A long-term study of NPC-12G gel in neurofibromatosis type I

JRCT ID: jRCT2051200032

Registered date:06/07/2020

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Neurofibromatosis type 1
• Date of first enrollment: 08/07/2020
• Target sample size: 100
• Study type: Interventional
• Intervention(s): NPC-12G gel is applied to the lesion twice a day for 52 weeks or longer.

Outcome(s)

Primary Outcome

1) Discontinuation rates associated with adverse events (Kaplan-Meier method)
2) Adverse events leading to discontinuation

Secondary Outcome

Safety
1) Adverse events and side effects
2) Adverse events and side effects leading to temporary withdrawal (total or partial)
3) Side effects leading to discontinuation
4) Serious adverse events and serious side effects
5) Adverse events and side effects leading to modification of dosage
6) Evaluation of vital signs and clinical tests
7) Assessment of blood sirolimus levels

Efficacy
1) Longitudinal trend of tumor volume reduction based on 3D camera measurements
2) Patient Satisfaction
3) Other exploratory items

Key inclusion & exclusion criteria

• Age minimum: >= 3age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1)Patients diagnosed as neurofibromatosis type 1 based on the clinical diagnostic criteria in guideline of Japanese Dermatological Association
2)Patients participated in a Phase II/III investigator-initiated clinical trial for neurofibromatosis type I (OSD-001-004) who wish to continue treatment of the study drug
3)At the time of enrollment, patients who are able to choose ten evaluable target lesions for efficacy (at least 5)..
4)Patients who have evaluable skin neurofibromas at baseline.
5)Males and females who are 3 years old or elder at the time of informed consent.
6)Patients who (or whose guardian) give a written informed consent in understanding and
willingness after having received enough explanation regarding the study participation.
7)Patients whose use or continued use of the study drug is judged to be reasonable by the principal investigator or sub-investigator.

Exclude criteria

1)Patients who occurred serious adverse effects, who discontinued the sudy due to adverse effects, or who discontinued the sudy due to withdrew of their consent in the Phase II/III investigator-initiated study of neurofibromatosis type I (OSD-001-004)
2)Patients with abnormal findings (e.g., pneumonia) in the results of chest x-ray at the time of screening
3)Patients with creatinine clearance of less than 50 mL/min
4)Patients with poorly controlled dyslipidemia (serum triglycerides >500 mg/dL or LDL cholesterol >190 mg/dL even with treatment)
5)Patients who have complications such as infection, cardiac, hepatic, pulmonary, renal, or hematological diseases and malignant tumors that are considered unsuitable for participation in this study.
6)Patients with alcohol sensitivity or allergy to an ingredient of the study drug (Sirolimus)
7)Female patients who are pregnant, may be pregnant, or are lactating
8)Patients (including male patients with a fertile partner) who cannot consent to use adequate contraception from the date of consent to 12 weeks after the end of treatment
9)Patients who participated in any other clinical trial or clinical study, other than the OSD-001-004 study, and have taken an investigational or clinical study drug within 6 months prior to the date of the consent
10)Patients who are participating in an observational study during this clinical study
11)Patients who are considered by the investigator as unsuitable for participation in this clinical study

Related Information

• Primary Sponsor: Kaneda Mari