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Name:
UMIN ID:

Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000028905
Receipt No. R000033078
Scientific Title A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus in the Treatment of intractable lymphatic anomalies (SILA study)
Date of disclosure of the study information 2017/08/30
Last modified on 2017/11/07

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Basic information
Public title A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus in the Treatment of intractable lymphatic anomalies (SILA study)
Acronym Sirolimus for Intractable Lymphatic Anomalies
Scientific Title A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus in the Treatment of intractable lymphatic anomalies (SILA study)
Scientific Title:Acronym Sirolimus for Intractable Lymphatic Anomalies
Region
Japan

Condition
Condition Intractable Lymphatic Anomalies
Classification by specialty
Hematology and clinical oncology Vascular surgery Pediatrics
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 To assess efficacy and safety of mTOR inhibitor sirolimus in patients with intractable lymphatic 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 52 weeks of treatments
Key secondary outcomes Target lesion response rate determined by Independent Review Facility after 12, 24 weeks of treatments
Respiratory function 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 NO
Dynamic allocation NO
Institution consideration
Blocking NO
Concealment No need to know

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 BSA >= 0.6m2 at entry and judged by the investigator/subinvestigator to be able to take tablets
2) Patients definitively diagnosed with lymphangioma (cystic lymphatic malformation) who have craniocervical, intraperitoneal or retroperitoneal cystic lesions, lymphangiomatosis (generalized lymphatic anomaly) or Gorham-Stout disease according to the diagnostic criteria
3) Patients having one or more measurable lesions evaluated by pretreatment MR imaging
4) Patients must have lymphatic 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 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) 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 who do not agree birth control during clinical trial.
18) 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
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
Email 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
Email rasada@gifu-u.ac.jp

Sponsor
Institute Gifu University
Institute
Department

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
Email

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 08 Month 30 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 06 Month 15 Day
Date of IRB
Anticipated trial start date
2017 Year 10 Month 01 Day
Last follow-up date
2019 Year 03 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2017 Year 08 Month 30 Day
Last modified on
2017 Year 11 Month 07 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000033078

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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