- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03520036
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
February 23, 2024 updated by: Mitsubishi Tanabe Pharma America Inc.
A Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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.
Study Type
Interventional
Enrollment (Actual)
102
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
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California
-
Los Angeles, California, United States, 90036
- ACTCA, A Member of the Alliance, Inc.
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San Francisco, California, United States, 94143
- University of California at San Francisco
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Florida
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Miami, Florida, United States, 33136
- University of Miami Miller School of Medicine
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Massachusetts
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Brighton, Massachusetts, United States, 02135
- Metro Boston Clinical Partners, LLC
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New York
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New York, New York, United States, 10029
- Ichan School of Medicine at Mount Sinai
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North Carolina
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Winston-Salem, North Carolina, United States, 27157
- Wake Forest Baptist Medical Center
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Ohio
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Columbus, Ohio, United States, 43215
- Remington-Davis, Inc
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Texas
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Galveston, Texas, United States, 77555
- University of Texas Medical Branch Porphyria Center
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Utah
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Salt Lake City, Utah, United States, 84108
- University of Utah
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-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 75 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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).
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: Placebo
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Placebo QD, oral, 16 weeks
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Experimental: MT-7117 low dose
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MT-7117 low dose QD, oral, 16 weeks
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Experimental: MT-7117 high dose
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MT-7117 high dose QD, oral, 16 weeks
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change From Baseline in Average Daily Time (Minutes) to First Prodromal Symptom Associated With Sunlight Exposure Between Hour Post Sunrise and 1 Hour Pre-Sunset at Week 16.
Time Frame: Baseline (Week 0) and Week 16
|
Duration in minutes, of sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset.
The average Duration in minutes, of sunlight exposure before the first prodromal symptom between 1 hour post sunrise and 1 hour pre-sunset.
The average duration means that average of daily durations in 14-day windows before Day 1 (or week 16 Day) applied.
|
Baseline (Week 0) and Week 16
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Change From Baseline in Average Daily Duration (Minutes) of Sunlight Exposure Between 1 Hour Post Sunrise and 1 Hour Pre-Sunset Without Prodromal Symptoms at Week 16
Time Frame: Baseline (Week 0), and Week 16
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Change from baseline to week 16 in Average Daily Duration (Minutes) of Sunlight Exposure sums any sunlight exposure time excluding any overlapped time with prodromal symptoms, including if the patients go out multiple times on the same day after the prodromal symptom had previously ended.
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Baseline (Week 0), and Week 16
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Change From Baseline in Average Daily Mean Duration (Minutes) of Sunlight Exposure Between 1 Hour Post Sunrise and 1 Hour Pre-Sunset Without Prodromal Symptoms at Week 16
Time Frame: Baseline (Week 0), and Week 16
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Change from baseline to week 16 in Average Daily Mean Duration (Minutes) of Sunlight Exposure without prodromal symptoms divided by the number of sunlight exposures periods applicable that day.
|
Baseline (Week 0), and Week 16
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Total Number of Sunlight Exposure Episodes With Prodromal Symptoms During 16-Week Double-Blind Treatment Period
Time Frame: Week 16
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Week 16
|
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Change From Baseline in Average Daily Mean Intensity of Prodromal Symptoms During 16-week Double-blind Treatment Period in 11-point Likert Scale
Time Frame: Baseline (Week 0), and Week 16
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The Intensity of Prodromal Symptoms is measured by 11-point Likert scale ranges from 0 (no symptom) to 10 (greatest severity of symptom).
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Baseline (Week 0), and Week 16
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Change From Baseline in Average Daily Duration (Minutes) of Prodromal Symptoms at 16-Week Double-Blind Treatment Period
Time Frame: Baseline (Week 0), and Week 16
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Baseline (Week 0), and Week 16
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Change From Baseline in Pigmentation as Measured by Melanin Density for Average of 6 Skin Segments at Week 8 and Week 16.
Time Frame: Baseline (Week 0), Week 8, and Week 16
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Pigmentation will be assessed in melanin density which are numeric scores measured by spectrophotometer on 6 skin segments (forehead, left cheek, right inside upper arm, left medial forearm, right-hand side of abdomen, and left-hand side of buttock).
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Baseline (Week 0), Week 8, and Week 16
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Percent Change From Baseline in Pigmentation as Measured by Melanin Density for Average of 6 Skin Segments at Week 8 and Week 16.
Time Frame: Baseline (Week 0), Week 8, and Week 16
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Baseline (Week 0), Week 8, and Week 16
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Total Number of Pain Events During 16-Week Double-Blind Treatment Period
Time Frame: Week 16
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Week 16
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Other Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Total Number of Sunlight Exposure Episodes
Time Frame: Baseline (Week 0) and Week 16
|
Baseline (Week 0) and Week 16
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Change in Pigmentation as Measured by Melanin Density
Time Frame: Baseline (Week 0) and Week 16
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Baseline (Week 0) and Week 16
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The Quality of Life as Measured by the Patient Reported Outcomes Measurement Information System (PROMIS) 57
Time Frame: Baseline (Week 0) and Week 16
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Baseline (Week 0) and Week 16
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Study Director: Head of Medical Science, Mitsubishi Tanabe Pharma America Inc.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
July 5, 2018
Primary Completion (Actual)
September 28, 2019
Study Completion (Actual)
September 28, 2019
Study Registration Dates
First Submitted
April 23, 2018
First Submitted That Met QC Criteria
May 8, 2018
First Posted (Actual)
May 9, 2018
Study Record Updates
Last Update Posted (Actual)
February 28, 2024
Last Update Submitted That Met QC Criteria
February 23, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- MT-7117-A01
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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