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Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria

Primary Purpose

Erythropoietic Protoporphyria (EPP)

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
MT-7117 low dose
MT-7117 high dose
Placebo
Sponsored by
Mitsubishi Tanabe Pharma America Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Erythropoietic Protoporphyria (EPP)

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • ACTCA, A Member of the Alliance, Inc.
  • University of California at San Francisco
  • University of Miami Miller School of Medicine
  • Metro Boston Clinical Partners, LLC
  • Ichan School of Medicine at Mount Sinai
  • Wake Forest Baptist Medical Center
  • Remington-Davis, Inc
  • University of Texas Medical Branch Porphyria Center
  • University of Utah

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

MT-7117 low dose

MT-7117 high dose

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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.
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.
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
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.
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
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.

Secondary Outcome Measures

Total Number of Sunlight Exposure Episodes With Prodromal Symptoms During 16-Week Double-Blind Treatment Period
Change From Baseline in Average Daily Mean Intensity of Prodromal Symptoms During 16-week Double-blind Treatment Period in 11-point Likert Scale
The Intensity of Prodromal Symptoms is measured by 11-point Likert scale ranges from 0 (no symptom) to 10 (greatest severity of symptom).
Change From Baseline in Average Daily Duration (Minutes) of Prodromal Symptoms at 16-Week Double-Blind Treatment Period
Change From Baseline in Pigmentation as Measured by Melanin Density for Average of 6 Skin Segments at Week 8 and Week 16.
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).
Percent Change From Baseline in Pigmentation as Measured by Melanin Density for Average of 6 Skin Segments at Week 8 and Week 16.
Total Number of Pain Events During 16-Week Double-Blind Treatment Period

Full Information

First Posted
April 23, 2018
Last Updated
May 12, 2023
Sponsor
Mitsubishi Tanabe Pharma America Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03520036
Brief Title
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Completed
Study Start Date
July 5, 2018 (Actual)
Primary Completion Date
September 28, 2019 (Actual)
Study Completion Date
September 28, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mitsubishi Tanabe Pharma America Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Erythropoietic Protoporphyria (EPP)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
102 (Actual)

8. Arms, Groups, and Interventions

Arm Title
MT-7117 low dose
Arm Type
Experimental
Arm Title
MT-7117 high dose
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
MT-7117 low dose
Intervention Description
MT-7117 low dose QD, oral, 16 weeks
Intervention Type
Drug
Intervention Name(s)
MT-7117 high dose
Intervention Description
MT-7117 high dose QD, oral, 16 weeks
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo QD, oral, 16 weeks
Primary Outcome Measure Information:
Title
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.
Description
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.
Time Frame
Baseline (Week 0) and Week 16
Title
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
Description
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.
Time Frame
Baseline (Week 0), and Week 16
Title
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
Description
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.
Time Frame
Baseline (Week 0), and Week 16
Secondary Outcome Measure Information:
Title
Total Number of Sunlight Exposure Episodes With Prodromal Symptoms During 16-Week Double-Blind Treatment Period
Time Frame
Week 16
Title
Change From Baseline in Average Daily Mean Intensity of Prodromal Symptoms During 16-week Double-blind Treatment Period in 11-point Likert Scale
Description
The Intensity of Prodromal Symptoms is measured by 11-point Likert scale ranges from 0 (no symptom) to 10 (greatest severity of symptom).
Time Frame
Baseline (Week 0), and Week 16
Title
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
Title
Change From Baseline in Pigmentation as Measured by Melanin Density for Average of 6 Skin Segments at Week 8 and Week 16.
Description
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).
Time Frame
Baseline (Week 0), Week 8, and Week 16
Title
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
Title
Total Number of Pain Events During 16-Week Double-Blind Treatment Period
Time Frame
Week 16
Other Pre-specified Outcome Measures:
Title
Total Number of Sunlight Exposure Episodes
Time Frame
Baseline (Week 0) and Week 16
Title
Change in Pigmentation as Measured by Melanin Density
Time Frame
Baseline (Week 0) and Week 16
Title
The Quality of Life as Measured by the Patient Reported Outcomes Measurement Information System (PROMIS) 57
Time Frame
Baseline (Week 0) and Week 16

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility 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).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Head of Medical Science
Organizational Affiliation
Mitsubishi Tanabe Pharma America Inc.
Official's Role
Study Director
Facility Information:
Facility Name
ACTCA, A Member of the Alliance, Inc.
City
Los Angeles
State/Province
California
ZIP/Postal Code
90036
Country
United States
Facility Name
University of California at San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
University of Miami Miller School of Medicine
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Metro Boston Clinical Partners, LLC
City
Brighton
State/Province
Massachusetts
ZIP/Postal Code
02135
Country
United States
Facility Name
Ichan School of Medicine at Mount Sinai
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Wake Forest Baptist Medical Center
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Remington-Davis, Inc
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43215
Country
United States
Facility Name
University of Texas Medical Branch Porphyria Center
City
Galveston
State/Province
Texas
ZIP/Postal Code
77555
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria

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