A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
Primary Purpose
Neurofibromatosis
Status
Terminated
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
NPC-12G gel
Sponsored by
About this trial
This is an interventional treatment trial for Neurofibromatosis
Eligibility Criteria
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
Sites / Locations
- Osaka University Hospital
- Jikei University Hospital
- Fukuoka University Hospital
- Tottori University Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
NPC-12G gel
Arm Description
NPC-12G gel is containing 0.2% Sirolimus
Outcomes
Primary Outcome Measures
Discontinuation rate associated with adverse events (Kaplan-Meier method)
Number of Adverse events leading to discontinuation
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04461886
Brief Title
A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
Official Title
A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
Study Type
Interventional
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
Terminated
Why Stopped
Sponsor decision
Study Start Date
July 8, 2020 (Actual)
Primary Completion Date
December 7, 2021 (Actual)
Study Completion Date
December 7, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Nobelpharma
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neurofibromatosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
100 (Actual)
8. Arms, Groups, and Interventions
Arm Title
NPC-12G gel
Arm Type
Experimental
Arm Description
NPC-12G gel is containing 0.2% Sirolimus
Intervention Type
Drug
Intervention Name(s)
NPC-12G gel
Intervention Description
NPC-12G gel is containing 0.2% Sirolimus
Primary Outcome Measure Information:
Title
Discontinuation rate associated with adverse events (Kaplan-Meier method)
Description
Number of Adverse events leading to discontinuation
Time Frame
52 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Osaka University Hospital
City
Suita-shi
State/Province
Osaka
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Jikei University Hospital
City
Minato-ku
State/Province
Tokyo
Country
Japan
Facility Name
Fukuoka University Hospital
City
Fukuoka
Country
Japan
Facility Name
Tottori University Hospital
City
Tottori
Country
Japan
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
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