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

Primary Purpose

EPP, XLP

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Placebo
MT-7117 Low Dose
MT-7117 High Dose
Sponsored by
Mitsubishi Tanabe Pharma America Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for EPP

Eligibility Criteria

12 Years - 75 Years (Child, 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. 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).

Sites / Locations

  • Marvel Clinical Research, LLC
  • University Of Miami School Of Medicine, Center For Liver Diseases
  • MetroBoston Clinical Partners, LLC
  • Kansas City Research Institute
  • Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
  • Wake Forest University Baptist Health
  • Remington-Davis Clinical Research
  • Thomas Jefferson University
  • The University of Texas Medical Branch (UTMB)
  • University of Washington-Seattle Cancer Care Alliance
  • The Wesley Hospital
  • Royal Melbourne Hospital (RMH)
  • University of Alberta
  • Westfaelische Wilhelms-Universitaet Muenster
  • Charite - Universitaetsmedizin Berlin
  • Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
  • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
  • U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
  • I.F.O Hospital Centro Porfirie e Malattie Rare
  • Kobe University Hospital
  • Sophia Dermatology Clinic
  • Investigator site
  • Osaka Medical College Hospital
  • Tokyo Saiseikai Central Hospital
  • Toyama University Hospital
  • Haukeland University Hospital
  • Hospital Clínic de Barcelona
  • Hospital Universitario 12 de Octubre
  • Karolinska University Hospital
  • University of Manchester
  • Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Placebo Comparator

Experimental

Experimental

Arm Label

Placebo Comparator

MT-7117 Low Dose

MT-7117 High Dose

Arm Description

Oral tablet of placebo once a day.

Oral tablet of MT-7117 Low Dose once a day.

Oral tablet of MT-7117 High Dose once a day.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Patient Global Impression of Change (PGIC).
PGIC: Scale from 1 to 7, where 7 is worse.
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.

Full Information

First Posted
May 20, 2020
Last Updated
May 12, 2023
Sponsor
Mitsubishi Tanabe Pharma America Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04402489
Brief Title
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Completed
Study Start Date
June 1, 2020 (Actual)
Primary Completion Date
December 14, 2021 (Actual)
Study Completion Date
July 26, 2022 (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

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
EPP, XLP

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
184 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo Comparator
Arm Type
Placebo Comparator
Arm Description
Oral tablet of placebo once a day.
Arm Title
MT-7117 Low Dose
Arm Type
Experimental
Arm Description
Oral tablet of MT-7117 Low Dose once a day.
Arm Title
MT-7117 High Dose
Arm Type
Experimental
Arm Description
Oral tablet of MT-7117 High Dose once a day.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo
Intervention Type
Drug
Intervention Name(s)
MT-7117 Low Dose
Other Intervention Name(s)
Dersimelagon
Intervention Description
MT-7117 Low Dose
Intervention Type
Drug
Intervention Name(s)
MT-7117 High Dose
Other Intervention Name(s)
Dersimelagon
Intervention Description
MT-7117 High Dose
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
Patient Global Impression of Change (PGIC).
Description
PGIC: Scale from 1 to 7, where 7 is worse.
Time Frame
Week 26
Title
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 Pre-specified Outcome Measures:
Title
Change from baseline for total score in the domain of pain intensity in the PROMIS-57.
Description
Pain intensity: 0 to 10, where 10 is worst pain imaginable.
Time Frame
Baseline (Week 0) and Week 26
Title
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
Title
Change from baseline for total score in the domain of physical function in the PROMIS-57.
Description
Physical function: 1-5, where 5 is without any difficulty.
Time Frame
Baseline (Week 0) and Week 26

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Additional screening criteria check may apply for qualification. Inclusion Criteria: Subjects provided written informed consent to participate. For minor subjects, both minor assent and parental consent will be provided. 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. Subjects have a body weight of ≥30 kg. Subjects are willing and able to travel to the study sites for all scheduled visits. 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). 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. 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: History or presence of photodermatoses other than EPP or XLP. 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. Presence of clinically significant hepatobiliary disease based on LFT values at Screening. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator. History of melanoma. Presence of melanoma and/or lesions suspicious for melanoma at Screening. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child). 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. 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. 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. 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. Female subjects who are pregnant, lactating, or intending to become pregnant during the study. Treatment with phototherapy within 3 months before Randomization (Visit 2). Treatment with afamelanotide within 3 months before Randomization (Visit 2). Treatment with cimetidine within 4 weeks before Randomization (Visit 2). 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). 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. 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. Previous exposure to MT-7117 (this does not include placebo treated subjects). 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
Marvel Clinical Research, LLC
City
Huntington Beach
State/Province
California
ZIP/Postal Code
92647
Country
United States
Facility Name
University Of Miami School Of Medicine, Center For Liver Diseases
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
MetroBoston Clinical Partners, LLC
City
Brighton
State/Province
Massachusetts
ZIP/Postal Code
02135
Country
United States
Facility Name
Kansas City Research Institute
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64131
Country
United States
Facility Name
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Wake Forest University Baptist Health
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27109
Country
United States
Facility Name
Remington-Davis Clinical Research
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43215
Country
United States
Facility Name
Thomas Jefferson University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
The University of Texas Medical Branch (UTMB)
City
Galveston
State/Province
Texas
ZIP/Postal Code
77555
Country
United States
Facility Name
University of Washington-Seattle Cancer Care Alliance
City
Seattle
State/Province
Washington
ZIP/Postal Code
19023
Country
United States
Facility Name
The Wesley Hospital
City
Brisbane
State/Province
Queensland
ZIP/Postal Code
4066
Country
Australia
Facility Name
Royal Melbourne Hospital (RMH)
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
University of Alberta
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
AB T6G 2R3
Country
Canada
Facility Name
Westfaelische Wilhelms-Universitaet Muenster
City
Muenster
State/Province
Northrhein Westalien
ZIP/Postal Code
48149
Country
Germany
Facility Name
Charite - Universitaetsmedizin Berlin
City
Berlin
ZIP/Postal Code
10117
Country
Germany
Facility Name
Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
City
Brescia
ZIP/Postal Code
1 25123
Country
Italy
Facility Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
City
Milan
ZIP/Postal Code
20122
Country
Italy
Facility Name
U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
City
Modena
ZIP/Postal Code
41125
Country
Italy
Facility Name
I.F.O Hospital Centro Porfirie e Malattie Rare
City
Rome
ZIP/Postal Code
144 Rome RM
Country
Italy
Facility Name
Kobe University Hospital
City
Kobe
State/Province
Hyogo
Country
Japan
Facility Name
Sophia Dermatology Clinic
City
Kanazawa
State/Province
Ishikawa
ZIP/Postal Code
921-8035
Country
Japan
Facility Name
Investigator site
City
Osakasayama
State/Province
Osaka
Country
Japan
Facility Name
Osaka Medical College Hospital
City
Takatsuki
State/Province
Osaka
Country
Japan
Facility Name
Tokyo Saiseikai Central Hospital
City
Minato-ku
State/Province
Tokyo
Country
Japan
Facility Name
Toyama University Hospital
City
Sugitani
State/Province
Toyama
ZIP/Postal Code
930-0194
Country
Japan
Facility Name
Haukeland University Hospital
City
Bergen
Country
Norway
Facility Name
Hospital Clínic de Barcelona
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Facility Name
Karolinska University Hospital
City
Stockholm
ZIP/Postal Code
171 64
Country
Sweden
Facility Name
University of Manchester
City
Salford
State/Province
Manchester
ZIP/Postal Code
M13 9PL
Country
United Kingdom
Facility Name
Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
City
London
ZIP/Postal Code
SE1 7EH
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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

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