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
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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Discontinuation rate associated with adverse events (Kaplan-Meier method)
Condition
Neurofibromatosis
Intervention
NPC-12G gel
Study Arms / Comparison Groups
NPC-12G gel
Description: NPC-12G gel is containing 0.2% Sirolimus
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
100
Start Date
July 8, 2020
Completion Date
April 30, 2022
Primary Completion Date
April 30, 2022
Eligibility Criteria
Inclusion 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
investigational 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 investigational drug is judged to be
reasonable by the principal investigator or sub-investigator.
Exclusion Criteria:
1. 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)
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 trial.
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 investigational drug
within 6 months prior to the date of the consent
10. Patients who are participating in an observational study during this trial
11. Patients who are considered by the investigator as unsuitable for participation in
this trial
Gender
All
Ages
3 Years - N/A
Accepts Healthy Volunteers
No
Contacts
, ,
Location Countries
Japan
Location Countries
Japan
Administrative Informations
NCT ID
NCT04461886
Organization ID
NPC-12G-5
Responsible Party
Sponsor
Study Sponsor
Nobelpharma
Study Sponsor
, ,
Verification Date
April 2021