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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04461886
Recruitment Status : Active, not recruiting
First Posted : July 8, 2020
Last Update Posted : April 13, 2021
Sponsor:
Information provided by (Responsible Party):
Nobelpharma

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.

Condition or disease Intervention/treatment Phase
Neurofibromatosis Drug: NPC-12G gel Phase 3

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.

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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
Estimated Primary Completion Date : April 30, 2022
Estimated Study Completion Date : April 30, 2022


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




Primary Outcome Measures :
  1. 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
Criteria

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

Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Nobelpharma
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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: April 13, 2021
Last Verified: April 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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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