Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
Primary Purpose
EPP, XLP
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
MT-7117
Sponsored by
About this trial
This is an interventional treatment trial for EPP
Eligibility Criteria
Inclusion Criteria:
- 1. Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided.
- 2. Subjects who complete MT-7117-G01 (complete through Week 58 [Visit 12])
- 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 can understand the nature of the study and any risks involved in participation, and is 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 childbearing potential must agree to use 2 effective methods of contraception including barrier method (especially for female subjects, one method must be highly effective method)
Exclusion Criteria:
A subject will NOT be eligible for this study if ANY of the following criteria apply:
- 1. History or presence of photodermatoses other than EPP or XLP.
- 2. Presence or history of any hepatobiliary disease at Screening, determined as clinically significant by the Investigator after the discussion with the Sponsor Medical Monitor.
- 3. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
- 4. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
- 5. History of melanoma.
- 6. Presence of melanoma and/or lesions suspicious for melanoma at Screening.
- 7. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
- 8. 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.
- 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; an estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m2 as calculated by the CKD-EPI creatinine equation (2009) for adults and by the Schwartz creatinine equation for adolescents (2009). MDRD can be used for adults per local recommendations.
- 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. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
- 13. Treatment with phototherapy or afamelanotide within 3 months before baseline (Visit 2).
- 14. Treatment with cimetidine or 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 baseline (Visit 2).
- 15. 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 baseline (Visit 2). Acute use of scheduled narcotics greater than 3 months prior to baseline, over-the-counter medications (OTCs), such as non-steroidal anti-inflammatory drugs (NSAIDs) or aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of baseline (Visit 2) are not excluded.
- 16. 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.
- 17. Previous treatment with any investigational agent other than dersimelagon within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).
Sites / Locations
- Marvel Clinical Research, LLC
- MetroBoston Clinical Partners, LLC
- Kansas City Research Institute
- Icahn School of Medicine at Mount Sinai (ISSMS)-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
- Wesley Medical Research
- Royal Melbourne Hospital (RMH)
- University of Alberta Hospital
- 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
- IFO-San Gallicano IRCCS
- Kobe University Hospital
- Sophia Dermatology Clinic
- Osaka Medical College Hospital
- Tokyo Saiseikai Central Hospital
- Toyama University Hospital
- Haukeland University Hospital
- Hospital Clínic de Barcelona
- Hospital Universitario
- Karolinska University Hospital
- Salford Royal NHS Foundation Trust
- Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Dersimelagon 200mg
Arm Description
Outcomes
Primary Outcome Measures
Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]).
Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature), Clinical laboratory examinations (hematology, coagulation, biochemistry, urinalysis, and others), 12-lead electrocardiogram (ECG) parameters (Mean Heart Rate, PR Interval, QRS Duration, QT interval, QTcB and QTcF) will be assessed.
Number of patients with abnormal Physical examination data
Physical examination consists of assessment of abdominal, respiratory, cardiovascular, general appearance, and others.
Number of patients with Nevi appearance
Secondary Outcome Measures
Full Information
NCT ID
NCT05005975
First Posted
July 28, 2021
Last Updated
July 25, 2023
Sponsor
Mitsubishi Tanabe Pharma America Inc.
1. Study Identification
Unique Protocol Identification Number
NCT05005975
Brief Title
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
Official Title
A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 10, 2021 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)
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
To evaluate the long-term safety and tolerability of oral dersimelagon.
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
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
151 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Dersimelagon 200mg
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
MT-7117
Other Intervention Name(s)
Dersimelagon
Intervention Description
MT-7117
Primary Outcome Measure Information:
Title
Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]).
Description
Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature), Clinical laboratory examinations (hematology, coagulation, biochemistry, urinalysis, and others), 12-lead electrocardiogram (ECG) parameters (Mean Heart Rate, PR Interval, QRS Duration, QT interval, QTcB and QTcF) will be assessed.
Time Frame
up to 30 further months
Title
Number of patients with abnormal Physical examination data
Description
Physical examination consists of assessment of abdominal, respiratory, cardiovascular, general appearance, and others.
Time Frame
up to 30 further months
Title
Number of patients with Nevi appearance
Time Frame
up to 30 further months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
1. Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided.
2. Subjects who complete MT-7117-G01 (complete through Week 58 [Visit 12])
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 can understand the nature of the study and any risks involved in participation, and is 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 childbearing potential must agree to use 2 effective methods of contraception including barrier method (especially for female subjects, one method must be highly effective method)
Exclusion Criteria:
A subject will NOT be eligible for this study if ANY of the following criteria apply:
1. History or presence of photodermatoses other than EPP or XLP.
2. Presence or history of any hepatobiliary disease at Screening, determined as clinically significant by the Investigator after the discussion with the Sponsor Medical Monitor.
3. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
4. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
5. History of melanoma.
6. Presence of melanoma and/or lesions suspicious for melanoma at Screening.
7. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
8. 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.
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; an estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m2 as calculated by the CKD-EPI creatinine equation (2009) for adults and by the Schwartz creatinine equation for adolescents (2009). MDRD can be used for adults per local recommendations.
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. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
13. Treatment with phototherapy or afamelanotide within 3 months before baseline (Visit 2).
14. Treatment with cimetidine or 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 baseline (Visit 2).
15. 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 baseline (Visit 2). Acute use of scheduled narcotics greater than 3 months prior to baseline, over-the-counter medications (OTCs), such as non-steroidal anti-inflammatory drugs (NSAIDs) or aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of baseline (Visit 2) are not excluded.
16. 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.
17. Previous treatment with any investigational agent other than dersimelagon 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
MetroBoston Clinical Partners, LLC
City
Brighton
State/Province
Massachusetts
ZIP/Postal Code
02135-3211
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 (ISSMS)-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
27157
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
98109
Country
United States
Facility Name
Wesley Medical Research
City
Brisbane
State/Province
Queensland
ZIP/Postal Code
4066
Country
Australia
Facility Name
Royal Melbourne Hospital (RMH)
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
University of Alberta Hospital
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
Edmonton AB T6G 2G3
Country
Canada
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 BS
ZIP/Postal Code
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
IFO-San Gallicano IRCCS
City
Rome
ZIP/Postal Code
5300144
Country
Italy
Facility Name
Kobe University Hospital
City
Kobe
State/Province
Hyogo
ZIP/Postal Code
650-0017
Country
Japan
Facility Name
Sophia Dermatology Clinic
City
Kanazawa
State/Province
Ishikawa
ZIP/Postal Code
921-8035
Country
Japan
Facility Name
Osaka Medical College Hospital
City
Takatsuki
State/Province
Osaka
ZIP/Postal Code
569-8686
Country
Japan
Facility Name
Tokyo Saiseikai Central Hospital
City
Minato-ku
State/Province
Tokyo
ZIP/Postal Code
108-0073
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
ZIP/Postal Code
N5021
Country
Norway
Facility Name
Hospital Clínic de Barcelona
City
Barcelona
ZIP/Postal Code
8036
Country
Spain
Facility Name
Hospital Universitario
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Facility Name
Karolinska University Hospital
City
Stockholm
ZIP/Postal Code
14186
Country
Sweden
Facility Name
Salford Royal NHS Foundation Trust
City
Manchester
State/Province
MN
ZIP/Postal Code
M6 8HD
Country
United Kingdom
Facility Name
Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
City
London
ZIP/Postal Code
SE1 9RT
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
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