INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

NCT ID: NCT06144840

Last Updated: 2025-12-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

165 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-12-11

Study Completion Date

2026-06-30

Brief Summary

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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.

Detailed Description

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Conditions

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Erythropoietic Protoporphyria (EPP) X-Linked Protoporphyria (XLP)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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MT-7117

Group Type EXPERIMENTAL

Dersimelagon

Intervention Type DRUG

MT-7117

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo

Interventions

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Dersimelagon

MT-7117

Intervention Type DRUG

Placebo

Placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Subjects provided written informed consent to participate. For minor subjects, both minor's assent and parental consent will be required.
2. Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history.
3. Subjects aged 12 years to 75 years, inclusive, at Screening.
4. Subjects are willing and able to travel to the study sites for all scheduled visits.
5. In the Investigator's opinion, subject can 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 and receiving direct sunlight exposure as much as possible).
6. 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.
7. Female subjects of childbearing potential and male subjects with partner of child-bearing potential currently using/willing to use 2 effective methods of contraception.

Additional screening criteria check may apply for qualification.

Exclusion Criteria

1. History or presence of photodermatoses other than EPP or XLP.
2. Subjects who are unwilling or unable to go outside in sunlight during daylight hours most days (e.g., between 1-hour post-sunrise and 1 hour pre-sunset) during the study.
3. Presence or history of any hepatobiliary disease, including druginduced liver injury at screening, determined as clinically significant by the Investigator after the discussion with the Sponsor Medical Monitor.
4. Subjects with aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP) ≥ 2.0 × upper limit of normal (ULN) or total bilirubin \>1.5 × ULN at Screening.
5. History (in the last 2 years) or presence of alcohol abuse, or abuse of illicit drugs in the opinion of the Investigator.
6. History of melanoma.
7. 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.
8. 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.
9. Presence of clinically significant acute or chronic renal disease or subjects with an estimated glomerular filtration rate (eGFR) \<60 mL/min as calculated by the Chronic Kidney DiseaseEpidemiology Collaboration (CKD-EPI) creatinine equation (2021) for adults and by the Schwartz creatinine equation for adolescents (2009). Modification of Diet in Renal Disease can be used for adults per local recommendations.
10. 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.
11. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
12. Treatment with any of the following medications or therapy within each period before Randomization (Visit 2);

* Afamelanotide within 3 months
* Phototherapy within 3 months
* Cimetidine within 4 weeks
* Antioxidant agents within 4 weeks, 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.

Note: Acute use of scheduled narcotics more than 3 months prior to randomization are allowed. Non-steroidal anti-inflammatory drug, aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of screening is allowed.
13. Dermatological treatments 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 at screening, such as, for example, tanning agents.
14. Subjects who participated in any previous MT-7117 clinical studies.
15. Previous treatment with any investigational agent such as bitopertin, within 12 weeks before Screening or 5 half-lives of the investigational product (whichever is longer).
16. Use of sunscreens with zinc oxide. Note: Sunscreens without zinc oxide are allowed, however their use, in frequency, quantity and body surface area should be maintained relatively stable throughout the duration of the study.
17. History of any hypersensitivity to the active ingredient and/or excipients (lactose monohydrate, hydroxypropylcellulose, carmellose calcium, magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, iron oxide red, and iron oxide black). (EU ONLY)
18. 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.(EU ONLY)
19. History of any hypersensitivity to the active ingredient and/or excipients contained in MT-7117 IMP (lactose monohydrate, hydroxypropyl cellulose, carmellose calcium, magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, iron oxide red, and iron oxide black). (UK ONLY)

Additional screening criteria check may apply for qualification.
Minimum Eligible Age

12 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Tanabe Pharma America, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Head of Medical Science

Role: STUDY_DIRECTOR

Tanabe Pharma America, Inc.

Locations

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Marvel Clinical Research, LLC

Huntington Beach, California, United States

Site Status

University of California at San Francisco

San Francisco, California, United States

Site Status

University Of Miami School Of Medicine, Center For Liver Diseases

Miami, Florida, United States

Site Status

MGH

Boston, Massachusetts, United States

Site Status

MetroBoston Clinical Partners, LLC

Brighton, Massachusetts, United States

Site Status

Henry Ford Health System

Detroit, Michigan, United States

Site Status

Kansas City Research Institute

Kansas City, Missouri, United States

Site Status

Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)

New York, New York, United States

Site Status

Wake Forest University Baptist Health

Winston-Salem, North Carolina, United States

Site Status

Remington-Davis Clinical Research

Columbus, Ohio, United States

Site Status

Einstein Medical Center (EMC)

Philadelphia, Pennsylvania, United States

Site Status

The University of Texas Medical Branch (UTMB)

Galveston, Texas, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

Royal Melbourne Hospital (RMH)

Parkville, Victoria, Australia

Site Status

University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski - Porphyria Center

Sofia, , Bulgaria

Site Status

Institute for Clinical and Experimental Medicine - IKEM

Prague, , Czechia

Site Status

Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre

Bordeaux, , France

Site Status

CHU Nantes

Nantes, , France

Site Status

Hôpital Bichat - Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine

Paris, , France

Site Status

Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier

Paris, , France

Site Status

Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia

Brescia, , Italy

Site Status

Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo

Cuneo, , Italy

Site Status

Ospedalle Galliera

Genova, , Italy

Site Status

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano

Milan, , Italy

Site Status

U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena

Modena, , Italy

Site Status

IFO-San Gallicano IRCCS

Rome, , Italy

Site Status

Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica

Trieste, , Italy

Site Status

Mazda Hospital

Aki-gun, Hiroshima, Japan

Site Status

Hamamatsu University Hospital

Hamamatsu, Shizuoka, Japan

Site Status

Tokyo Saiseikai Central Hospital

Tokyo, , Japan

Site Status

Universitair Medisch Centrum Rotterdam

Rotterdam, , Netherlands

Site Status

Instytut Hematologii I Transfuzjologii

Warsaw, , Poland

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Hospital General Universitario De Valencia

Valencia, , Spain

Site Status

Southampton General Hospital - University Hospital Southampton NHS Foundation Trust

Southamption, Southamption, United Kingdom

Site Status

Guy's Hospital

London, , United Kingdom

Site Status

Countries

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United States Australia Bulgaria Czechia France Italy Japan Netherlands Poland Spain United Kingdom

Other Identifiers

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jRCT2031230656

Identifier Type: REGISTRY

Identifier Source: secondary_id

2023-506735-15-00

Identifier Type: CTIS

Identifier Source: secondary_id

MT-7117-A-302

Identifier Type: -

Identifier Source: org_study_id

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