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Anti-thrombotic agent

Defibrotide for Sinusoidal Obstruction Syndrome

Phase 2

Recruiting

Led By Mitchell Cairo, MD

Research Sponsored by New York Medical College

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 100 days

Awards & highlights

Study Summary

"This trial is testing higher doses of defibrotide in a single patient with liver and/or lung issues after a stem cell transplant. The patient did not respond well to standard doses of defibrot

Who is the study for?

This trial is for patients aged 1 month to 75 years who have had a stem cell transplant and are suffering from sinusoidal obstructive syndrome (SOS)/veno-occlusive disease (VOD) with kidney or lung problems. It's specifically for those not fully responding to standard defibrotide treatment or whose condition has worsened.Check my eligibility

What is being tested?

The study tests the safety of increasing doses of defibrotide in patients with SOS/VOD after a hematopoietic cell transplantation (HCT). The goal is to see if higher doses can be tolerated and more effective when standard treatments fail.See study design

What are the potential side effects?

While specific side effects aren't listed, defibrotide may cause bleeding complications, low blood pressure, diarrhea, vomiting, nausea and could potentially affect liver function. Side effects vary by individual.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 100 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~100 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and 100 days for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

To determine the incidence of grade 3 or 4 adverse events related to defibrotide

Side effects data

From 2018 Phase 4 trial • 20 Patients • NCT02876601

81%

Headache

38%

Flu-like Symptoms

19%

Chills

13%

Nausea

13%

Arthralgia

Precollapse

Dizziness

Vertigo

100%

80%

60%

40%

20%

Study treatment Arm

Defibrotide Plus LPS

Placebo Plus LPS

Defibrotide Plus Placebo

Placebo/Placebo

Trial Design

1Treatment groups

Experimental Treatment

Group I: DefibrotideExperimental Treatment1 Intervention

7.1.1 HCT recipients with SOS/VOD and renal and/or pulmonary dysfunction with either PR after 21 days of standard doses of defibrotide (25mg/kg/day) or SD, after 14 days of standard doses of defibrotide (25mg/kg/day) progressive disease after 7 days on defibrotide (25mg/kg/day) will undergo intra-patient dose escalation every 4 days until a complete response is obtained up until the highest dose level of 100mg/kg/day at which point an endpoint of CR, PR or SD will be sought (see 7.2 for definition of response) (Maximum of 4 dose levels) (7.1.2):

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Defibrotide

2018

Completed Phase 4

~2490

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

New York Medical CollegeLead Sponsor

69 Previous Clinical Trials

6,041 Total Patients Enrolled

Mitchell Cairo, MDPrincipal InvestigatorNew York Medical College

13 Previous Clinical Trials

316 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are participants currently being recruited for this research study?

"As per clinicaltrials.gov, this investigation is presently enrolling participants. The study was initially disclosed on March 20th, 2024 and its latest update occurred on March 22nd of the same year."

Answered by AI

How many individuals are partaking in this clinical trial as participants overall?

"Indeed, data from clinicaltrials.gov highlights the ongoing search for suitable participants in this medical investigation. Initially shared on March 20th, 2024 and subsequently revised by March 22nd, 2024, the trial aims to recruit a total of twenty patients from one designated site."

Answered by AI

Is the medical study open to individuals younger than 65 years old?

"Participants eligible for this research study must be between 1 month and 75 years old. Notably, there are a total of 387 trials focused on individuals under 18 and 1060 trials catering to those above the age of 65."

Answered by AI

Has Defibrotide received approval from the FDA?

"Based on our evaluation at Power, the safety rating for Defibrotide is a 2. This assessment stems from it being a Phase 2 trial wherein there exist some preliminary data regarding its safety profile but none yet on efficacy."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Defibrotide Dose-escalation for SOS Post-HSCT

2024. "Defibrotide Dose-escalation for SOS Post-HSCT". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05987124.