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

Last updated: April 16, 2025
Sponsor: Mitsubishi Tanabe Pharma America Inc.
Overall Status: Active - Not Recruiting

Phase

3

Condition

N/A

Treatment

Dersimelagon

Placebo

Clinical Study ID

NCT06144840
MT-7117-A-302
jRCT2031230656
2023-506735-15-00
  • Ages 12-75
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Subjects provided written informed consent to participate. For minor subjects, bothminor's assent and parental consent will be required.

  2. Male and female subjects with a confirmed diagnosis of EPP or XLP based on medicalhistory.

  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 andany risks involved in participation, and willing to cooperate and comply with theprotocol restrictions and requirements (including travel and receiving directsunlight exposure as much as possible).

  6. Female subjects who are non-lactating and have a negative urine pregnancy test atbaseline visit prior to receiving the first dose of study drug.

  7. Female subjects of childbearing potential and male subjects with partner ofchild-bearing potential currently using/willing to use 2 effective methods ofcontraception.

Additional screening criteria check may apply for qualification.

Exclusion

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 hoursmost days (e.g., between 1-hour post-sunrise and 1 hour pre-sunset) during thestudy.

  3. Presence or history of any hepatobiliary disease, including druginduced liver injuryat screening, determined as clinically significant by the Investigator after thediscussion 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 illicitdrugs in the opinion of the Investigator.

  6. History of melanoma.

  7. Presence of squamous cell carcinoma, basal cell carcinoma, or other malignant skinlesions. Any suspicious lesions or nevi will be evaluated. If the suspicious lesionor nevi cannot be resolved through biopsy or excision, the subject will be excludedfrom the study.

  8. History or presence of psychiatric disease judged to be clinically significant bythe Investigator and which may interfere with the study evaluation and/or safety ofthe subjects.

  9. Presence of clinically significant acute or chronic renal disease or subjects withan estimated glomerular filtration rate (eGFR) <60 mL/min as calculated by theChronic 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 localrecommendations.

  10. Presence of any clinically significant disease or laboratory abnormality which, inthe opinion of the Investigator, can interfere with the study objectives and/orsafety of the subjects.

  11. Female subjects who are pregnant, lactating, or intending to become pregnant duringthe study.

  12. Treatment with any of the following medications or therapy within each period beforeRandomization (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 theInvestigator, may affect study endpoints (including but not limited tobeta-carotene, cysteine, pyridoxine).

  • Chronic treatment with any scheduled analgesic agents including, but notlimited to, opioids and opioid derivatives such as morphine, hydrocodone,oxycodone, fentanyl, or their combination with other unscheduled analgesics ornon-steroidal anti-inflammatory drug (Percocet and Vicodin-like prescriptiondrugs) within 4 weeks. Note: Acute use of scheduled narcotics more than 3 months prior to randomization areallowed. Non-steroidal anti-inflammatory drug, aspirin for analgesia, or priortemporary use of scheduled agents within 3 months of screening is allowed.

  1. Dermatological treatments with any drugs or supplements which, in the opinion of theInvestigator, can interfere with the objectives of the study or safety of thesubjects at screening, such as, for example, tanning agents.

  2. Subjects who participated in any previous MT-7117 clinical studies.

  3. Previous treatment with any investigational agent such as bitopertin, within 12weeks before Screening or 5 half-lives of the investigational product (whichever islonger).

  4. 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 maintainedrelatively stable throughout the duration of the study.

  5. History of any hypersensitivity to the active ingredient and/or excipients (lactosemonohydrate, hydroxypropylcellulose, carmellose calcium, magnesium stearate,hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, ironoxide red, and iron oxide black). (EU ONLY)

  6. Subjects who are unable to swallow tablets or have diseases significantly affectingthe gastrointestinal function such as malabsorption syndrome, resection of thestomach or small bowel, bariatric surgery procedures, symptomatic inflammatory boweldisease, or partial or complete bowel obstruction.(EU ONLY)

  7. History of any hypersensitivity to the active ingredient and/or excipients containedin MT-7117 IMP (lactose monohydrate, hydroxypropyl cellulose, carmellose calcium,magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, ironoxide yellow, iron oxide red, and iron oxide black). (UK ONLY)

Additional screening criteria check may apply for qualification.

Study Design

Total Participants: 165
Treatment Group(s): 2
Primary Treatment: Dersimelagon
Phase: 3
Study Start date:
December 11, 2023
Estimated Completion Date:
June 30, 2026

Connect with a study center

  • Royal Prince Alfred Hospital

    Camperdown, New South Wales 02050
    Australia

    Site Not Available

  • Royal Melbourne Hospital (RMH)

    Parkville, Victoria 03050
    Australia

    Site Not Available

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

    Sofia, 01000
    Bulgaria

    Site Not Available

  • Institute for Clinical and Experimental Medicine - IKEM

    Prague, 01958
    Czechia

    Site Not Available

  • Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre

    Bordeaux, 33000
    France

    Site Not Available

  • CHU Nantes

    Nantes, 44000
    France

    Site Not Available

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

    Paris, 92701
    France

    Site Not Available

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

    Paris, 75018
    France

    Site Not Available

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

    Brescia, 25123
    Italy

    Site Not Available

  • Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo

    Cuneo, 12100
    Italy

    Site Not Available

  • Ospedalle Galliera

    Genova, 16128
    Italy

    Site Not Available

  • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano

    Milan, 20122
    Italy

    Site Not Available

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

    Modena, 41124
    Italy

    Site Not Available

  • IFO-San Gallicano IRCCS

    Rome, 00144
    Italy

    Site Not Available

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

    Trieste, 34137
    Italy

    Site Not Available

  • Mazda Hospital

    Aki-gun, Hiroshima-ken 735-8585
    Japan

    Site Not Available

  • Hamamatsu University Hospital

    Hamamatsu, Shizuoka 431-3194
    Japan

    Site Not Available

  • Tokyo Saiseikai Central Hospital

    Tokyo, 108-0073
    Japan

    Site Not Available

  • Universitair Medisch Centrum Rotterdam

    Rotterdam, 03015
    Netherlands

    Site Not Available

  • Instytut Hematologii I Transfuzjologii

    Warsaw, 02-776
    Poland

    Site Not Available

  • Hospital Universitario 12 de Octubre

    Madrid, 28041
    Spain

    Site Not Available

  • Hospital General Universitario De Valencia

    Valencia, 46014
    Spain

    Site Not Available

  • Guy's Hospital

    London, SE1 9RT
    United Kingdom

    Site Not Available

  • Southampton General Hospital - University Hospital Southampton NHS Foundation Trust

    Southamption, SO16 6YD
    United Kingdom

    Site Not Available

  • Marvel Clinical Research, LLC

    Huntington Beach, California 92647
    United States

    Site Not Available

  • University of California at San Francisco

    San Francisco, California 94143
    United States

    Site Not Available

  • University Of Miami School Of Medicine, Center For Liver Diseases

    Miami, Florida 33136
    United States

    Site Not Available

  • MGH

    Boston, Massachusetts 02129
    United States

    Site Not Available

  • MetroBoston Clinical Partners, LLC

    Brighton, Massachusetts 02135-3511
    United States

    Site Not Available

  • Henry Ford Health System

    Detroit, Michigan 48202
    United States

    Site Not Available

  • Kansas City Research Institute

    Kansas City, Missouri 64131
    United States

    Site Not Available

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

    New York, New York 10029-0311
    United States

    Site Not Available

  • Wake Forest University Baptist Health

    Winston-Salem, North Carolina 27103
    United States

    Site Not Available

  • Remington-Davis Clinical Research

    Columbus, Ohio 43215
    United States

    Site Not Available

  • Einstein Medical Center (EMC)

    Philadelphia, Pennsylvania 19141
    United States

    Site Not Available

  • The University of Texas Medical Branch (UTMB)

    Galveston, Texas 77555-0342
    United States

    Site Not Available

  • University of Washington

    Seattle, Washington 98195
    United States

    Site Not Available

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