UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000038973 |
|---|---|
| Receipt number | R000044448 |
| Scientific Title | A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomalies |
| Date of disclosure of the study information | 2019/12/25 |
| Last modified on | 2021/03/02 12:57:22 |
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Basic information
Public title
A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomalies
Acronym
Sirolimus for Intractable Vascular Anomalies(SIVA)
Scientific Title
A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomalies
Scientific Title:Acronym
Sirolimus for Intractable Vascular Anomalies(SIVA)
Region
| Japan |
Condition
Condition
Kaposiform hemangioendothelioma or Tufted angioma
Lymphangioma (cystic lymphatic malformation), lymphangiomatosis (generalized lymphatic anomaly) or Gorham-Stout disease
Venous malformation or blue rubber bleb nevus syndrome
Complex-combined vascular malformations or Klippel-Trenanay-Weber syndrome
Classification by specialty
| Hematology and clinical oncology | Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To assess efficacy and safety of mTOR inhibitor sirolimus granules and tablets in patients with intractable vascular anomalies
Basic objectives2
Efficacy
Basic objectives -Others
Trial characteristics_1
Confirmatory
Trial characteristics_2
Developmental phase
Phase III
Assessment
Primary outcomes
Target lesion response rate determined by Independent Review Facility after 24 weeks of treatments
Key secondary outcomes
Target lesion response rate determined by Independent Review Facility after 12 and 52 weeks of treatments
Improvement of Skin lesion after 12, 24 and 52 weeks of treatments
Evaluation of pleural effusion after 12, 24 and 52 weeks of treatments
Evaluation of ascites after 12, 24 and 52 weeks of treatments
Blood coagulation parameters after 12, 24 and 52 weeks of treatments
Bleeding after 12, 24 and 52 weeks of treatments
Pain after 12, 24 and 52 weeks of treatments
QOL improvement rates after 12, 24 and 52 weeks of treatments
ADL improvement rates after 12, 24 and 52 weeks of treatments
Adverse events and side effects
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
An initial dose of sirolimus is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL.
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
| 1 | months-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1) Corrected aged 1 month or more at consent
2) Patients definitively diagnosed with the following diseases.
Kaposiform hemangioendothelioma or Tufted angioma
Lymphangioma (cystic lymphatic malformation), lymphangiomatosis (generalized lymphatic anomaly) or Gorham-Stout disease
Venous malformation or blue rubber bleb nevus syndrome
Complex-combined vascular malformations or Klippel-Trenanay-Weber syndrome
3) Patients having one or more measurable lesions evaluated by pretreatment MR imaging
4) Patients must have vascular anomalies that have potential to cause significant morbidity.
5) Normal liver, renal, and cardiac function at entry
Total bilirubin < 3 x ULN for age
CRE < 3 x ULN for age
6) 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) Past usage of mTOR inhibitors excluding sirolimus or other molecular target drugs relating mTOR pathway within 8 weeks
2) Patients who currently have an uncontrolled infection
3) Karnofsky Performance Status (PS) <= 30 (10 years of age) or Lansky play PS <= 30 (< 10 years of age)
4) Interstitial lung disease, uncontrolled diabetes, uncontrolled hypertension, uncontrolled hyperlipidemia, chronic liver disease, or chronic renal disease
5) Chronic treatment (>= 4 weeks) with systemic steroids or another immunosuppressive agent at entry. Patients with endocrine deficiencies are allowed to receive physiologic or stress doses of steroids if necessary.
6) History of allergy to sirolimus, or additive substance
7) Patients must also avoid strong inducers of CYP3A4, and may not have received these medications within 1 week of entry.
8) Known history of HIV seropositivity or known immunodeficiency
9) Hepatitis B virus carrier and/or Hepatitis C virus carrier
10) Malabsorption of sirolimus
11) Patients who have undergone surgical resection or interventional radiology procedures for target lesions within 2 weeks
12) Patients who have received therapeutic medication for a target disease within 2 weeks
13) Patients who have received chemotherapy drugs that cause bone marrow suppression, biological drug, or off-label products within 4 weeks
14) Patients who have received radiation therapy for target lesions within 24 weeks
15) Patients who have participated another clinical trial within 4 weeks
16) Patients who have dental braces or prosthesis only if it interferes with radiologic analysis of lymphatic anomaly
17) Pregnant, probably pregnant, or breast-feeding woman.
Patients or their partners who do not agree birth control during clinical trial.
18) Patients who have participated in clinical trial of sirolimus in the past.
19) Patient who is judged inappropriate to participate in this study by the investigators
Target sample size
10
Research contact person
Name of lead principal investigator
| 1st name | Michio |
| Middle name | |
| Last name | Ozeki |
Organization
Gifu University Hospital
Division name
Pediatrics
Zip code
501-1194
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 | Michio |
| Middle name | |
| Last name | Ozeki |
Organization
Gifu University Hospital
Division name
Pediatrics
Zip code
501-1194
Address
1-1 Yanagido, Gifu City 501-1194, Japan
TEL
058-230-6000
Homepage URL
michioo@gifu-u.ac.jp
Sponsor or person
Institute
Gifu University Hospital
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
Gifu University Hospital IRB
Address
1-1 Yanagido, Gifu City 501-1194, Japan
Tel
058-230-6000
chikenj@gifu-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
Other administrative information
Date of disclosure of the study information
| 2019 | Year | 12 | Month | 25 | 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
No longer recruiting
Date of protocol fixation
| 2019 | Year | 09 | Month | 06 | Day |
Date of IRB
| 2019 | Year | 11 | Month | 14 | Day |
Anticipated trial start date
| 2020 | Year | 01 | Month | 06 | Day |
Last follow-up date
| 2022 | Year | 02 | Month | 28 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2019 | Year | 12 | Month | 24 | Day |
Last modified on
| 2021 | Year | 03 | Month | 02 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000044448
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| Registered date | File name |
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