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

Neurofibromatosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04461886
• Other study ID #: NPC-12G-5
• Status: Terminated
• Phase: Phase 3
• Start date: July 8, 2020
• Est. completion date: December 7, 2021

Study information

• Verified date: May 2022
• Source: Nobelpharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 100
• Est. completion date: December 7, 2021
• Est. primary completion date: December 7, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Neurofibroma
• Neurofibromatoses
• Neurofibromatosis
• Neurofibromatosis 1

Intervention

Drug:
• NPC-12G gel
NPC-12G gel is containing 0.2% Sirolimus

Locations

Country	Name	City	State
Japan	Fukuoka University Hospital	Fukuoka
Japan	Jikei University Hospital	Minato-ku	Tokyo
Japan	Osaka University Hospital	Suita-shi	Osaka
Japan	Tottori University Hospital	Tottori

Sponsors (1)

Lead Sponsor	Collaborator
Nobelpharma

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Discontinuation rate associated with adverse events (Kaplan-Meier method)	Number of Adverse events leading to discontinuation	52 weeks