UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000030522 |
|---|---|
| Receipt number | R000034316 |
| Scientific Title | A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies |
| Date of disclosure of the study information | 2017/12/22 |
| Last modified on | 2018/01/02 11:35:58 |
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Basic information
Public title
A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies
Acronym
Sirolimus for intractable vascular anomalies
Scientific Title
A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies
Scientific Title:Acronym
Sirolimus for intractable vascular anomalies
Region
| Japan |
Condition
Condition
Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lymphatic anomaly, Kaposiform lymphangiomatosis), Gorham-Stout disease, Kaposiform hemangioendothelioma and Tuffted angioma with Kasabach-Merritt phenomenon, Nenous malformation, Arteriovenous malformation, Klippel-Trenaunay-Weber syndrome,Bluerubber bleb nevus syndrome, Complex-combined vascular malformations
Classification by specialty
| Medicine in general | Pneumology | Endocrinology and Metabolism |
| Hematology and clinical oncology | Surgery in general | Vascular surgery |
| Chest surgery | Pediatrics | Dermatology |
| Oto-rhino-laryngology | Orthopedics | Oral surgery |
| Neurosurgery | Plastic surgery | Aesthetic surgery |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To assess efficacy and safety of mTOR inhibitor sirolimus in patients with intractable vascular anomalies.
Basic objectives2
Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Adverse effects and side effects
Key secondary outcomes
Target lesion response rate determined by Independent Review Facility after 24 and 52 weeks of treatments
Respiratory function after 24 and 52 weeks of treatments
Evaluation of pleural effusion after 24 and 52 weeks of treatments
Evaluation of ascites after 24 and 52 weeks of treatments
Blood coagulation parameters after 5, 12, 24 and 52 weeks of treatments
Bleeding after 24 and 52 weeks of treatments
Pain after 24 and 52 weeks of treatments
QOL improvement rates after 24 and 52 weeks of treatments
ADL improvement rates after 24 and 52 weeks of treatments
Laboratory values
Vital signs
Pharmacokinetics
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Body surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
Interventions/Control_2
Interventions/Control_3
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
| Not applicable |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1) Patients with intractable vascular anomaly diagnosed by the investigator/subinvestigator
2) Patients must have vascular anomalies that have potential to cause significant morbidity.
3) Normal liver, renal, and cardiac function at entry
Total bilirubin < 3 x ULN for age
CRE < 3 x ULN for age
4) Written consent to participate in this clinical trial has been given by the subject in person or by a legal guardian (when the subject is younger than 20 years at consent).
Key exclusion criteria
1)Patients who currently have an uncontrolled infection
2) Uncontrolled diabetes, uncontrolled hypertension, uncontrolled hyperlipidemia, chronic liver disease, or chronic renal disease
3) History of allergy to sirolimus, or additive substance
4) Known history of HIV seropositivity or known immunodeficiency
5) Patients who have undergone surgical resection or interventional radiology procedures for target lesions within 2 weeks
6) Pregnant, probably pregnant, or breast-feeding woman.
Patients who do not agree birth control during clinical trial.
7) Patient who is judged inappropriate to participate in this study by the investigators
Target sample size
50
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Michio Ozeki |
Organization
Gifu University Hospital
Division name
Pediatrics
Zip code
Address
1-1 Yanagido, Gifu City 501-1194, Japan
TEL
058-230-6000
michioo@gifu-u.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Ryuta Asada |
Organization
Gifu University Hospital
Division name
Innovative and Clinical Research Promotion Center
Zip code
Address
1-1 Yanagido, Gifu City 501-1194, Japan
TEL
058-230-6000
Homepage URL
rasada@gifu-u.ac.jp
Sponsor or person
Institute
Gifu University
Institute
Department
Personal name
Funding Source
Organization
AMED
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
Address
Tel
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
慶應義塾大学病院（東京都）
国立成育医療研究センター（東京都）
岐阜大学医学部附属病院（岐阜県）
京都府立医科大学附属病院（京都府）
九州大学病院（福岡県）
Other administrative information
Date of disclosure of the study information
| 2017 | Year | 12 | 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
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Open public recruiting
Date of protocol fixation
| 2017 | Year | 11 | Month | 02 | Day |
Date of IRB
Anticipated trial start date
| 2017 | Year | 11 | Month | 14 | Day |
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2017 | Year | 12 | Month | 22 | Day |
Last modified on
| 2018 | Year | 01 | Month | 02 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034316
