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Name:
UMIN ID:

Recruitment status Completed
Unique ID issued by UMIN UMIN000013455
Receipt No. R000015712
Scientific Title Efficacy and safety study of defibrotide (DF) for the prophylaxis of venoocclusive disease (VOD).
Date of disclosure of the study information 2014/03/18
Last modified on 2017/02/06

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Basic information
Public title Efficacy and safety study of defibrotide (DF) for the prophylaxis of venoocclusive disease (VOD).
Acronym Efficacy and safety study of defibrotide (DF) for the prophylaxis of venoocclusive disease (VOD).
Scientific Title Efficacy and safety study of defibrotide (DF) for the prophylaxis of venoocclusive disease (VOD).
Scientific Title:Acronym Efficacy and safety study of defibrotide (DF) for the prophylaxis of venoocclusive disease (VOD).
Region
Japan

Condition
Condition Veno Occlusive Disease
Classification by specialty
Hematology and clinical oncology Pediatrics
Classification by malignancy Malignancy
Genomic information NO

Objectives
Narrative objectives1 [Primary objective]
To evaluate efficacy of DF for the prophylaxis of VOD following allogeneic hematopoietic stem cell transplantation in both pediatric and adult patients.
[Secondary objectives]
To evaluate safety of DF.
To evaluate pharmacokinetics of DF in both pediatric and adult patients.
Basic objectives2 Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes Incidence of VOD until day 30 post stem cell transplant in patients who undergo prophylaxis with DF.

Principal or other investigator should evaluate the development of VOD according to the revised Seattle criteria. VOD is defined as those who meet at least 2 of the following criteria by day 35 post stem cell transplant.
-T-Bil>=2mg/dL
-Hepatomegaly
-Right hypochondriac pain
-Ascites
-Unexplained weight gain of>5% from baseline.
Key secondary outcomes To compare the following outcomes both in DF prophylaxis group and control (no prophylaxis) group.
1)Incidence of VOD at day 30, 100 post stem cell transplant.
2)Incidence of VOD according to the Baltimore criteria at day 30, 100 post stem cell transplant.
3)Severity of VOD in patients who developed VOD.
4)Incidence of total, grade II-IV, and III-IV acute GVHD at day 100 post stem cell transplant.
5)Survival at day 100, 180 post stem cell transplant.
6)Survival at day 100, 180 post stem cell transplant in patients who developed VOD.
7)Incidence and severity of adverse events and drug-related adverse event.
8)Date of engraftment.
9)Remission status of the original disease at day 30, 100, and 180 after stem cell transplant in patients with malignancy.

Base
Study type Interventional

Study design
Basic design Parallel
Randomization Randomized
Randomization unit Individual
Blinding Open -no one is blinded
Control No treatment
Stratification YES
Dynamic allocation YES
Institution consideration Institution is not considered as adjustment factor.
Blocking NO
Concealment Central registration

Intervention
No. of arms 2
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 Defibrotide(DF-01)
1)Intravenous infusion of DF 6.25 mg/kg/dose over 2 hours,4 doses per day (every 6 hours).
2)From 1 day before starting conditioning regimen until day 30 post stem cell transplant (for a maximum of 100 days after transplantation).
Interventions/Control_2 Control Group(non-administration group):Standard Treatment
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
50 years-old >
Gender Male and Female
Key inclusion criteria 1.younger than 50 years old (at informed consent).
2.Primary disease is one of the following:
1)malignant tumor not in remission
2)malignant tumor in remission.
3)osteopetrosis
4)non-malignant disease other than osteopetrosis
3.Patients with one or more following risk factors of VOD who undergo allogeneic stem cell transplantation with myeloablative conditioning regimen.
-Second myeloablative transplant
-Not in remission at transplant
-Performance status (ECOG) of 2 or more.
-Conditioning regimen including Bu/Mel or Bu/Cy.
-Liver dysfunction before stem cell transplant.
-Positive for anti-HCV antibody.
-Administration of gemutuzumab ozogamicin within 100 days before stem cell transplant.
-Osteopetrosis
4.Witten informed consent to participate in the study from the subject or legally acceptable representative before screening tests.
Key exclusion criteria 1.Using medication that increases risk of hemorrhage.
2.Acute bleeding that is not controlled.
3.Unstable hemodynamic status that require more than one vasopressor or decreased mean atrial pressure (MAP).
4.Complicated with viral fulminant hepatitis
5.Past history of organ transplant other than hematopoietic cell transplant.
6.Complicated with Grade IV GVHD
7.Females with pregnancy, breastfeeding, possible pregnancy. Male who will not consent contraception
8.Judged as inappropriate for participating in the study by the principal or other investigator for other reasons.
Target sample size 75

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Atsushi Kikuta
Organization Fukushima Medical University Hospital
Division name Clinical Oncology Center/Children's Oncology Division
Zip code
Address 1 Hikariga-oka, Fukushima City 960-1295, JAPAN
TEL 024-547-1111
Email akikuta@fmu.ac.jp

Public contact
Name of contact person
1st name
Middle name
Last name Miwa Izutsu
Organization CTD Inc.
Division name -
Zip code
Address 3-3-2Tsukiji,Chuo-ku,Tokyo,140-0045,Japan
TEL 03-6228-4835
Homepage URL
Email chosei@fmu-df.jp

Sponsor
Institute Fukushima Medical University Hospital
Atsushi Kikuta
Institute
Department

Funding Source
Organization Ministry of Health, Labour and Welfare
Organization
Division
Category of Funding Organization
Nationality of Funding Organization

Other related organizations
Co-sponsor LINK Phamaceuticals KK
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 公立大学法人 福島県立医科大学附属病院(福島県)
独立行政法人 国立がん研究センター中央病院(東京都)
国家公務員共済組合連合会 虎の門病院(東京都)
地方独立行政法人 神奈川県立病院機構 神奈川県立こども医療センター(神奈川県)
名古屋大学医学部附属病院(愛知県)
独立行政法人 国立病院機構 名古屋医療センター(愛知県)
公立大学法人 大阪市立大学医学部附属病院(大阪府)
兵庫医科大学病院(兵庫県)

Other administrative information
Date of disclosure of the study information
2014 Year 03 Month 18 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 Completed
Date of protocol fixation
2014 Year 01 Month 17 Day
Date of IRB
Anticipated trial start date
2014 Year 05 Month 07 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
2014 Year 03 Month 18 Day
Last modified on
2017 Year 02 Month 06 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000015712

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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