Status:
TERMINATED
A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
Lead Sponsor:
Nobelpharma
Conditions:
Neurofibromatosis
Eligibility:
All Genders
3+ years
Phase:
PHASE3
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 ent...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
July 8 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 7 2021
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT04461886
Start Date
July 8 2020
End Date
December 7 2021
Last Update
May 24 2022
Active Locations (4)
Enter a location and click search to find clinical trials sorted by distance.
1
Osaka University Hospital
Suita-shi, Osaka, Japan, 565-0871
2
Jikei University Hospital
Minato-ku, Tokyo, Japan
3
Fukuoka University Hospital
Fukuoka, Japan
4
Tottori University Hospital
Tottori, Japan