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

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.

Study Overview

• Status: Terminated
• Conditions: Neurofibromatosis
• Intervention / Treatment: Drug: NPC-12G gel

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 Type: Interventional
• Enrollment (Actual): 100
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Fukuoka, Japan
    • Fukuoka University Hospital
  • Tottori, Japan
    • Tottori University Hospital
  • Osaka
    • Suita-shi, Osaka, Japan, 565-0871
      • Osaka University Hospital
  • Tokyo
    • Minato-ku, Tokyo, Japan
      • Jikei University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NPC-12G gel; NPC-12G gel is containing 0.2% Sirolimus	Drug: NPC-12G gel; NPC-12G gel is containing 0.2% Sirolimus

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Nobelpharma

Study record dates

Study Major Dates

• Study Start (Actual): July 8, 2020
• Primary Completion (Actual): December 7, 2021
• Study Completion (Actual): December 7, 2021

Study Registration Dates

• First Submitted: July 3, 2020
• First Submitted That Met QC Criteria: July 7, 2020
• First Posted (Actual): July 8, 2020

Study Record Updates

• Last Update Posted (Actual): May 24, 2022
• Last Update Submitted That Met QC Criteria: May 17, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Histologic Type; Neoplasms; Genetic Diseases, Inborn; Neuromuscular Diseases; Neurodegenerative Diseases; Neoplasms, Nerve Tissue; Peripheral Nervous System Diseases; Nervous System Neoplasms; Heredodegenerative Disorders, Nervous System; Neoplastic Syndromes, Hereditary; Nerve Sheath Neoplasms; Neurocutaneous Syndromes; Peripheral Nervous System Neoplasms; Neurofibromatoses; Neurofibromatosis 1; Neurofibroma
• Other Study ID Numbers: NPC-12G-5

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No