-7117Also: DersimelagonMT-7117 | Drug | Dersimelagon 200mg |
Primary outcome
Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]).
Time frame: up to 66 further months
Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature), Clinical laboratory examinations (hematology, coagulation, biochemistry, urinalysis, and others), 12-lead electrocardiogram (ECG) parameters (Mean Heart Rate, PR Interval, QRS Duration, QT interval, QTcB and QTcF) will be assessed.
Number of patients with abnormal Physical examination data
Time frame: up to 66 further months
Physical examination consists of assessment of abdominal, respiratory, cardiovascular, general appearance, and others.
Number of patients with Nevi appearance
Time frame: up to 66 further months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Head of Medical Science study director
Tanabe Pharma America, Inc.
45 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing MT-7117 in people with EPP and XLP, run by Tanabe Pharma America, Inc. at 45 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 45 sites in United States, Italy, Japan, France, Spain and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]). (time frame: up to 66 further months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Sep 2026.