Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0 Level: LLT Classification code 10015289 Term: Erythropoietic protoporphyria System Organ Class: 100000004850
Conditions
Sponsors
Mitsubishi Tanabe Pharma Development America (MTDA), Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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 Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (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 prior to receiving the first dose of IMP. 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 Section 8.7.1. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 138 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 9
Exclusion criteria
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 Liver function test (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; an estimated glomerular filtration rate (eGFR) <60 ml/min as calculated by the Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) 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. 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, Over the counter (OTCs), such as Non-steroidal anti-inflammatory drug (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 produc
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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 Erythropoietic Protoporphyria (EPP)or X-Linked Protoporphyria (XLP).;Secondary Objective: - To investigate the effect on patient-reported QOL adult and adolescent with EPP or XLP. - To investigate the effect on the percentage of responders based on the within-subject meaningful threshold for time to first prodromal symptom in adults and adolescents with EPP or XLP. - To investigate the effect on number and severity of sunlight-induced pain events (prodrome and phototoxic reactions) in adults and adolescents with EPP or XLP.;Primary end point(s): 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).;Timepoint(s) of evaluation of this end point: To calculate the average daily duration, a 14-day window on or before a time-point (Week 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, and 26) will be used. For baseline, a 14-day window before Day 1 will be used. A 14-day window will be applied to similar situations for other efficacy endpoints related to sunlight exposure diary. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Patient Global Impression of Change (PGIC) at Week 26. 2. 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. 3. Change from baseline for total score in the domain of pain intensity in the Patient-reported Outcomes Measurement Information (PROMIS)-57 at Week 26. 4. 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 pre-sunset defined by within-subject meaningful change of 66 minutes increase from baseline to Week 26. 5. Change from baseline for total score in the domain of physical function in the PROMIS-57 at Week 26.;Timepoint(s) of evaluation of this end point: 1.Patient Global impression of change (PGIC), change from baseline for total score in domain of pain intensity, change from baseline for total score of physical function: at week 26 2. Total number of prodrome symptoms and percentage of subjects who are responders based on average daily sunlight exposure: from baseline to week 26. | — |
Countries
Australia, Canada, Finland, Germany, Italy, Japan, Norway, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactGeneral Information
Mitsubishi Tanabe Pharma Development America (MTDA), Inc.
Outcome results
None listed