A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
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ClinicalTrials.gov Identifier: NCT04461886 |
Recruitment Status :
Terminated
(Sponsor decision)
First Posted : July 8, 2020
Last Update Posted : May 24, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Neurofibromatosis | Drug: NPC-12G gel | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 100 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I |
Actual Study Start Date : | July 8, 2020 |
Actual Primary Completion Date : | December 7, 2021 |
Actual Study Completion Date : | December 7, 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: NPC-12G gel
NPC-12G gel is containing 0.2% Sirolimus
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Drug: NPC-12G gel
NPC-12G gel is containing 0.2% Sirolimus |
- Discontinuation rate associated with adverse events (Kaplan-Meier method) [ Time Frame: 52 weeks ]Number of Adverse events leading to discontinuation

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Ages Eligible for Study: | 3 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04461886
Japan | |
Osaka University Hospital | |
Suita-shi, Osaka, Japan, 565-0871 | |
Jikei University Hospital | |
Minato-ku, Tokyo, Japan | |
Fukuoka University Hospital | |
Fukuoka, Japan | |
Tottori University Hospital | |
Tottori, Japan |
Responsible Party: | Nobelpharma |
ClinicalTrials.gov Identifier: | NCT04461886 |
Other Study ID Numbers: |
NPC-12G-5 |
First Posted: | July 8, 2020 Key Record Dates |
Last Update Posted: | May 24, 2022 |
Last Verified: | May 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Neurofibromatoses Neurofibromatosis 1 Neurofibroma Nerve Sheath Neoplasms Neoplasms, Nerve Tissue Neoplasms by Histologic Type Neoplasms Neoplastic Syndromes, Hereditary Neurocutaneous Syndromes |
Nervous System Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Peripheral Nervous System Diseases Neuromuscular Diseases Peripheral Nervous System Neoplasms Nervous System Neoplasms |