Study to Evaluate Efficacy, Safety, and Tolerability of M... | NCT04402489 | Trialant
NCT04402489
Sponsor
Tanabe Pharma America, Inc.
Status
Completed
Last Update Posted
Dec 30, 2025Actual
Enrollment
184Actual
Phase
Phase 3
Conditions
EPP
XLP
Interventions
Placebo
MT-7117 Low Dose
MT-7117 High Dose
Countries
United States
Australia
Canada
Germany
Italy
Japan
Norway
Spain
Sweden
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT04402489
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
MT-7117-G01
Secondary IDs
ID
Type
Description
Link
jRCT2080225355
Registry Identifier
Japan Registry of Clinical Trials (jRCT)
2019-004226-16
EudraCT Number
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
Acronym
Not provided
Organization
Tanabe Pharma America, Inc.INDUSTRY
Status Module
Record Verification Date
Dec 2025
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Jun 1, 2020Actual
Primary Completion Date
Dec 14, 2021Actual
Completion Date
Jul 26, 2022Actual
First Submitted Date
May 20, 2020
First Submission Date that Met QC Criteria
May 20, 2020
First Posted Date
May 26, 2020Actual
Results Waived
Not provided
Results First Submitted Date
Nov 26, 2024
Results First Submitted that Met QC Criteria
Mar 18, 2025
Results First Posted Date
Mar 21, 2025Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Dec 5, 2022
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Mar 21, 2025Actual
Last Update Submitted Date
Dec 12, 2025
Last Update Posted Date
Dec 30, 2025Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Tanabe Pharma America, Inc.INDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Not provided
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
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, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP aged 12-75.
Detailed Description
Not provided
Conditions Module
Conditions
EPP
XLP
Keywords
Not provided
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 3
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
184Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Placebo Comparator
Placebo Comparator
Oral tablet of placebo once a day.
Drug: Placebo
MT-7117 Low Dose
Experimental
Oral tablet of MT-7117 Low Dose once a day.
Drug: MT-7117 Low Dose
MT-7117 High Dose
Experimental
Oral tablet of MT-7117 High Dose once a day.
Drug: MT-7117 High Dose
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Placebo
Drug
Placebo
Placebo Comparator
MT-7117 Low Dose
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
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 (Visit 7)
From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)
Secondary Outcomes
Measure
Description
Time Frame
Patient Global Impression of Change (PGIC) at Week 26
PGIC: Scale from 1 to 7, where 7 is worse.
Week 26
Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.
Other Outcomes
Not provided
Eligibility Module
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; an estimated glomerular filtration rate (eGFR) <60 mL/min as calculated by the Chronic Kidney Disease-Epidemiology Collaboration (CKDEPI) creatinine equation (2009) for adults and by the Schwartz creatinine equation for adolescents (2009). Modification of Diet in Renal Disease (MDRD) can be used for adults per local recommendations.
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).
Accepts Healthy Volunteers
No
Sex
All
Sex/Gender Based
Not provided
Sex/Gender Description
Not provided
Minimum Age
12 Years
Maximum Age
75 Years
Standard Ages
ChildAdultOlder Adult
Study Population
Not provided
Sampling Method
Not provided
Contacts/Locations Module
Central Contacts
Not provided
Overall Officials
Name
Affiliation
Role
Head of Medical Science
Tanabe Pharma America, Inc.
Study Director
Locations
Facility
Status
City
State
ZIP
Country
Contacts
Marvel Clinical Research, LLC
Huntington Beach
California
92647
United States
University Of Miami School Of Medicine, Center For Liver Diseases
Ogasawara A, Ide R, Inoue S, Tsuda M, Teng R. Assessment of Potential Drug-Drug Interactions for Novel Oral Melanocortin-1 Receptor Agonist Dersimelagon. Pharmacol Res Perspect. 2025 Feb;13(1):e70069. doi: 10.1002/prp2.70069.
See Also Links
Not provided
Available IPD Information
Not provided
IPD Sharing Statement Module
Plan to Share IPD
Undecided
Description
Not provided
Types
Not provided
Time Frame
Not provided
Access Criteria
Not provided
URL
Not provided
Results Section
Participant Flow Module
Pre-assignment Details
Not provided
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Placebo
Subjects who were randomized to receive matching oral tablet of placebo once a day in Double-Blind treatment
FG001
MT-7117 Low Dose
Subjects who were randomized to receive oral tablet of MT-7117 Low Dose once a day in Double-Blind Treatment and Double-Blind Extension
Periods
Title
Milestones
Reasons Not Completed
26 Weeks Double-Blind Treatment (DBT)
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Not provided
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot
Yes
No
No
Study Protocol
Aug 9, 2021
Nov 26, 2024
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Finland
Submission Tracking
Estimated Results First Submitted Date
Not provided
Condition Browse Module
No data available
No data is available for this block.
Intervention Browse Module
No data available
No data is available for this block.
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
Triple
Masking Description
Not provided
Who Masked
ParticipantCare ProviderInvestigator
Drug
MT-7117 Low Dose
MT-7117 Low Dose
Dersimelagon
MT-7117 High Dose
Drug
MT-7117 High Dose
MT-7117 High Dose
Dersimelagon
The Likert scale used ranges from 0 to 10, where 0 indicates the lowest pain rating and 10 indicates the highest pain rating. Likewise, 0 indicates to best outcome and 10 indicates the worst outcome. The sum of the number of pain events with pain rating of 1 to 10 for the day is used as the number of sunlight-induced pain events in the day. The sum of the number of the pain events with pain rating of 1 to 10 in each day during the 26-week Double-blind Treatment Period is calculated as this endpoint.
During the 26-week double-blind treatment period
Change From Baseline for Total Score in the Domain of Pain Intensity in the PROMIS-57 at Week 26
Pain intensity: 0 to 10, where 10 is worst pain imaginable.
Baseline (Week 0) and Week 26
The Percentage of Subjects Who Are Responders
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 presunset defined by within-subject meaningful change of 66 minutes increase from baseline to Week 26
Week 26
Change From Baseline for Total Score in the Domain of Physical Function in the PROMIS-57 at Week 26
Physical function: 1-5, where 5 is without any difficulty.
Baseline (Week 0) and Week 26
Miami
Florida
33136
United States
MetroBoston Clinical Partners, LLC
Brighton
Massachusetts
02135
United States
Kansas City Research Institute
Kansas City
Missouri
64131
United States
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
New York
New York
10029
United States
Wake Forest University Baptist Health
Winston-Salem
North Carolina
27109
United States
Remington-Davis Clinical Research
Columbus
Ohio
43215
United States
Thomas Jefferson University
Philadelphia
Pennsylvania
19107
United States
The University of Texas Medical Branch (UTMB)
Galveston
Texas
77555
United States
University of Washington-Seattle Cancer Care Alliance
Seattle
Washington
19023
United States
The Wesley Hospital
Brisbane
Queensland
4066
Australia
Royal Melbourne Hospital (RMH)
Melbourne
Victoria
3050
Australia
University of Alberta
Edmonton
Alberta
AB T6G 2R3
Canada
Westfaelische Wilhelms-Universitaet Muenster
Münster
Northrhein Westalien
48149
Germany
Charite - Universitaetsmedizin Berlin
Berlin
10117
Germany
Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
Brescia
1 25123
Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan
20122
Italy
U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
Modena
41125
Italy
I.F.O Hospital Centro Porfirie e Malattie Rare
Rome
144 Rome RM
Italy
Kobe University Hospital
Kobe
Hyōgo
Japan
Sophia Dermatology Clinic
Kanazawa
Ishikawa-ken
921-8035
Japan
Investigator site
Sayama
Osaka
Japan
Osaka Medical College Hospital
Takatsuki
Osaka
Japan
Tokyo Saiseikai Central Hospital
Minato-ku
Tokyo
Japan
Toyama University Hospital
Sugitani
Toyama
930-0194
Japan
Haukeland University Hospital
Bergen
Norway
Hospital Clínic de Barcelona
Barcelona
08036
Spain
Hospital Universitario 12 de Octubre
Madrid
28041
Spain
Karolinska University Hospital
Stockholm
171 64
Sweden
University of Manchester
Salford
Manchester
M13 9PL
United Kingdom
Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust
London
SE1 7EH
United Kingdom
FG002
MT-7117 High Dose
Subjects who were randomized to receive oral tablet of MT-7117 High Dose once a day in Double-Blind Treatment and Double-Blind Extension
FG003
Placebo -> MT-7117 Low Dose
Subjects who received placebo in Double-Blind Treatment, and after that received MT-7117 low dose in Double-Blind Extension
FG004
Placebo -> MT-7117 High Dose
Subjects who received placebo in Double-Blind Treatment, and after that received MT-7117 high dose in Double-Blind Extension
FG00061 subjects
FG00163 subjects
FG00260 subjects
FG0030 subjects
FG0040 subjects
COMPLETED
FG00057 subjects
FG00160 subjects
FG00258 subjects
FG0030 subjects
FG0040 subjects
NOT COMPLETED
FG0004 subjects
FG0013 subjects
FG0022 subjects
FG0030 subjects
FG0040 subjects
Type
Comment
Reasons
Adverse Event
FG0000 subjects
FG0011 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
Physician Decision
FG0001 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Withdrawal by Subject
FG0003 subjects
FG0012 subjects
FG0022 subjects
FG0030 subjects
FG004
26 Weeks Double-Blind Extension (DBE)
Type
Comment
Milestone Data
STARTED
FG0000 subjectsOf 57 participants randomized to placebo in 26 weeks double-blind period, 28 were re-randomized to MT-7117 low dose, and 28 were re-randomized to MT-7117 high dose.
1 participant did not take the offer to participate in period 2
FG00156 subjects4 participants did not take the offer to participate in period 2
FG00255 subjects3 participants did not take the offer to participate in period 2
FG00328 subjectsOf 57 participants who were randomized to placebo in period 1, 28 participants were allocated to MT-7117 low dose in period 2
FG00428 subjectsOf 57 participants who were randomized to placebo in period 1, 28 participants were allocated to MT-7117 high dose in period 2
COMPLETED
FG0000 subjects
FG00155 subjects
FG00252 subjects
FG00327 subjects
FG004
NOT COMPLETED
FG0000 subjects
FG0011 subjects
FG0023 subjects
FG0031 subjects
FG004
Type
Comment
Reasons
Adverse Event
FG0000 subjects
FG0010 subjects
FG0021 subjects
FG003
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Placebo
Subjects who were randomized to receive matching oral tablet of placebo once a day in Double-Blind treatment
BG001
MT-7117 Low Dose
Subjects who were randomized to receive oral tablet of MT-7117 Low Dose once a day in Double-Blind Treatment and Double-Blind Extension
BG002
MT-7117 High Dose
Subjects who were randomized to receive oral tablet of MT-7117 High Dose once a day in Double-Blind Treatment and Double-Blind Extension
BG003
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00061
BG00163
BG00260
BG003184
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Categorical
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
<=18 years
BG00012
BG00113
BG00212
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG00030
BG00130
BG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0007
BG0017
BG002
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
American Indian or Alaska Native
BG0000
BG0010
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
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 (Visit 7)
Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.
Posted
Least Squares Mean
Standard Error
minute
From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)
ID
Title
Description
OG000
DBT ITT1 Placebo
Oral tablet of placebo once a day in DBT
OG001
DBT ITT1 MT- 7117 Low Dose
Oral tablet of MT-7117 Low Dose once a day in DBT
OG002
DBT ITT1 MT- 7117 High Dose
Oral tablet of MT-7117 High Dose once a day in DBT
Units
Counts
Participants
OG00060
OG00163
OG00260
Title
Denominators
Categories
Title
Measurements
OG00020.59± 10.29
OG00130.39± 10.04
OG00243.29± 10.13
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Change from BL at Week 26 (DBT EOT) - lower dose
Mixed-effect model for repeated measures
0.496
Least Square Mean Difference vs Placebo
9.8
Standard Error of the Mean
14.35
2-Sided
95
-18.52
38.12
Superiority
OG000
OG002
Change from BL at Week 26 (DBT EOT) - higher dose
Secondary
Patient Global Impression of Change (PGIC) at Week 26
PGIC: Scale from 1 to 7, where 7 is worse.
Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.
Posted
Least Squares Mean
Standard Error
point
Week 26
ID
Title
Description
OG000
DBT ITT1 Placebo
Oral tablet of placebo once a day in DBT
OG001
DBT ITT1 MT- 7117 Low Dose
Oral tablet of MT-7117 Low Dose once a day in DBT
OG002
DBT ITT1 MT- 7117 High Dose
Oral tablet of MT-7117 High Dose once a day in DBT
Units
Counts
Participants
OG000
Secondary
Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.
The Likert scale used ranges from 0 to 10, where 0 indicates the lowest pain rating and 10 indicates the highest pain rating. Likewise, 0 indicates to best outcome and 10 indicates the worst outcome. The sum of the number of pain events with pain rating of 1 to 10 for the day is used as the number of sunlight-induced pain events in the day. The sum of the number of the pain events with pain rating of 1 to 10 in each day during the 26-week Double-blind Treatment Period is calculated as this endpoint.
Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.
Posted
Geometric Mean
Standard Deviation
events
During the 26-week double-blind treatment period
ID
Title
Description
OG000
DBT ITT1 Placebo
Oral tablet of placebo once a day in DBT
OG001
DBT ITT1 MT- 7117 Low Dose
Oral tablet of MT-7117 Low Dose once a day in DBT
OG002
DBT ITT1 MT- 7117 High Dose
Secondary
Change From Baseline for Total Score in the Domain of Pain Intensity in the PROMIS-57 at Week 26
Pain intensity: 0 to 10, where 10 is worst pain imaginable.
Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.
Posted
Least Squares Mean
Standard Error
total score
Baseline (Week 0) and Week 26
ID
Title
Description
OG000
DBT ITT1 Placebo
Oral tablet of placebo once a day in DBT
OG001
DBT ITT1 MT- 7117 Low Dose
Oral tablet of MT-7117 Low Dose once a day in DBT
OG002
DBT ITT1 MT- 7117 High Dose
Oral tablet of MT-7117 High Dose once a day in DBT
Units
Counts
Participants
Secondary
The Percentage of Subjects Who Are Responders
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 presunset defined by within-subject meaningful change of 66 minutes increase from baseline to Week 26
Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.
Posted
Number
percentage of participants
Week 26
ID
Title
Description
OG000
DBT ITT1 Placebo
Oral tablet of placebo once a day in DBT
OG001
DBT ITT1 MT- 7117 Low Dose
Oral tablet of MT-7117 Low Dose once a day in DBT
OG002
DBT ITT1 MT- 7117 High Dose
Oral tablet of MT-7117 High Dose once a day in DBT
Units
Counts
Secondary
Change From Baseline for Total Score in the Domain of Physical Function in the PROMIS-57 at Week 26
Physical function: 1-5, where 5 is without any difficulty.
Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.
Posted
Least Squares Mean
Standard Error
total score
Baseline (Week 0) and Week 26
ID
Title
Description
OG000
DBT ITT1 Placebo
Oral tablet of placebo once a day in DBT
OG001
DBT ITT1 MT- 7117 Low Dose
Oral tablet of MT-7117 Low Dose once a day in DBT
OG002
DBT ITT1 MT- 7117 High Dose
Oral tablet of MT-7117 High Dose once a day in DBT
Units
Counts
Participants
Time Frame
through study completion, an average of 58 weeks
Description
Total Number At Risk of Adverse event is the number of patients who enrolled in DBT or DBE period and who received at least 1 dose of study medication during the period. For "DBE: MT-7117 high dose", 55 patients were enrolled in DBE period and 54 patients received at least 1 dose of study medication during DBE period. Therefore, 54 patients are the Total Number At Risk for "DBE: MT-7117 high dose".
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
DBT: Placebo
Subjects who were randomized to receive matching oral tablet of placebo once a day in Double-Blind Treatment
0
61
0
61
30
61
EG001
DBT: MT-7117 Low Dose
Subjects who were randomized to receive oral tablet of MT-7117 Low Dose once a day in Double-Blind Treatment
0
63
2
63
35
63
EG002
DBT: MT-7117 High Dose
Subjects who were randomized to receive oral tablet of MT-7117 High Dose once a day in Double-Blind Treatment
1
60
2
60
40
60
EG003
DBE: MT-7117 Low Dose Switched From Placebo
Subjects who received MT-7117 low dose in Double-Blind Extension The subjects were switched from Placebo for Double-Blind Extension.
0
28
2
28
19
28
EG004
DBE: MT-7117 High Dose Switched From Placebo
Subjects who received MT-7117 high dose in Double-Blind Extension. The subjects were switched from Placebo for Double-Blind Extension.
0
28
1
28
18
28
EG005
DBE: MT-7117 Low Dose
Subjects who received oral tablet of MT-7117 Low Dose once a day in Double-Blind Extension
0
56
1
56
24
56
EG006
DBE: MT-7117 High Dose
Subjects who were received oral tablet of MT-7117 High Dose once a day in Double-Blind Extension
0
54
2
54
21
54
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Myocarditis
Cardiac disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0011 affected63 at risk
EG0020 affected60 at risk
EG0030 affected28 at risk
EG0040 affected28 at risk
EG0050 affected56 at risk
EG0060 affected54 at risk
Pericarditis
Cardiac disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0020 affected60 at risk
EG003
Porphyria non-acute
Congenital, familial and genetic disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0020 affected60 at risk
EG003
Oesophagitis
Gastrointestinal disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0020 affected60 at risk
EG003
Cholestasis
Hepatobiliary disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0021 affected60 at risk
EG003
Meningitis viral
Infections and infestations
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0011 affected63 at risk
EG0020 affected60 at risk
EG003
Fibula fracture
Injury, poisoning and procedural complications
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0020 affected60 at risk
EG003
Hepatic enzyme increased
Investigations
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0020 affected60 at risk
EG003
Asthma
Respiratory, thoracic and mediastinal disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0021 affected60 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0020 affected60 at risk
EG0030 affected28 at risk
EG0042 affected28 at risk
EG0050 affected56 at risk
EG0060 affected54 at risk
Porphyria non-acute
Congenital, familial and genetic disorders
MedDRA 23.1
Systematic Assessment
EG0005 affected61 at risk
EG0011 affected63 at risk
EG0021 affected60 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA 23.1
Systematic Assessment
EG0001 affected61 at risk
EG0011 affected63 at risk
EG0024 affected60 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA 23.1
Systematic Assessment
EG0001 affected61 at risk
EG0015 affected63 at risk
EG0028 affected60 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA 23.1
Systematic Assessment
EG0004 affected61 at risk
EG0014 affected63 at risk
EG00217 affected60 at risk
EG003
Cyst
General disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0020 affected60 at risk
EG003
Fatigue
General disorders
MedDRA 23.1
Systematic Assessment
EG0004 affected61 at risk
EG0013 affected63 at risk
EG0023 affected60 at risk
EG003
COVID-19
Infections and infestations
MedDRA 23.1
Systematic Assessment
EG0002 affected61 at risk
EG0015 affected63 at risk
EG0021 affected60 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA 23.1
Systematic Assessment
EG0001 affected61 at risk
EG0011 affected63 at risk
EG0021 affected60 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA 23.1
Systematic Assessment
EG0005 affected61 at risk
EG0011 affected63 at risk
EG0022 affected60 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0011 affected63 at risk
EG0021 affected60 at risk
EG003
Alanine aminotransferase increased
Investigations
MedDRA 23.1
Systematic Assessment
EG0001 affected61 at risk
EG0013 affected63 at risk
EG0020 affected60 at risk
EG003
Aspartate aminotransferase increased
Investigations
MedDRA 23.1
Systematic Assessment
EG0001 affected61 at risk
EG0014 affected63 at risk
EG0020 affected60 at risk
EG003
Blood creatine phosphokinase increased
Investigations
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0011 affected63 at risk
EG0021 affected60 at risk
EG003
Gamma-glutamyltransferase increased
Investigations
MedDRA 23.1
Systematic Assessment
EG0005 affected61 at risk
EG0013 affected63 at risk
EG0021 affected60 at risk
EG003
SARS-CoV-2 test positive
Investigations
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0020 affected60 at risk
EG003
Vitamin D decreased
Investigations
MedDRA 23.1
Systematic Assessment
EG0001 affected61 at risk
EG0010 affected63 at risk
EG0020 affected60 at risk
EG003
Iron deficiency
Metabolism and nutrition disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0011 affected63 at risk
EG0020 affected60 at risk
EG003
Vitamin B12 deficiency
Metabolism and nutrition disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0010 affected63 at risk
EG0020 affected60 at risk
EG003
Vitamin D deficiency
Metabolism and nutrition disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0011 affected63 at risk
EG0020 affected60 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 23.1
Systematic Assessment
EG0000 affected61 at risk
EG0011 affected63 at risk
EG0021 affected60 at risk
EG003
Melanocytic naevus
Neoplasms benign, malignant and unspecified (incl cysts and polyps)