Hepatic Veno-occlusive DiseaseNCT00143546
Compassionate Use of Defibrotide for Patients With Veno-occlusive Disease of the Liver
A study testing Defibrotide in people with Hepatic Veno-occlusive Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hepatic Veno-occlusive Disease
- What is being tested?
- Defibrotide
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Nov 2003to not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: Compassionate Use of Defibrotide for Patients With Veno-Occlusive Disease
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.
Read the 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.
- Conditions
- Hepatic Veno-occlusive Disease
- Being tested
- Defibrotide
- Keywords
- Hepatic Veno-occlusive Disease
- Medical subject headings
- Hepatic Veno-Occlusive Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DefibrotideDrug
Also called Veno-occlusive disease, VOD, Hepatic veno-occlusive disease.
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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2005 (estimated)
- Study startNov 2003
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatic Veno-occlusive DiseaseStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Wing Leung, M.D., Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00143546
- Sponsor's protocol ID
- DETIDE
- Status last verified
- Apr 2017
- First submitted
- 1 Sep 2005
- Last updated
- 25 Apr 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use of Defibrotide for Patients With Veno-Occlusive Disease
- ClinicalTrials.gov ID
- NCT00143546
- Acronym / sponsor ID
- DETIDE
- Conditions
- Hepatic Veno-occlusive Disease
- Keywords
- Hepatic Veno-occlusive Disease
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- Nov 2003
- First posted
- 2 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DefibrotideAlso: Veno-occlusive disease, VOD, Hepatic veno-occlusive disease.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. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Apr 2017.
- St. Jude Children's Research HospitalLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wing Leung, M.D. principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Defibrotide in people with Hepatic Veno-occlusive Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 25 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Apr 2017.