Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT06144840
Registration number
NCT06144840
Ethics application status
Date submitted
17/11/2023
Date registered
22/11/2023
Date last updated
13/05/2024
Titles & IDs
Public title
INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP
Query!
Scientific 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
Query!
Secondary ID [1]
0
0
jRCT2031230656
Query!
Secondary ID [2]
0
0
MT-7117-A-302
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
INSPIRE
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Erythropoietic Protoporphyria (EPP)
0
0
Query!
X-Linked Protoporphyria (XLP)
0
0
Query!
Condition category
Condition code
Skin
0
0
0
0
Query!
Other skin conditions
Query!
Blood
0
0
0
0
Query!
Other blood disorders
Query!
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Dersimelagon
Treatment: Drugs - Placebo
Experimental: MT-7117 -
Placebo Comparator: Placebo -
Treatment: Drugs: Dersimelagon
MT-7117
Treatment: Drugs: Placebo
Placebo
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
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 16
Query!
Assessment method [1]
0
0
The comparison between MT-7117 treatment group and placebo group will be performed.
Query!
Timepoint [1]
0
0
Week 16
Query!
Secondary outcome [1]
0
0
Patient Global Impression of Change (PGIC) at Week 16.
Query!
Assessment method [1]
0
0
The comparison between MT-7117 treatment group and placebo group will be performed.
Query!
Timepoint [1]
0
0
Week 16
Query!
Secondary outcome [2]
0
0
Total number of sunlight-induced pain events defined as prodromal symptoms (burning, tingling, itching, or stinging) with pain rating of 1-10 on the Likert scale during the 16-week double-blind treatment period.
Query!
Assessment method [2]
0
0
The comparison between MT-7117 treatment group and placebo group will be performed.
Query!
Timepoint [2]
0
0
Week 16
Query!
Secondary outcome [3]
0
0
Total number of sunlight-induced non-prodrome, phototoxic reactions during the 16-week double-blind treatment period.
Query!
Assessment method [3]
0
0
The comparison between MT-7117 treatment group and placebo group will be performed.
Query!
Timepoint [3]
0
0
Week 16
Query!
Eligibility
Key inclusion criteria
1. Subjects provided written informed consent to participate. For minor subjects, both
minor's assent and parental consent will be required.
2. Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical
history.
3. Subjects aged 12 years to 75 years, inclusive, at Screening.
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 willing to cooperate and comply with the protocol
restrictions and requirements (including travel and receiving direct sunlight exposure
as much as possible).
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.
Additional screening criteria check may apply for qualification.
Query!
Minimum age
12
Years
Query!
Query!
Maximum age
75
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
1. History or presence of photodermatoses other than EPP or XLP.
2. Subjects who are unwilling or unable to go outside in sunlight during daylight hours
most days (e.g., between 1-hour post-sunrise and 1 hour pre-sunset) during the study.
3. Presence or history of any hepatobiliary disease, including druginduced liver injury
at screening, determined as clinically significant by the Investigator after the
discussion with the Sponsor Medical Monitor.
4. Subjects with aspartate aminotransferase (AST), alanine aminotransferase (ALT),
alkaline phosphatase (ALP) = 2.0 × upper limit of normal (ULN) or total bilirubin >1.5
× ULN at Screening.
5. History (in the last 2 years) or presence of alcohol abuse, or abuse of illicit drugs
in the opinion of the Investigator.
6. History of melanoma.
7. 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.
8. 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.
9. Presence of clinically significant acute or chronic renal disease or subjects with an
estimated glomerular filtration rate (eGFR) <60 mL/min as calculated by the Chronic
Kidney DiseaseEpidemiology Collaboration (CKD-EPI) creatinine equation (2021) for
adults and by the Schwartz creatinine equation for adolescents (2009). Modification of
Diet in Renal Disease can be used for adults per local recommendations.
10. 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.
11. Female subjects who are pregnant, lactating, or intending to become pregnant during
the study.
12. Treatment with any of the following medications or therapy within each period before
Randomization (Visit 2);
- Afamelanotide within 3 months
- Phototherapy within 3 months
- Cimetidine within 4 weeks
- Antioxidant agents within 4 weeks, 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.
Note: Acute use of scheduled narcotics more than 3 months prior to randomization are
allowed. Non-steroidal anti-inflammatory drug, aspirin for analgesia, or prior
temporary use of scheduled agents within 3 months of screening is allowed.
13. Dermatological treatments 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
at screening, such as, for example, tanning agents.
14. Subjects who participated in any previous MT-7117 clinical studies.
15. Previous treatment with any investigational agent such as bitopertin, within 12 weeks
before Screening or 5 half-lives of the investigational product (whichever is longer).
16. Use of sunscreens with zinc oxide. Note: Sunscreens without zinc oxide are allowed,
however their use, in frequency, quantity and body surface area should be maintained
relatively stable throughout the duration of the study.
17. History of any hypersensitivity to the active ingredient and/or excipients (lactose
monohydrate, hydroxypropylcellulose, carmellose calcium, magnesium stearate,
hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, iron
oxide red, and iron oxide black). (EU ONLY)
18. Subjects who are unable to swallow tablets or have diseases significantly affecting
the gastrointestinal function such as malabsorption syndrome, resection of the stomach
or small bowel, bariatric surgery procedures, symptomatic inflammatory bowel disease,
or partial or complete bowel obstruction.(EU ONLY)
19. History of any hypersensitivity to the active ingredient and/or excipients contained
in MT-7117 IMP (lactose monohydrate, hydroxypropyl cellulose, carmellose calcium,
magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, iron
oxide yellow, iron oxide red, and iron oxide black). (UK ONLY)
Additional screening criteria check may apply for qualification.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
11/12/2023
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
1/06/2026
Query!
Actual
Query!
Sample size
Target
150
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Query!
Recruitment hospital [1]
0
0
Royal Prince Alfred Hospital - Camperdown
Query!
Recruitment hospital [2]
0
0
Royal Melbourne Hospital (RMH) - Parkville
Query!
Recruitment postcode(s) [1]
0
0
02050 - Camperdown
Query!
Recruitment postcode(s) [2]
0
0
03050 - Parkville
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Florida
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Massachusetts
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Michigan
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Missouri
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
New York
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
North Carolina
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Ohio
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Pennsylvania
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Texas
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Washington
Query!
Country [12]
0
0
Bulgaria
Query!
State/province [12]
0
0
Sofia
Query!
Country [13]
0
0
Czechia
Query!
State/province [13]
0
0
Prague
Query!
Country [14]
0
0
France
Query!
State/province [14]
0
0
Bordeaux
Query!
Country [15]
0
0
France
Query!
State/province [15]
0
0
Nantes
Query!
Country [16]
0
0
France
Query!
State/province [16]
0
0
Paris
Query!
Country [17]
0
0
Italy
Query!
State/province [17]
0
0
Brescia
Query!
Country [18]
0
0
Italy
Query!
State/province [18]
0
0
Cuneo
Query!
Country [19]
0
0
Italy
Query!
State/province [19]
0
0
Genova
Query!
Country [20]
0
0
Italy
Query!
State/province [20]
0
0
Milan
Query!
Country [21]
0
0
Italy
Query!
State/province [21]
0
0
Modena
Query!
Country [22]
0
0
Italy
Query!
State/province [22]
0
0
Rome
Query!
Country [23]
0
0
Italy
Query!
State/province [23]
0
0
Trieste
Query!
Country [24]
0
0
Japan
Query!
State/province [24]
0
0
Hiroshima-ken
Query!
Country [25]
0
0
Japan
Query!
State/province [25]
0
0
Shizuoka
Query!
Country [26]
0
0
Japan
Query!
State/province [26]
0
0
Tokyo
Query!
Country [27]
0
0
Netherlands
Query!
State/province [27]
0
0
Rotterdam
Query!
Country [28]
0
0
Poland
Query!
State/province [28]
0
0
Warsaw
Query!
Country [29]
0
0
Spain
Query!
State/province [29]
0
0
Madrid
Query!
Country [30]
0
0
Spain
Query!
State/province [30]
0
0
Valencia
Query!
Country [31]
0
0
United Kingdom
Query!
State/province [31]
0
0
London
Query!
Country [32]
0
0
United Kingdom
Query!
State/province [32]
0
0
Southamption
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Mitsubishi Tanabe Pharma America Inc.
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
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.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT06144840
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Head of Medical Science
Query!
Address
0
0
Mitsubishi Tanabe Pharma America Inc.
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Clinical Trials Information Desk, To prevent mis-communication,
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
please email:
Query!
Fax
0
0
Query!
Email
0
0
[email protected]
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT06144840
Download to PDF