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

NCT04461886 | Terminated Phase 3

• 1 other identifier: NPC-12G-5
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 4

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.

Conditions

Neurofibromatosis

Outcome Measures

Primary Outcomes (1)

• Discontinuation rate associated with adverse events (Kaplan-Meier method)

  Number of Adverse events leading to discontinuation

  52 weeks

Study Arms (1)

NPC-12G gel

EXPERIMENTAL

NPC-12G gel is containing 0.2% Sirolimus

Drug: NPC-12G gel

Interventions

NPC-12G gel DRUG

NPC-12G gel is containing 0.2% Sirolimus

NPC-12G gel

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Nobelpharma: lead

Study Sites (4)

Osaka University Hospital

Suita-shi, Osaka, 565-0871, Japan

Location

Jikei University Hospital

Minato-ku, Tokyo, Japan

Location

Fukuoka University Hospital

Fukuoka, Japan

Location

Tottori University Hospital

Tottori, Japan

Location

MeSH Terms

Conditions

Neurofibromatoses

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 3, 2020
• First Posted: July 8, 2020
• Study Start: July 8, 2020
• Primary Completion: December 7, 2021
• Study Completion: December 7, 2021
• Last Updated: May 24, 2022

Record last verified: 2022-05

Locations

JP (4)