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NCT06144840 | ACTIVE NOT RECRUITING | Erythropoietic Protoporphyria (EPP)

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

Mitsubishi Tanabe Pharma America Inc.

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.

Condition or disease

Erythropoietic Protoporphyria (EPP)

X-Linked Protoporphyria (XLP)

Intervention/treatment

Dersimelagon

Placebo

Phase

PHASE3

Study Type : INTERVENTIONAL
Estimated Enrollment : 165 participants
Masking : QUADRUPLE
Primary Purpose : TREATMENT
Official Title : A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria
Actual Study Start Date : 2023-12-11
Estimated Primary Completion Date : 2026-04
Estimated Study Completion Date : 2026-06

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 12 Years to 75 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
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.
INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

Location Details

NCT06144840

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

Not yet recruiting

United States, California

Marvel Clinical Research, LLC

Huntington Beach, California, United States, 92647

Not yet recruiting

United States, California

University of California at San Francisco

San Francisco, California, United States, 94143

Not yet recruiting

United States, Florida

University Of Miami School Of Medicine, Center For Liver Diseases

Miami, Florida, United States, 33136

Not yet recruiting

United States, Massachusetts

MGH

Boston, Massachusetts, United States, 02129

Not yet recruiting

United States, Massachusetts

MetroBoston Clinical Partners, LLC

Brighton, Massachusetts, United States, 02135-3511

Not yet recruiting

United States, Road cancer

Henry Ford Health System

Detroit, Road cancer, United States, 48202

Not yet recruiting

United States, Missouri

Kansas City Research Institute

Kansas City, Missouri, United States, 64131

Not yet recruiting

United States, New York

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

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

Not yet recruiting

United States, North Carolina

Wake Forest University Baptist Health

Winston-Salem, North Carolina, United States, 27103

Not yet recruiting

United States, Ohio

Remington-Davis Clinical Research

Columbus, Ohio, United States, 43215

Not yet recruiting

United States, Pennsylvania

Einstein Medical Center (EMC)

Philadelphia, Pennsylvania, United States, 19141

Not yet recruiting

United States, Texas

The University of Texas Medical Branch (UTMB)

Galveston, Texas, United States, 77555-0342

Not yet recruiting

United States, Washington

University of Washington

Seattle, Washington, United States, 98195

Not yet recruiting

Australia, Victoria

Royal Melbourne Hospital (RMH)

Parkville, Victoria, Australia, 03050

Not yet recruiting

Bulgaria,

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

Sofia, Bulgaria, 01000

Not yet recruiting

Czechia,

Institute for Clinical and Experimental Medicine - IKEM

Prague, Czechia, 01958

Not yet recruiting

France,

Bordeaux University Hospital Center - Hospital Saint - Andre

Bordeaux, France, 33000

Not yet recruiting

France,

Chu nantes

Nantes, France, 44000

Not yet recruiting

France,

Bichat Hospital - Bichat -University Hospital University Paris Nord Val de Seine

Paris, France, 75018

Not yet recruiting

France,

Public assistance-hopitals of Paris (AP-HP)-Louis-Mourier hospital

Paris, France, 92701

Not yet recruiting

Italy,

Civil Spedali Civil Hospital of Brescia-University of Brescia Studies

Brescia, Italy, 25123

Not yet recruiting

Italy,

HOSPITAL HOSPITAL COMPANY SANTA CROCE AND CARLE - CUNEO

Wedge, Italy, 12100

Not yet recruiting

Italy,

Galliera Hospital

Genova, Italy, 16128

Not yet recruiting

Italy,

IRCCS Foundation Ca Granda Maggiore Policlinico Hospital of Milan

Milan, Italy, 20122

Not yet recruiting

Italy,

U.O.C. Internal Medicine University Hospital Policlinico di Modena

Modena, Italy, 41124

Not yet recruiting

Italy,

IFO-SAN GALLICANO IRCCS

Rome, Italy, 00144

Not yet recruiting

Italy,

Institute of maternal -infantile scientific hospitalization and treatment (IRCCS) - Burlo Garofolo - Pediatric clinic

Trieste, Italy, 34137

Not yet recruiting

Japan, Hiroshima Prefecture

Mazda Hospital

Battery, Hiroshima-Prefecture, Japan, 735-8585

Not yet recruiting

Japan, Shizuoka

Hamamatsu University Hospital

Hamamatsu, Shizuoka, Japan, 431-3194

Not yet recruiting

Japan,

Tokyo Regeneration Meeting Battle T-RL Supplement S-PitaL

Tokyo, Japan, 108-0073

Not yet recruiting

Netherlands,

University Medical Center Rotterdam

Rotterdam, Netherlands, 03015

Not yet recruiting

Poland,

Institute of Hematology and Transfusion

Warsaw, Poland, 02-776

Not yet recruiting

Spain,

October 12 University Hospital

Madrid, Spain, 28041

Not yet recruiting

Spain,

General University Hospital of Valencia

Valencia, Spain, 46014

Not yet recruiting

United Kingdom,

Guy's Hospital

London, United Kingdom, SE1 9RT

Not yet recruiting

United Kingdom,

Southampton General Hospital - University Hospital Southampton NHS Foundation Trust

Southamption, United Kingdom, SO16 6YD