Efficacy and safety study of defibrotide (DF) for the treatment of venoocclusive disease (VOD).

Efficacy and safety study of defibrotide (DF) for the treatment of venoocclusive disease (VOD). - Efficacy and safety study of defibrotide (DF) for the treatment of venoocclusive disease (VOD).

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000013454

Enrollment

Registered

2014-03-18

Start date

2014-05-07

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Veno Occlusive Disease

Interventions

Defibrotide(DF-01) 1)Intended dose regimen:Intravenous infusion of DF 6.25 mg/kg/dose over 2 hours, 4 doses per day (every 6 hours). 2)Planned duration of intervention:At least 21 days (for a maximu

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1.VOD is defined as those patients with jaundice (T-Bil>=2mg/dL) and who meet at least 2 of the following criteria by day 35 post stem cell transplant. 1)Hepatomegaly 2)Ascites 3)Unexplained weight gain of>5% from baseline. 2. Witten informed consent to participate in the study from the subject or legally acceptable representative.

Exclusion criteria

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.Patient who will not consent contraception 8.Judged as inappropriate for participating in the study by the principal or other investigator for other reasons.

Design outcomes

Primary

Measure	Time frame
Survival at day 100 post stem cell transplant in patients with VOD after stem cell transplant.	—

Secondary

Measure	Time frame
1)Efficacy-related outcomes -Survival at day 100, 180 post stem cell transplant and survival time in patients with severe VOD. -Survival at day 100, 180 post stem cell transplant, survival time in patients with VOD at any severity. -Remission of VOD by day 100 after stem cell transplant in patients with severe VOD or VOD of any severity. -Survival at day 100 post stem cell transplant, remission of VOD, and survival time in patients with VOD who started DF within 2 days or on or after 3 days from the onset of VOD. 2)Safety-related outcomes -Bleeding event -Profile of adverse events -Severe adverse events -Discontinuation due to toxicity 3)Phamacokinetic parameters of DF 4)Date of engraftment. 5)Remission status of the original disease before administration and 21 days after starting DF, at day 100 and 180 after stem cell transplant in patients with malignancy.	—

Measure

Time frame

1)Efficacy-related outcomes -Survival at day 100, 180 post stem cell transplant and survival time in patients with severe VOD. -Survival at day 100, 180 post stem cell transplant, survival time in patients with VOD at any severity. -Remission of VOD by day 100 after stem cell transplant in patients with severe VOD or VOD of any severity. -Survival at day 100 post stem cell transplant, remission of VOD, and survival time in patients with VOD who started DF within 2 days or on or after 3 days from the onset of VOD. 2)Safety-related outcomes -Bleeding event -Profile of adverse events -Severe adverse events -Discontinuation due to toxicity 3)Phamacokinetic parameters of DF 4)Date of engraftment. 5)Remission status of the original disease before administration and 21 days after starting DF, at day 100 and 180 after stem cell transplant in patients with malignancy.

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Countries

Japan

Contacts

Public ContactMiwa Izutsu

CTD Inc. -

chosei@fmu-df.jp03-6228-4835

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026