NeurofibromatosisPhase 3NCT04461886
A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
A Phase 3 study testing NPC-12G gel in people with Neurofibromatosis, run by Nobelpharma at 4 sites in Japan.
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. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Neurofibromatosis
- What is being tested?
- NPC-12G gel
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 4 sitesJapan
- Study dates
- 8 Jul 2020to 7 Dec 2021
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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)
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- 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
Trial locations and contacts
4 sites in 1 country.
Japan4 sites
Fukuoka University Hospital
Fukuoka
Jikei University Hospital
Minato-ku, Tokyo
Osaka University Hospital
Suita-shi, Osaka, 565-0871
Tottori University Hospital
Tottori
About this study
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.
Read the 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.
- Conditions
- Neurofibromatosis
- Being tested
- NPC-12G gel
- Medical subject headings
- Neurofibromatoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NPC-12G gelDrug
NPC-12G gel is containing 0.2% Sirolimus
| Study group | Role | What they receive |
|---|---|---|
| NPC-12G gel | Experimental | NPC-12G gel is containing 0.2% Sirolimus Receives: Drug: NPC-12G gel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Discontinuation rate associated with adverse events (Kaplan-Meier method)
Number of Adverse events leading to discontinuation
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2020
- Study start8 Jul 2020
- Primary completion7 Dec 2021
- Study completion7 Dec 2021
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeurofibromatosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nobelpharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04461886
- Sponsor's protocol ID
- NPC-12G-5
- Status last verified
- May 2022
- First submitted
- 3 Jul 2020
- Last updated
- 24 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
- ClinicalTrials.gov ID
- NCT04461886
- Acronym / sponsor ID
- NPC-12G-5
- Conditions
- Neurofibromatosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 100 participants (actual)
- Lead sponsor
- NobelpharmaIndustry (pharma, biotech, devices)
- Study start
- 8 Jul 2020 (actual)
- Primary completion
- 7 Dec 2021 (actual)
- Study completion
- 7 Dec 2021 (actual)
- First posted
- 8 Jul 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NPC-12G gel | Experimental | Drug: NPC-12G gelNPC-12G gel is containing 0.2% Sirolimus |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NPC-12G gelNPC-12G gel is containing 0.2% Sirolimus | Drug | NPC-12G gel |
Primary outcome
Discontinuation rate associated with adverse events (Kaplan-Meier method)
Time frame: 52 weeks
Number of Adverse events leading to discontinuation
Results and safety
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 May 2022.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NPC-12G gel in people with Neurofibromatosis, run by Nobelpharma at 4 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Discontinuation rate associated with adverse events (Kaplan-Meier method) (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 May 2022.