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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04402489




Registration number
NCT04402489
Ethics application status
Date submitted
20/05/2020
Date registered
26/05/2020
Date last updated
15/05/2023

Titles & IDs
Public title
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
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
Secondary ID [1] 0 0
MT-7117-G01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
EPP 0 0
XLP 0 0
Condition category
Condition code
Skin 0 0 0 0
Other skin conditions
Blood 0 0 0 0
Other blood disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Placebo
Treatment: Drugs - MT-7117 Low Dose
Treatment: Drugs - MT-7117 High Dose

Placebo comparator: Placebo Comparator - Oral tablet of placebo once a day.

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

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


Treatment: Drugs: Placebo
Placebo

Treatment: Drugs: MT-7117 Low Dose
MT-7117 Low Dose

Treatment: Drugs: MT-7117 High Dose
MT-7117 High Dose
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

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 26.
Timepoint [1] 0 0
Baseline (Week 0) and 26 weeks
Secondary outcome [1] 0 0
Patient Global Impression of Change (PGIC).
Timepoint [1] 0 0
Week 26
Secondary outcome [2] 0 0
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.
Timepoint [2] 0 0
Baseline (Week 0) and Week 26

Eligibility
Key inclusion criteria
Additional screening criteria check may apply for qualification.



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.
Minimum age
12 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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).

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/07/2022
Sample size
Target
Accrual to date
Final
184
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
The Wesley Hospital - Brisbane
Recruitment hospital [2] 0 0
Royal Melbourne Hospital (RMH) - Melbourne
Recruitment postcode(s) [1] 0 0
4066 - Brisbane
Recruitment postcode(s) [2] 0 0
3050 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
United States of America
State/province [10] 0 0
Washington
Country [11] 0 0
Canada
State/province [11] 0 0
Alberta
Country [12] 0 0
Germany
State/province [12] 0 0
Northrhein Westalien
Country [13] 0 0
Germany
State/province [13] 0 0
Berlin
Country [14] 0 0
Italy
State/province [14] 0 0
Brescia
Country [15] 0 0
Italy
State/province [15] 0 0
Milan
Country [16] 0 0
Italy
State/province [16] 0 0
Modena
Country [17] 0 0
Italy
State/province [17] 0 0
Rome
Country [18] 0 0
Japan
State/province [18] 0 0
Hyogo
Country [19] 0 0
Japan
State/province [19] 0 0
Ishikawa
Country [20] 0 0
Japan
State/province [20] 0 0
Osaka
Country [21] 0 0
Japan
State/province [21] 0 0
Tokyo
Country [22] 0 0
Japan
State/province [22] 0 0
Toyama
Country [23] 0 0
Norway
State/province [23] 0 0
Bergen
Country [24] 0 0
Spain
State/province [24] 0 0
Barcelona
Country [25] 0 0
Spain
State/province [25] 0 0
Madrid
Country [26] 0 0
Sweden
State/province [26] 0 0
Stockholm
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Manchester
Country [28] 0 0
United Kingdom
State/province [28] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Mitsubishi Tanabe Pharma America Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT04402489
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Head of Medical Science
Address 0 0
Mitsubishi Tanabe Pharma America Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04402489