Unique ID issued by UMIN | UMIN000013455 |
---|---|
Receipt number | 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 11:13:22 |
Efficacy and safety study of defibrotide (DF) for the prophylaxis of venoocclusive disease (VOD).
Efficacy and safety study of defibrotide (DF) for the prophylaxis of venoocclusive disease (VOD).
Efficacy and safety study of defibrotide (DF) for the prophylaxis of venoocclusive disease (VOD).
Efficacy and safety study of defibrotide (DF) for the prophylaxis of venoocclusive disease (VOD).
Japan |
Veno Occlusive Disease
Hematology and clinical oncology | Pediatrics |
Malignancy
NO
[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.
Efficacy
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.
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.
Interventional
Parallel
Randomized
Individual
Open -no one is blinded
No treatment
YES
YES
Institution is not considered as adjustment factor.
NO
Central registration
2
Treatment
Medicine |
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).
Control Group(non-administration group):Standard Treatment
Not applicable |
50 | years-old | > |
Male and Female
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.
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.
75
1st name | |
Middle name | |
Last name | Atsushi Kikuta |
Fukushima Medical University Hospital
Clinical Oncology Center/Children's Oncology Division
1 Hikariga-oka, Fukushima City 960-1295, JAPAN
024-547-1111
akikuta@fmu.ac.jp
1st name | |
Middle name | |
Last name | Miwa Izutsu |
CTD Inc.
-
3-3-2Tsukiji,Chuo-ku,Tokyo,140-0045,Japan
03-6228-4835
chosei@fmu-df.jp
Fukushima Medical University Hospital
Atsushi Kikuta
Ministry of Health, Labour and Welfare
LINK Phamaceuticals KK
NO
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)
2014 | Year | 03 | Month | 18 | Day |
Unpublished
Completed
2014 | Year | 01 | Month | 17 | Day |
2014 | Year | 05 | Month | 07 | Day |
2014 | Year | 03 | Month | 18 | Day |
2017 | Year | 02 | Month | 06 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000015712