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

NCT06144840 | Active Not Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: MT-7117-A-302jRCT20312306562023-506735-15-00
• Study Type: interventional
• Target: 165
• Locations: 11 countries
• Sites: 36

Brief 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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• 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 16

  The comparison between MT-7117 treatment group and placebo group will be performed.

  Week 16

Secondary Outcomes (3)

• Patient Global Impression of Change (PGIC) at Week 16.

  Week 16

• Total number of sunlight-induced pain events defined as prodromal symptoms (burning, tingling, itching, or stinging) with pain rating of 1-10 on the Likert scale during the 16-week double-blind treatment period.

  Week 16

• Total number of sunlight-induced non-prodrome, phototoxic reactions during the 16-week double-blind treatment period.

  Week 16

Study Arms (2)

MT-7117

EXPERIMENTAL

Drug: Dersimelagon

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Dersimelagon DRUG

MT-7117

MT-7117

Placebo DRUG

Placebo

Placebo

Eligibility Criteria

• Age: 12 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects provided written informed consent to participate. For minor subjects, both minor's assent and parental consent will be required.
• Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history.
• Subjects aged 12 years to 75 years, inclusive, at Screening.
• Subjects are willing and able to travel to the study sites for all scheduled visits.
• 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).
• 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.
• Additional screening criteria check may apply for qualification.

You may not qualify if:

• History or presence of photodermatoses other than EPP or XLP.
• 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.
• 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.
• 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.
• History (in the last 2 years) or presence of alcohol abuse, or abuse of illicit drugs in the opinion of the Investigator.
• History of melanoma.
• 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 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.
• 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 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

Sponsors & Collaborators

• Tanabe Pharma America, Inc.: lead

Study Sites (36)

Marvel Clinical Research, LLC

Huntington Beach, California, 92647, United States

Location

University of California at San Francisco

San Francisco, California, 94143, United States

Location

University Of Miami School Of Medicine, Center For Liver Diseases

Miami, Florida, 33136, United States

Location

MGH

Boston, Massachusetts, 02129, United States

Location

MetroBoston Clinical Partners, LLC

Brighton, Massachusetts, 02135-3511, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Kansas City Research Institute

Kansas City, Missouri, 64131, United States

Location

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

New York, New York, 10029-0311, United States

Location

Wake Forest University Baptist Health

Winston-Salem, North Carolina, 27103, United States

Location

Remington-Davis Clinical Research

Columbus, Ohio, 43215, United States

Location

Einstein Medical Center (EMC)

Philadelphia, Pennsylvania, 19141, United States

Location

The University of Texas Medical Branch (UTMB)

Galveston, Texas, 77555-0342, United States

Location

University of Washington

Seattle, Washington, 98195, United States

Location

Royal Melbourne Hospital (RMH)

Parkville, Victoria, 03050, Australia

Location

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

Sofia, 01000, Bulgaria

Location

Institute for Clinical and Experimental Medicine - IKEM

Prague, 01958, Czechia

Location

Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre

Bordeaux, 33000, France

Location

CHU Nantes

Nantes, 44000, France

Location

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

Paris, 75018, France

Location

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

Paris, 92701, France

Location

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

Brescia, 25123, Italy

Location

Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo

Cuneo, 12100, Italy

Location

Ospedalle Galliera

Genova, 16128, Italy

Location

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano

Milan, 20122, Italy

Location

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

Modena, 41124, Italy

Location

IFO-San Gallicano IRCCS

Rome, 00144, Italy

Location

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

Trieste, 34137, Italy

Location

Mazda Hospital

Aki-gun, Hiroshima, 735-8585, Japan

Location

Hamamatsu University Hospital

Hamamatsu, Shizuoka, 431-3194, Japan

Location

Tokyo Saiseikai Central Hospital

Tokyo, 108-0073, Japan

Location

Universitair Medisch Centrum Rotterdam

Rotterdam, 03015, Netherlands

Location

Instytut Hematologii I Transfuzjologii

Warsaw, 02-776, Poland

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital General Universitario De Valencia

Valencia, 46014, Spain

Location

Southampton General Hospital - University Hospital Southampton NHS Foundation Trust

Southamption, Southamption, SO16 6YD, United Kingdom

Location

Guy's Hospital

London, SE1 9RT, United Kingdom

Location

MeSH Terms

Conditions

Protoporphyria, Erythropoietic; Protoporphyria, Erythropoietic, X-Linked Dominant

Condition Hierarchy (Ancestors)

Porphyrias, Hepatic; Liver Diseases; Digestive System Diseases; Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Porphyrias; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Head of Medical Science

  Tanabe Pharma America, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2023
• First Posted: November 22, 2023
• Study Start: December 11, 2023
• Primary Completion: April 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: December 11, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

BU (1); ES (2); US (13); JP (3); PL (1); GB (2); NL (1); AU (1); IT (7); CZ (1); FR (4)