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Study to Evaluate Efficacy, Safety, and Tolerability of MT‑7117 in Subjects With Erythropoietic 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. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03520036
Recruitment Status : Active, not recruiting
First Posted : May 9, 2018
Last Update Posted : April 30, 2019
Sponsor:
Information provided by (Responsible Party):
Mitsubishi Tanabe Pharma Development America, Inc.

Brief Summary:
The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration without symptoms and tolerance in subjects with EPP.

Condition or disease Intervention/treatment Phase
Erythropoietic Protoporphyria (EPP) Drug: MT-7117 low dose Drug: MT-7117 high dose Drug: Placebo Phase 2

Detailed Description:
This is a Phase 2, randomized, double-blind, placebo controlled study to assess the efficacy, tolerability, and safety of MT-7117 in subjects with EPP. The study consists of a 2 week screening period, a 16 week double-blind treatment period, and a 6 week follow-up period at Week 22. The total participation period is approximately 24 weeks.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 102 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT‑7117 in Subjects With Erythropoietic Protoporphyria
Actual Study Start Date : July 5, 2018
Estimated Primary Completion Date : September 2019
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
Experimental: MT-7117 low dose Drug: MT-7117 low dose
MT-7117 low dose QD, oral, 16 weeks

Experimental: MT-7117 high dose Drug: MT-7117 high dose
MT-7117 high dose QD, oral, 16 weeks

Placebo Comparator: Placebo Drug: Placebo
Placebo QD, oral, 16 weeks




Primary Outcome Measures :
  1. Change from baseline in average daily duration of sunlight exposure without symptoms [ Time Frame: Baseline (Week 0) and Week 16 ]
    Duration in minutes, of sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset


Other Outcome Measures:
  1. Total number of sunlight exposure episodes [ Time Frame: Baseline (Week 0) and Week 16 ]
  2. Change in pigmentation as measured by melanin density [ Time Frame: Baseline (Week 0) and Week 16 ]
  3. The quality of life as measured by the Patient Reported Outcomes Measurement Information System (PROMIS) 57 [ Time Frame: Baseline (Week 0) and Week 16 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 75 Years   (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.
  • 2. Male and female subjects with a confirmed diagnosis of EPP based on medical history, aged 18 years to 75 years, inclusive, at Screening.
  • 3. Subjects are willing and able to travel to the study sites for all scheduled visits.
  • 4. 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).

Exclusion Criteria:

  • 1. History or presence of photodermatoses other than EPP.
  • 2. Subjects who are unwilling or unable to go outside during daylight hours (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 or presence of melanoma and/or atypical nevus at Screening.
  • 7. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
  • 8. History or presence of pre-malignant skin lesion squamous cell carcinoma, basal cell carcinoma, or other malignant skin lesions.
  • 9. 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.
  • 10. 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 a glomerular filtration rate (GFR) <60 ml/min.
  • 11. 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.
  • 12. Pregnancy or lactation.
  • 13. Females of child bearing potential and male subjects with partners of child-bearing potential unwilling to use adequate contraception measures as described in the protocol.
  • 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 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 Randomization (Visit 2).
  • 18. Chronic treatment with prescription-based analgesic agents including but not limited to opioids and opioid derivatives such as morphine, hydrocodone, oxycodone or their combination with other analgesics or non-steroidal anti-inflammatory drug (NSAID, as Percocet and Vicodin-like prescription drugs) within 4 weeks before Randomization (Visit 2).
  • 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.
  • 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): NCT03520036


Locations
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United States, California
ACTCA, A Member of the Alliance, Inc.
Los Angeles, California, United States, 90036
University of California at San Francisco
San Francisco, California, United States, 94143
United States, Florida
University of Miami Miller School of Medicine
Miami, Florida, United States, 33136
United States, Massachusetts
Metro Boston Clinical Partners, LLC
Brighton, Massachusetts, United States, 02135
United States, New York
Ichan School of Medicine at Mount Sinai
New York, New York, United States, 10029
United States, North Carolina
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
Remington-Davis, Inc
Columbus, Ohio, United States, 43215
United States, Texas
University of Texas Medical Branch Porphyria Center
Galveston, Texas, United States, 77555
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84108
Sponsors and Collaborators
Mitsubishi Tanabe Pharma Development America, Inc.
Investigators
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Study Director: Head of Clinical Development, Mitsubishi Tanabe Pharma Development America, Inc.

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Responsible Party: Mitsubishi Tanabe Pharma Development America, Inc.
ClinicalTrials.gov Identifier: NCT03520036     History of Changes
Other Study ID Numbers: MT-7117-A01
First Posted: May 9, 2018    Key Record Dates
Last Update Posted: April 30, 2019
Last Verified: April 2019

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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