Summary

Eligibility
for people ages 12-75 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around

Description

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.

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

Keywords

Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP), Erythropoietic Protoporphyria, X-Linked Genetic Diseases, Dersimelagon

Eligibility

You can join if…

Open to people ages 12-75

  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.

You CAN'T join if...

  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.

    Additional screening criteria check may apply for qualification.

Locations

  • University of California at San Francisco not yet accepting patients
    San Francisco California 94143 United States
  • Marvel Clinical Research, LLC accepting new patients
    Huntington Beach California 92647 United States
  • Remington-Davis Clinical Research accepting new patients
    Columbus Ohio 43215 United States

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
Mitsubishi Tanabe Pharma America Inc.
ID
NCT06144840
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 150 study participants
Last Updated