EPPPhase 3NCT04402489
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
A Phase 3 study testing MT-7117 Low Dose and MT-7117 High Dose in people with EPP and XLP, run by Tanabe Pharma America, Inc. at 31 sites in 10 countries.
Trial summary
The primary objective of this study is 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 aged 12-75. Read more about this study
- Who is it for?
- Ages 12 to 75 years
- What is being tested?
- MT-7117 Low Dose and MT-7117 High Dose
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 184 participants
- Where
- 31 sitesin 10 countries
- Study dates
- 1 Jun 2020to 26 Jul 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Subjects provided written informed consent to participate. For minor subjects, both minor assent and parental consent will be provided.
- 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.
- Subjects have a body weight of ≥30 kg.
- Subjects are willing and able to travel to the study sites for all scheduled visits.
- In the Investigator's opinion, subject is able to 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).
- 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 including barrier method as described in the protocol.
Who cannot join
- History or presence of photodermatoses other than EPP or XLP.
- Subjects who are unwilling or unable to go outside during daylight hours most days (e.g., between 1 hour post sunrise and 1 hour pre-sunset) during the study.
- Presence of clinically significant hepatobiliary disease based on LFT values at Screening.
- Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
- Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
- History of melanoma.
Show all 23Show fewer
- Presence of melanoma and/or lesions suspicious for melanoma at Screening.
- History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
- 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.
- 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.
- 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 as calculated by the Chronic Kidney Disease-Epidemiology Collaboration (CKDEPI) creatinine equation (2009) for adults and by the Schwartz creatinine equation for adolescents (2009). Modification of Diet in Renal Disease (MDRD) can be used for adults per local recommendations.
- 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.
- Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
- Treatment with phototherapy within 3 months before Randomization (Visit 2).
- Treatment with afamelanotide within 3 months before Randomization (Visit 2).
- Treatment with cimetidine within 4 weeks before Randomization (Visit 2).
- Treatment with antioxidant agents within 4 weeks before Randomization (Visit 2), at doses which, in the opinion of the Investigator, may affect study endpoints (including but not limited to beta-carotene, cysteine, pyridoxine).
- Chronic treatment with any scheduled analgesic agents including, but not limited to, opioids and opioid derivatives such as morphine, hydrocodone, oxycodone, fentanyl, or their combination with other unscheduled analgesics or non-steroidal anti-inflammatory drug (Percocet and Vicodin-like prescription drugs) within 4 weeks before Randomization (Visit 2).
- Acute use of scheduled narcotics greater than 3 months prior to randomization, OTCs, such as NSAIDs or aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of screening are not excluded.
- 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.
- Previous exposure to MT-7117 (this does not include placebo treated subjects).
- Previous treatment with any investigational agent within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).
Trial locations and contacts
31 sites in 10 countries.
31 sites in 10 countries
United States10 sites
MetroBoston Clinical Partners, LLC
Brighton, Massachusetts, 02135
Remington-Davis Clinical Research
Columbus, Ohio, 43215
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 (ISMMS) - The Mount Sinai Hospital (MSH)
New York, New York, 10029
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
University of Washington-Seattle Cancer Care Alliance
Seattle, Washington, 19023
Wake Forest University Baptist Health
Winston-Salem, North Carolina, 27109
Japan6 sites
Sophia Dermatology Clinic
Kanazawa, Ishikawa-ken, 921-8035
Kobe University Hospital
Kobe, Hyōgo
Tokyo Saiseikai Central Hospital
Minato-ku, Tokyo
Investigator site
Sayama, Osaka
Toyama University Hospital
Sugitani, Toyama, 930-0194
Osaka Medical College Hospital
Takatsuki, Osaka
Italy4 sites
Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
Brescia, 1 25123
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
Rome, 144 Rome RM
Australia2 sites
The Wesley Hospital
Brisbane, Queensland, 4066
Royal Melbourne Hospital (RMH)
Melbourne, Victoria, 3050
Germany2 sites
Charite - Universitaetsmedizin Berlin
Berlin, 10117
Westfaelische Wilhelms-Universitaet Muenster
Münster, Northrhein Westalien, 48149
Spain2 sites
Hospital Clínic de Barcelona
Barcelona, 08036
Hospital Universitario 12 de Octubre
Madrid, 28041
United Kingdom2 sites
Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
London, SE1 7EH
University of Manchester
Salford, Manchester, M13 9PL
About this study
Official title: 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
The primary objective of this study is 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 aged 12-75.
- Being tested
- Placebo, MT-7117 Low Dose, MT-7117 High Dose
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Placebo
MT-7117 Low DoseDrug
Also called Dersimelagon
MT-7117 Low Dose
MT-7117 High DoseDrug
Also called Dersimelagon
MT-7117 High Dose
| Study group | Role | What they receive |
|---|---|---|
| Placebo Comparator | Placebo comparator | Oral tablet of placebo once a day. Receives: Drug: Placebo |
| MT-7117 Low Dose | Experimental | Oral tablet of MT-7117 Low Dose once a day. Receives: Drug: MT-7117 Low Dose |
| MT-7117 High Dose | Experimental | Oral tablet of MT-7117 High Dose once a day. Receives: Drug: MT-7117 High Dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Average Daily Sunlight Exposure Time (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 26 (Visit 7)
Measured: From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)
Secondary outcomes (5)
Patient Global Impression of Change (PGIC) at Week 26
PGIC: Scale from 1 to 7, where 7 is worse.
Measured: Week 26
Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.
The Likert scale used ranges from 0 to 10, where 0 indicates the lowest pain rating and 10 indicates the highest pain rating. Likewise, 0 indicates to best outcome and 10 indicates the worst outcome. The sum of the number of pain events with pain rating of 1 to 10 for the day is used as the number of sunlight-induced pain events in the day. The sum of the number of the pain events with pain rating of 1 to 10 in each day during the 26-week Double-blind Treatment Period is calculated as this endpoint.
Measured: During the 26-week double-blind treatment period
Change From Baseline for Total Score in the Domain of Pain Intensity in the PROMIS-57 at Week 26
Pain intensity: 0 to 10, where 10 is worst pain imaginable.
Measured: Baseline (Week 0) and Week 26
The Percentage of Subjects Who Are Responders
The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post-sunrise and 1 hour presunset defined by within-subject meaningful change of 66 minutes increase from baseline to Week 26
Measured: Week 26
Change From Baseline for Total Score in the Domain of Physical Function in the PROMIS-57 at Week 26
Physical function: 1-5, where 5 is without any difficulty.
Measured: Baseline (Week 0) and Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2020
- Study start1 Jun 2020
- Primary completion14 Dec 2021
- Study completion26 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 184 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
- NCT04402489
- Sponsor's protocol ID
- MT-7117-G01
- Other IDs
- jRCT2080225355, 2019-004226-16, 2019
- Status last verified
- Dec 2025
- First submitted
- 20 May 2020
- Last updated
- 30 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Ogasawara A, Ide R, Inoue S, Tsuda M, Teng R. Assessment of Potential Drug-Drug Interactions for Novel Oral Melanocortin-1 Receptor Agonist Dersimelagon. Pharmacol Res Perspect. 2025 Feb;13(1):e70069. doi: 10.1002/prp2.70069.
PubMed 39887900
Study at a glance
From the registry record, last updated by the sponsor on 30 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04402489
- Acronym / sponsor ID
- MT-7117-G01
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 184 participants (actual)
- Lead sponsor
- Tanabe Pharma America, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Jun 2020 (actual)
- Primary completion
- 14 Dec 2021 (actual)
- Study completion
- 26 Jul 2022 (actual)
- First posted
- 26 May 2020 (actual)
- Results
- Posted 21 Mar 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is 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 aged 12-75.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo Comparator | Placebo comparator | Drug: PlaceboOral tablet of placebo once a day. |
| MT-7117 Low Dose | Experimental | Drug: MT-7117 Low DoseOral tablet of MT-7117 Low Dose once a day. |
| MT-7117 High Dose | Experimental | Drug: MT-7117 High DoseOral tablet of MT-7117 High Dose once a day. |
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)
- Subjects provided written informed consent to participate. For minor subjects, both minor assent and parental consent will be provided.
- 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.
- Subjects have a body weight of ≥30 kg.
- Subjects are willing and able to travel to the study sites for all scheduled visits.
- In the Investigator's opinion, subject is able to 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).
Key exclusion criteria (23 listed)
- History or presence of photodermatoses other than EPP or XLP.
- Subjects who are unwilling or unable to go outside during daylight hours most days (e.g., between 1 hour post sunrise and 1 hour pre-sunset) during the study.
- Presence of clinically significant hepatobiliary disease based on LFT values at Screening.
- Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
- Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
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 |
|---|---|---|
| PlaceboPlacebo | Drug | Placebo Comparator |
| MT-7117 Low DoseAlso: DersimelagonMT-7117 Low Dose | Drug | MT-7117 Low Dose |
| MT-7117 High DoseAlso: DersimelagonMT-7117 High Dose | Drug | MT-7117 High Dose |
Primary outcome
Change From Baseline in Average Daily Sunlight Exposure Time (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 26 (Visit 7)
Time frame: From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)
5 secondary outcomes
Patient Global Impression of Change (PGIC) at Week 26
Time frame: Week 26
Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.
Time frame: During the 26-week double-blind treatment period
Change From Baseline for Total Score in the Domain of Pain Intensity in the PROMIS-57 at Week 26
Time frame: Baseline (Week 0) and Week 26
The Percentage of Subjects Who Are Responders
Time frame: Week 26
Change From Baseline for Total Score in the Domain of Physical Function in the PROMIS-57 at Week 26
Time frame: Baseline (Week 0) and Week 26
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Dec 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39887900Ogasawara A, Ide R, Inoue S, Tsuda M, Teng R. Assessment of Potential Drug-Drug Interactions for Novel Oral Melanocortin-1 Receptor Agonist Dersimelagon. Pharmacol Res Perspect. 2025 Feb;13(1):e70069. doi: 10.1002/prp2.70069.
- Individual participant dataSharing planned: Undecided
Related trials for EPP
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HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
Erythropoietic Protoporphyria
RecruitingPhase 2/3
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Active, not recruitingPhase 3
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
EPP, XLP
RecruitingObservational study
Longitudinal Study of the Porphyrias
Acute Porphyrias, Cutaneous Porphyrias
RecruitingObservational study
Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)
Erythropoietic Protoporphyria (EPP), X-Linked Porphyria (XLP)
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.
31 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing MT-7117 Low Dose and MT-7117 High Dose in people with EPP and XLP, run by Tanabe Pharma America, Inc. at 31 sites in 10 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 23 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: finished, as of the sponsor's last update on 30 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in United States, Japan, Italy, Australia, Germany and 5 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 21 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in Average Daily Sunlight Exposure Time (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 26 (Visit 7) (time frame: From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04402489, which also lists 1 linked publication.
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 30 Dec 2025.