Liver DiseasesNCT00263978
Defibrotide Compassionate Use for Patients With Life Threatening Veno-Occlusive Disease of the Liver
A study testing Defibrotide in people with Liver Diseases, run by Emory University at 1 site in United States.
Trial summary
Severe veno-occlusive disease (VOD) of the liver is a life threatening complication of blood and marrow transplantation. Treatment with currently available (Food and Drug Administration [FDA] approved) agents fails in most cases. Read more about this study
- Who is it for?
- Ages 1 year to 22 years
- Conditions the study is for
- Liver Diseases
- What is being tested?
- Defibrotide
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Nov 2005to Jun 2009
- 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 1 year to 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Veno-occlusive disease of the liver
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
About this study
Official title: Defibrotide Compassionate Use Protocol for Patients With Life Threatening Veno-Occlusive Disease of the Liver
Severe veno-occlusive disease (VOD) of the liver is a life threatening complication of blood and marrow transplantation. Treatment with currently available (Food and Drug Administration [FDA] approved) agents fails in most cases. Recently conducted clinical studies indicate that patients benefit from defibrotide, a non-FDA approved agent. This protocol has been developed not with a research intent, but rather to ensure that defibrotide is used by the blood and marrow transplant programs at Children's Healthcare of Atlanta and at Emory University in a safe, effective and ethical manner.
Read the detailed description
Severe veno-occlusive disease (VOD) of the liver is a life threatening complication of blood and marrow transplantation. Treatment with currently available (FDA approved) agents fails in most cases. Recently conducted clinical studies indicate that patients benefit from defibrotide, a non-FDA approved agent. This protocol has been developed not with a research intent, but rather to ensure that defibrotide is used by the blood and marrow transplant programs at Children's Healthcare of Atlanta and at Emory University in a safe, effective and ethical manner.
- Conditions
- Liver Diseases
- Being tested
- Defibrotide
- Keywords
- Venous occlusive disease, VOD
- Medical subject headings
- Liver Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DefibrotideDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2005 (estimated)
- Study startNov 2005
- Primary completionJun 2009
- Study completionJun 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Liver DiseasesStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, John Horan, Emory University
- Investigators
- John Horan, MD, Principal investigator, Children's Healthcare of Atlanta
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00263978
- Sponsor's protocol ID
- 0966-2005
- Status last verified
- Jun 2014
- First submitted
- 9 Dec 2005
- Last updated
- 1 Jul 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Defibrotide Compassionate Use Protocol for Patients With Life Threatening Veno-Occlusive Disease of the Liver
- ClinicalTrials.gov ID
- NCT00263978
- Acronym / sponsor ID
- 0966-2005
- Conditions
- Liver Diseases
- Keywords
- Venous occlusive disease, VOD
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- Nov 2005
- Primary completion
- Jun 2009 (actual)
- Study completion
- Jun 2009 (actual)
- First posted
- 12 Dec 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.
Severe veno-occlusive disease (VOD) of the liver is a life threatening complication of blood and marrow transplantation. Treatment with currently available (Food and Drug Administration [FDA] approved) agents fails in most cases. Recently conducted clinical studies indicate that patients benefit from defibrotide, a non-FDA approved agent. This protocol has been developed not with a research intent, but rather to ensure that defibrotide is used by the blood and marrow transplant programs at Children's Healthcare of Atlanta and at Emory University in a safe, effective and ethical manner.
Detailed description
Severe veno-occlusive disease (VOD) of the liver is a life threatening complication of blood and marrow transplantation. Treatment with currently available (FDA approved) agents fails in most cases. Recently conducted clinical studies indicate that patients benefit from defibrotide, a non-FDA approved agent. This protocol has been developed not with a research intent, but rather to ensure that defibrotide is used by the blood and marrow transplant programs at Children's Healthcare of Atlanta and at Emory University in a safe, effective and ethical manner.
Study design
Who may qualify
- Ages
- 1 year to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Veno-occlusive disease of the liver
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 |
|---|---|---|
| Defibrotide | 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 1 Jul 2014.
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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.
John Horan, MD principal investigator
Children's Healthcare of Atlanta
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 Liver Diseases, run by Emory University 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 1 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 1 Jul 2014. 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 1 Jul 2014.