Compassionate Use of Defibrotide for Patients With Veno-o... | NCT00143546

Studies
Compassionate Use of Defibrotide for Patients With Veno-occlusive Disease of the Liver

NCT00143546

Sponsor

St. Jude Children's Research Hospital

Status

No longer available

Last Update Posted

Apr 25, 2017Actual

Enrollment

Not provided

Phase

Not provided

Conditions

Hepatic Veno-occlusive Disease

Interventions

Defibrotide

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2017

Overall Recruitment Status or Expanded Access Status

No longer available

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Not provided

Start Date

Nov 2003

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Sep 1, 2005

First Submission Date that Met QC Criteria

Sep 1, 2005

First Posted Date

Sep 2, 2005Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 24, 2017

Last Update Posted Date

Apr 25, 2017Actual

Description Module

Brief Summary

Veno-occlusive disease (VOD) of the liver is a significant complication for some patients undergoing hematopoietic stem cell transplantation. This disease is thought to be a toxicity secondary to chemotherapy or radiation-induced damage to the epithelial cells of the blood vessels in the liver. VOD is categorized as mild, moderate or severe. Historically, there has been no method to treat the disease. Recently, however, there have been investigations into the use of a new agent called defibrotide.

The primary purpose of this protocol is to provide defibrotide to patients with severe VOD. Because this drug has not been approved by the FDA, use of this medication under the auspices of this IND treatment plan is for compassionate use only.

Detailed Description

Secondary objectives of this protocol include the following:

To describe the toxicities of defibrotide in patients with VOD.
To describe the response rate of VOD in patients receiving defibrotide.

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

No data available

No data is available for this block.

Biospecimen

No data available

No data is available for this block.

Enrollment

Not provided

Arms/Interventions Module

Arm Groups

Not provided

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Defibrotide	Drug	Defibrotide to be administered approximately every 6 hours at an initial dose of 6.25 mg/kg/dose. Subsequent doses will be escalated with a plateau of 15 mg/kg/dose (or 60 mg/kg/day total). For patients with persistent VOD, the dose may be escalated by 10 mg/kg/day as needed to a maximum of 110 mg/kg/day total.		Veno-occlusive disease VOD Hepatic veno-occlusive disease.

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Patients with clinical or pathologic diagnosis of veno-occlusive disease. The following criteria will be used:

Hyperbilirubinemia (greater than or equal to 2mg/dl) and two of the following: (hepatomegaly, ascites, or weight gain [greater than or equal to 5% of baseline]); OR,
Patient who do not fulfill criteria in #1 but who have pathologic or radiographic evidence of VOD.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Wing Leung, M.D.	St. Jude Children's Research Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
St. Jude Children's Research Hospital		Memphis	Tennessee	38105	United States

Label	URL
St. Jude Children's Research Hospital	View source

Condition Browse Module

MeSH Terms

ID	Term
D006504	Hepatic Veno-Occlusive Disease

Ancestor Terms

ID	Term
D008107	Liver Diseases
D004066	Digestive System Diseases
D014652	Vascular Diseases
D002318	Cardiovascular Diseases

Browse Leaves

Not provided

Browse Branches

Not provided