Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

September 7, 2026 updated by: Tanabe Pharma America, Inc.

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
286

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Australia
    • Queensland
      • Brisbane, Queensland, Australia, 4066
        • Wesley Medical Research
    • Victoria
      • Parkville, Victoria, Australia, 3050
        • Royal Melbourne Hospital (RMH)
  • Bulgaria
      • Sofia, Bulgaria, 1000
        • University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, Edmonton AB T6G 2G3
        • University of Alberta Hospital
  • Czechia
      • Prague, Czechia, 140 21
        • Institute for Clinical and Experimental Medicine - IKEM
  • France
      • Bordeaux, France, 33000
        • Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre
      • Colombes, France, 92700
        • Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier
      • Nantes, France, 44000
        • CHU Nantes
      • Paris, France, 75018
        • Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine
  • Germany
      • Berlin, Germany, 10117
        • Charite - Universitaetsmedizin Berlin
  • Italy
      • Brescia BS, Italy, 25123
        • Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
      • Cuneo CN, Italy, 12100
        • Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo
      • Genova GE, Italy, 16128
        • Ospedalle Galliera
      • Milan, Italy, 20122
        • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
      • Modena, Italy, 41125
        • U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
      • Trieste TS, Italy, 34137
        • Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica
    • Rome
      • Roma, Rome, Italy, 128
        • I.F.O. Hospital, Centro Porfirie e Malattie Rare
  • Japan
      • Shizuoka, Japan, 431-3125
        • Hamamatsu University Hospital
    • Hyōgo
      • Kobe, Hyōgo, Japan, 650-0017
        • Kobe University Hospital
    • Ishikawa-ken
      • Kanazawa, Ishikawa-ken, Japan, 921-8035
        • Sophia Dermatology Clinic
    • Osaka
      • Takatsuki, Osaka, Japan, 569-8686
        • Osaka Medical College Hospital
    • Tokyo
      • Minato-ku, Tokyo, Japan, 108-0073
        • Tokyo Saiseikai Central Hospital
    • Toyama
      • Sugitani, Toyama, Japan, 930-0194
        • Toyama University Hospital
  • Netherlands
      • Rotterdam, Netherlands, 3015 GD
        • Erasmus MC, Universitair Medisch Centrum Rotterdam
  • Norway
      • Bergen, Norway, N5021
        • Haukeland University Hospital
  • Poland
      • Warsaw, Poland, 02-776
        • Instytut Hematologii i Transfuzjologii
  • Spain
      • Barcelona, Spain, 8036
        • Hospital Clinic de Barcelona
      • Madrid, Spain, 28041
        • Hospital Universitario
      • Valencia, Spain, 46014
        • Hospital General Universitario De Valencia
  • Sweden
      • Stockholm, Sweden, 14186
        • Karolinska University Hospital
  • United Kingdom
      • London, United Kingdom, SE1 7EH
        • St. John's Institute of Dermatology-Guy's & St Thomas' NHS Foundation Trust
    • MN
      • Manchester, MN, United Kingdom, M6 8HD
        • Salford Royal NHS Foundation Trust
  • United States
    • California
      • Huntington Beach, California, United States, 92647
        • Marvel Clinical Research, LLC
      • San Francisco, California, United States, 94143
        • University of California at San Francisco - CSF Porphyria Center
    • Florida
      • Miami, Florida, United States, 33136
        • University Of Miami School Of Medicine, Center For Liver Diseases
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
      • Brighton, Massachusetts, United States, 02135-3211
        • MetroBoston Clinical Partners, LLC
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Henry Ford Health System
    • Missouri
      • Kansas City, Missouri, United States, 64131
        • Kansas City Research Institute
    • New York
      • New York, New York, United States, 10029
        • Icahn School of Medicine at Mount Sinai (ISSMS)-The Mount Sinai Hospital (MSH)
    • North Carolina
      • Winston-Salem, North Carolina, United States, 27157
        • Wake Forest University Baptist Health
    • Ohio
      • Columbus, Ohio, United States, 43215
        • Remington-Davis Clinical Research
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Thomas Jefferson University
    • Texas
      • Galveston, Texas, United States, 77555
        • The University of Texas Medical Branch (UTMB)
    • Washington
      • Seattle, Washington, United States, 98109
        • University of Washington-Seattle Cancer Care Alliance

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years to 75 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

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)

Exclusion Criteria:

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.
  • 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):

    1. 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.
    2. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Dersimelagon 200mg
Drug: MT-7117
MT-7117
Other Names:
  • Dersimelagon

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
up to 66 further months
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.
up to 66 further months
Number of patients with Nevi appearance
Time Frame: up to 66 further months
up to 66 further months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Tanabe Pharma America, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Head of Medical Science, Tanabe Pharma America, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 10, 2021

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 28, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 6, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 16, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 9, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 7, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • MT-7117-A-301
  • jRCT2041210146 (Registry Identifier: Japan Registry of Clinical Trials (jRCT))
  • 2021-001831-17 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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