UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000036527 |
|---|---|
| Receipt number | R000041614 |
| Scientific Title | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of NPC-12G for Skin Lesions in Patients with Neurofibromatosis type 1 |
| Date of disclosure of the study information | 2019/04/22 |
| Last modified on | 2021/10/19 10:35:15 |
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Basic information
Public title
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of NPC-12G for Skin Lesions in Patients with Neurofibromatosis type 1
Acronym
NEDOC-2 Study
Scientific Title
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of NPC-12G for Skin Lesions in Patients with Neurofibromatosis type 1
Scientific Title:Acronym
NEDOC-2 Study
Region
| Japan |
Condition
Condition
Neurofibromatosis type 1
Classification by specialty
| Dermatology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To evaluate efficacy and safety of NPC-12G at doses of 0.2% and 0.4% compared with placebo based on the changes in the volume of cutaneous tumor in patients with Neurofibromatosis type 1
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Developmental phase
Phase II,III
Assessment
Primary outcomes
Response rate on the changes from baseline in the volume of cutaneous tumor (measured with 3D camera) after 52 week treatment
Key secondary outcomes
1) Time course of changes from baseline in the volume of cutaneous tumor (measured with 3D camera)
2) Response rate on the changes from baseline in the volume of cutaneous tumor (measured with 3D camera) after 16 week, 28 week and 40 week treatment
3) Time course of changes from baseline in the area of cutaneous tumor (measured with ruler)
4) Improvement of cutaneous lesions after 28 and 52 week treatment judged by physician in charge
Base
Study type
Interventional
Study design
Basic design
Parallel
Randomization
Randomized
Randomization unit
Individual
Blinding
Double blind -all involved are blinded
Control
Placebo
Stratification
NO
Dynamic allocation
NO
Institution consideration
Institution is not considered as adjustment factor.
Blocking
NO
Concealment
No need to know
Intervention
No. of arms
3
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
0.2% NPC-12G gel topically administered twice a day for 52 weeks
Interventions/Control_2
0.4% NPC-12G gel topically administered twice a day for 52 weeks
Interventions/Control_3
Placebo gel topically administered twice a day for 52 weeks
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10
Eligibility
Age-lower limit
| 3 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1) Patients diagnosed as neurofibromatosis type 1 according to the clinical diagnostic criteria in guideline of Japanese Dermatological Association
2) Patients with 10 skin lesions (at least 5 lesions) that can be selected from those of the maximum size. The skin lesions can be taken pictures by 3D camera and must satisfy all of the following conditions.
a)Tumors of 3 mm or longer in longest diameter measured by ruler.
b)Tumors located solitary
c)Skin lesions without hair
d)Tumors of mountain-like shape with no shadows when taken pictures from above (bulb shape or gourd shape tumor should not be selected)
e)Tumors not located on the skin with large expansion and contraction such as inside of the elbow or the scruff.
3) Patients who is 3 years old or elder
4) Patients who provide written informed consent by themselves or legally acceptable representatives
Key exclusion criteria
1) Patients who treated with mTOR inhibitors (sirolimus, everolimus or temsirolimus), within 12 months prior to enrollment.
2) Patients who treated with RAS-MAPK inhibitors (sorafenib, regorafenib or lenvatinib), within 12 months prior to enrollment.
3) Patients who have active infectious lesions.
4) Patients who have abnormal findings (pneumonic lesions) by chest X-ray inspection.
5) Patients with creatinine clearance less than 50ml/min.
6) Patients with uncontrolled dyslipidemia (serum triglyceride is 500mg / dL or more, or LDL cholesterol is 190mg / dL or more even treated)
7) Patients who have severe complications such as cardiac disease, liver disease, pulmonary disease, hematological disorder or malignant tumor .
8) Patients who have previously experienced alcoholic sensitivity or allergy to sirolimus.
9) Patients who are pregnant or lactating.
10) Patients who cannot agree to use effective contraceptive methods during the study period and until 8 weeks after treatment.
11) Patients who have entered another clinical trial within 6 months prior to enrollment.
Target sample size
63
Research contact person
Name of lead principal investigator
| 1st name | Mari |
| Middle name | |
| Last name | Kaneda |
Organization
Osaka University Hospital
Division name
Department of Dermatology
Zip code
565-0871
Address
2-15 Yamadaoka, Suita, Osaka, Japan
TEL
06-6879-3031
mkaneda@derma.med.osaka-u.ac.jp
Public contact
Name of contact person
| 1st name | Mari |
| Middle name | |
| Last name | Kaneda |
Organization
Osaka University Hospital
Division name
Department of Dermatology
Zip code
565-0871
Address
2-15 Yamadaoka, Suita, Osaka, Japan
TEL
06-6879-3031
Homepage URL
mkaneda@derma.med.osaka-u.ac.jp
Sponsor or person
Institute
Osaka University Hospital
Institute
Department
Personal name
Funding Source
Organization
Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization
Japanese Governmental office
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Institutional Review Board of Osaka University hospital
Address
2-2 Yamadaoka, Suita, Osaka, Japan
Tel
06-6210-8290
shiken@hp-crc.med.osaka-u.ac.jp
Secondary IDs
Secondary IDs
NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW
Institutions
Institutions
大阪大学医学部附属病院（大阪府）、東京慈恵会医科大学附属病院（東京都）、
鳥取大学医学部附属病院（鳥取県）、福岡大学病院（福岡県）
Other administrative information
Date of disclosure of the study information
| 2019 | Year | 04 | Month | 22 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
76
Results
0.2% NPC-12G, 0.4% NPC12-G and placebo were topically administered to the skin lesion of the patients with neurofibromatosis type 1 for 52 weeks. There were no statistical differences between the NPC12-G group and the placebo group regarding the efficacy evaluation.
There were no serious side effects observed and there were no special concerns regarding the safety of 0.2% NPC-12G and 0.4% NPC12-G when topically administered to the skin lesion of the patients with neurofibromatosis type 1 for 52 weeks.
Results date posted
| 2021 | Year | 07 | Month | 05 | Day |
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Male: 22 cases, Female: 54 cases
Mean age: 48.3,
Minimum age: 19, Maximum age: 78
Participant flow
Informed consent obtained: 84 cases
Enrolled: 76 cases
0.2% group: 25 cases, 0.4% group: 24 cases, Placebo group: 27 cases
Adverse events
Adverse events observed were at 92.0% (23/25) of the cases in the 0.2% group, 87.5% (21/24) in the 0.4% group and 70.4% (19/27) in the placebo group. Side effects observed were at 52.0% (13/25) of the cases in the 0.2% group, 45.8% (11/24) in the 0.4% group and 37.0% (10/27) in the placebo group. No serious side effects were observed.
Outcome measures
Primary outcome measures showed there were no responders in any of the groups with the evaluation of the response rate after 52 weeks administration and there were no statistical differences observed between the NPC12-G group and the placebo group.
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Completed
Date of protocol fixation
| 2019 | Year | 03 | Month | 22 | Day |
Date of IRB
| 2019 | Year | 03 | Month | 22 | Day |
Anticipated trial start date
| 2019 | Year | 05 | Month | 10 | Day |
Last follow-up date
| 2021 | Year | 02 | Month | 28 | Day |
Date of closure to data entry
| 2021 | Year | 03 | Month | 31 | Day |
Date trial data considered complete
| 2021 | Year | 03 | Month | 31 | Day |
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2019 | Year | 04 | Month | 16 | Day |
Last modified on
| 2021 | Year | 10 | Month | 19 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000041614
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