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New oral drug shows promise for rare sun allergy conditions

NCT ID NCT05005975

Disease control Sponsor: Tanabe Pharma America, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 10, 2026 · Updated 4 times

Summary

This study is for people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), rare conditions that cause severe pain and skin reactions to sunlight. The purpose is to check the long-term safety of an oral medication called dersimelagon. About 301 participants who completed a previous dersimelagon study will take the drug and be monitored for side effects over an extended period.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

286 people

The number who actually took part.

Started

Aug 2021

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

erythropoietic protoporphyria protoporphyria, erythropoietic, 1 X-linked erythropoietic protoporphyria X-linked lymphoproliferative disease due to SH2D1A deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

EPP XLP

Contacts and locations

Locations

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

    Colombes, 92700, France

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

    Brescia BS, 25123, Italy

  • Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo

    Cuneo CN, 12100, Italy

  • CHU Nantes

    Nantes, 44000, France

  • Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre

    Bordeaux, 33000, France

  • Charite - Universitaetsmedizin Berlin

    Berlin, 10117, Germany

  • Erasmus MC, Universitair Medisch Centrum Rotterdam

    Rotterdam, 3015 GD, Netherlands

  • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano

    Milan, 20122, Italy

  • Hamamatsu University Hospital

    Shizuoka, 431-3125, Japan

  • Haukeland University Hospital

    Bergen, N5021, Norway

  • Henry Ford Health System

    Detroit, Michigan, 48202, United States

  • Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine

    Paris, 75018, France

  • Hospital Clínic de Barcelona

    Barcelona, 8036, Spain

  • Hospital General Universitario De Valencia

    Valencia, 46014, Spain

  • Hospital Universitario

    Madrid, 28041, Spain

  • I.F.O. Hospital, Centro Porfirie e Malattie Rare

    Roma, Rome, 128, Italy

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

    New York, New York, 10029, United States

  • Institute for Clinical and Experimental Medicine - IKEM

    Prague, 140 21, Czechia

  • Instytut hematologii i Transfuzjologii

    Warsaw, 02-776, Poland

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

    Trieste TS, 34137, Italy

  • Kansas City Research Institute

    Kansas City, Missouri, 64131, United States

  • Karolinska University Hospital

    Stockholm, 14186, Sweden

  • Kobe University Hospital

    Kobe, Hyōgo, 650-0017, Japan

  • Marvel Clinical Research, LLC

    Huntington Beach, California, 92647, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • MetroBoston Clinical Partners, LLC

    Brighton, Massachusetts, 02135-3211, United States

  • Osaka Medical College Hospital

    Takatsuki, Osaka, 569-8686, Japan

  • Ospedalle Galliera

    Genova GE, 16128, Italy

  • Remington-Davis Clinical Research

    Columbus, Ohio, 43215, United States

  • Royal Melbourne Hospital (RMH)

    Parkville, Victoria, 3050, Australia

  • Salford Royal NHS Foundation Trust

    Manchester, MN, M6 8HD, United Kingdom

  • Sophia Dermatology Clinic

    Kanazawa, Ishikawa-ken, 921-8035, Japan

  • St. John's Institute of Dermatology-Guy's & St Thomas' NHS Foundation Trust

    London, SE1 7EH, United Kingdom

  • The University of Texas Medical Branch (UTMB)

    Galveston, Texas, 77555, United States

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107, United States

  • Tokyo Saiseikai Central Hospital

    Minato-ku, Tokyo, 108-0073, Japan

  • Toyama University Hospital

    Sugitani, Toyama, 930-0194, Japan

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

    Modena, 41125, Italy

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

    Sofia, 1000, Bulgaria

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

    Miami, Florida, 33136, United States

  • University of Alberta Hospital

    Edmonton, Alberta, Edmonton AB T6G 2G3, Canada

  • University of California at San Francisco - CSF Porphyria Center

    San Francisco, California, 94143, United States

  • University of Washington-Seattle Cancer Care Alliance

    Seattle, Washington, 98109, United States

  • Wake Forest University Baptist Health

    Winston-Salem, North Carolina, 27157, United States

  • Wesley Medical Research

    Brisbane, Queensland, 4066, Australia

More trials for these conditions

Other studies related to the condition(s) this trial covers.