Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

JRCT ID: jRCT2041210146

Registered date:17/02/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
• Date of first enrollment: 11/05/2022
• Target sample size: 151
• Countries of recruitment: United States of America,Japan,Australia,Japan,Canada,Japan,Germany,Japan,Italy,Japan,Norway,Japan,Spain,Japan,Sweden,Japan,United Kingdom,Japan
• Study type: Interventional

Intervention(s)

MT-7117
Route: Oral
Frequency: Once daily

Outcome(s)

Primary Outcome

1.Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]). [ Time Frame: up to a maximum 30 months ]
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.
2.Number of patients with abnormal Physical examination data [ Time Frame: up to a maximum 30 months ]
Physical examination consists of assessment of abdominal, respiratory, cardiovascular, general appearance, and others.
3.Number of patients with Nevi appearance [ Time Frame: up to a maximum 30 months ]

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable
• Gender: Both

Include 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 child-bearing potential must agree to use 2 effective methods of contraception including barrier method (especially for female subjects, one method must be highly effective method).

Exclude criteria

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/1.73m^2 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).

Related Information

• Primary Sponsor: Kondou Kazuoki
• Secondary ID(s): NCT05005975,2021-001831-17