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

* This page includes information on clinical trials registered in UMIN clinical trial registed system.
* We don't aim to advertise certain products or treatments


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

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 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


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 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


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name