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Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoetic Protoporphyria or X-Linked Protoporphyria

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04402489
Recruitment Status : Recruiting
First Posted : May 26, 2020
Last Update Posted : June 23, 2020
Sponsor:
Information provided by (Responsible Party):
Mitsubishi Tanabe Pharma Development America, Inc.

Brief Summary:
The primary objective of this study is to investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, or stinging) associated with sunlight exposure in subjects with EPP or XLP aged 12-75.

Condition or disease Intervention/treatment Phase
EPP XLP Drug: Placebo Drug: MT-7117 Low Dose Drug: MT-7117 High Dose Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 159 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
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 : June 1, 2020
Estimated Primary Completion Date : September 2021
Estimated Study Completion Date : May 2022


Arm Intervention/treatment
Placebo Comparator: Placebo Comparator
Oral tablet of placebo once a day.
Drug: Placebo
Placebo

Experimental: MT-7117 Low Dose
Oral tablet of MT-7117 Low Dose once a day.
Drug: MT-7117 Low Dose
MT-7117 Low Dose
Other Name: Dersimelagon

Experimental: MT-7117 High Dose
Oral tablet of MT-7117 High Dose once a day.
Drug: MT-7117 High Dose
MT-7117 High Dose
Other Name: Dersimelagon




Primary Outcome Measures :
  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 26. [ Time Frame: Baseline (Week 0) and 26 weeks ]

Secondary Outcome Measures :
  1. Patient Global Impression of Change (PGIC). [ Time Frame: Week 26 ]
    PGIC: Scale from 1 to 7, where 7 is worse.

  2. Total number of sunlight-induced pain events with pain rating of 1-10 on the Likert scale during the 26-week double-blind treatment period. [ Time Frame: Baseline (Week 0) and Week 26 ]

Other Outcome Measures:
  1. Change from baseline for total score in the domain of pain intensity in the PROMIS-57. [ Time Frame: Baseline (Week 0) and Week 26 ]
    Pain intensity: 0 to 10, where 10 is worst pain imaginable.

  2. The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset defined by within-subject meaningful change. [ Time Frame: Week 26 ]
  3. Change from baseline for total score in the domain of physical function in the PROMIS-57. [ Time Frame: Baseline (Week 0) and Week 26 ]
    Physical function: 1-5, where 5 is without any difficulty.



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Ages Eligible for Study:   12 Years to 75 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Additional screening criteria check may apply for qualification.

Inclusion Criteria:

  1. Subjects provided written informed consent to participate. For minor subjects, both minor assent and parental consent will be provided.
  2. Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history, aged 12 years to 75 years, inclusive, at Screening.
  3. Subjects have a body weight of ≥30 kg.
  4. Subjects are willing and able to travel to the study sites for all scheduled visits.
  5. In the Investigator's opinion, subject is able to 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).
  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 including barrier method as described in the protocol.

Exclusion Criteria:

  1. History or presence of photodermatoses other than EPP or XLP.
  2. Subjects who are unwilling or unable to go outside during daylight hours most days (e.g., between 1 hour post sunrise and 1 hour pre-sunset) during the study.
  3. Presence of clinically significant hepatobiliary disease based on LFT values at Screening.
  4. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
  5. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
  6. History of melanoma.
  7. Presence of melanoma and/or lesions suspicious for melanoma at Screening.
  8. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
  9. 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.

  10. 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.
  11. Presence of clinically significant acute or chronic renal disease based upon the subject's medical records including hemodialysis; and a serum creatinine level of greater than 1.2 mg/dL or an estimated glomerular filtration rate (eGFR) <60 ml/min.
  12. 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.
  13. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
  14. Treatment with phototherapy within 3 months before Randomization (Visit 2).
  15. Treatment with afamelanotide within 3 months before Randomization (Visit 2).
  16. Treatment with cimetidine within 4 weeks before Randomization (Visit 2).
  17. Treatment with antioxidant agents within 4 weeks before Randomization (Visit 2), at doses which, in the opinion of the Investigator, may affect study endpoints (including but not limited to beta-carotene, cysteine, pyridoxine).
  18. 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 before Randomization (Visit 2).

    Acute use of scheduled narcotics greater than 3 months prior to randomization, OTCs, such as NSAIDs or aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of screening are not excluded.

  19. 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.
  20. Previous exposure to MT-7117 (this does not include placebo treated subjects).
  21. Previous treatment with any investigational agent within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04402489


Contacts
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Contact: Clinical Trials Information Desk, to prevent miscommunication, please email: information@mt-pharma-us.com

Locations
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United States, Florida
University Of Miami School Of Medicine, Center For Liver Diseases Not yet recruiting
Miami, Florida, United States, 33136
United States, Massachusetts
Newton-Brighton Dermatology Associates Recruiting
Brighton, Massachusetts, United States, 02135
United States, Missouri
Kansas City Research Institute Not yet recruiting
Kansas City, Missouri, United States, 64131
United States, New York
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) Not yet recruiting
New York, New York, United States, 10029
United States, North Carolina
Wake Forest University Baptist Health Not yet recruiting
Winston-Salem, North Carolina, United States, 27103
United States, Ohio
Remington-Davis Clinical Research Recruiting
Columbus, Ohio, United States, 43215
United States, Pennsylvania
Thomas Jefferson University Not yet recruiting
Philadelphia, Pennsylvania, United States, 19107
United States, Texas
The University of Texas Medical Branch (UTMB) Not yet recruiting
Galveston, Texas, United States, 77555
United States, Washington
University of Washington-Seattle Cancer Care Alliance Not yet recruiting
Seattle, Washington, United States, 98133
Canada, Alberta
University of Alberta Not yet recruiting
Edmonton, Alberta, Canada, AB T6G 2R3
Sponsors and Collaborators
Mitsubishi Tanabe Pharma Development America, Inc.
Investigators
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Study Director: Head of Medical Science Mitsubishi Tanabe Pharma Development America, Inc.
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Responsible Party: Mitsubishi Tanabe Pharma Development America, Inc.
ClinicalTrials.gov Identifier: NCT04402489    
Other Study ID Numbers: MT-7117-G01
First Posted: May 26, 2020    Key Record Dates
Last Update Posted: June 23, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Porphyrias
Metabolic Diseases