A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I

Study Overview
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Brief Summary
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The purpose of this tria is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel to patients with neurofibromatosis type I.

Detailed Description
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A Phase 3, open-label, uncontrolled, multicenter study to evaluate the safety and efficacy of long-term treatment with NPC-12G gel for patients with neurofibromatosis type I. Patients who meet all entry criteria for this trial apply NPC-12G gel twice a day for 52 weeks or more. Approximately 100 eligible patients will be enrolled.

Official Title
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A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I

Conditions
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Neurofibromatosis

Intervention / Treatment
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* Drug: NPC-12G gel


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients diagnosed as neurofibromatosis type 1 based on the clinical diagnostic criteria in guideline of Japanese Dermatological Association 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 investigational drug At the time of enrollment, patients who are able to choose ten evaluable target lesions for efficacy (at least 5).. Patients who have evaluable skin neurofibromas at baseline. Males and females who are 3 years old or elder at the time of informed consent. Patients who (or whose guardian) give a written informed consent in understanding and willingness after having received enough explanation regarding the study participation. Patients whose use or continued use of the investigational drug is judged to be reasonable by the principal investigator or sub-investigator. Exclusion Criteria: Patients who occurred serious adverse effects, who discontinued the sudy due to adverse effects, or who discontinued the trial due to withdrew of their consent in the Phase II/III investigator-initiated study of neurofibromatosis type I (OSD-001-004) Patients with abnormal findings (e.g., pneumonia) in the results of chest x-ray at the time of screening Patients with creatinine clearance of less than 50 mL/min Patients with poorly controlled dyslipidemia (serum triglycerides >500 mg/dL or LDL cholesterol >190 mg/dL even with treatment) 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 trial. Patients with alcohol sensitivity or allergy to an ingredient of the study drug (Sirolimus) Female patients who are pregnant, may be pregnant, or are lactating 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 Patients who participated in any other clinical trial or clinical study, other than the OSD-001-004 study, and have taken an investigational or investigational drug within 6 months prior to the date of the consent Patients who are participating in an observational study during this trial Patients who are considered by the investigator as unsuitable for participation in this trial

Ages Eligible for Study
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Minimum Age: 3 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NPC-12G gel<br>NPC-12G gel is containing 0.2% Sirolimus | Drug: NPC-12G gel<br>* NPC-12G gel is containing 0.2% Sirolimus<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Discontinuation rate associated with adverse events (Kaplan-Meier method) | Number of Adverse events leading to discontinuation | 52 weeks |