Hemophilia BPhase 1/2NCT00076557
Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
A Phase 1/2 study testing Adeno-Associated Viral with Human Factor IX in people with Hemophilia B, run by Avigen at 3 sites in United States.
Trial summary
In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector). Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia B
- What is being tested?
- Adeno-Associated Viral with Human Factor IX
- How many people
- 15 participants
- Where
- 3 sitesUnited States
- Study dates
- Jan 2004to not given
- Phase
- Phase 1/2
- 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 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
- Males with severe hemophilia B with Factor IX activity level < 1% of normal.
- Life expectancy of > 1 year.
- Age > 18 years old.
- Ability to give informed consent.
- Greater than twenty exposure days of treatment with Factor IX protein.
- No history or presence of an inhibitor to Factor IX protein.
- Subjects must be able to receive Factor IX protein on a home infusion protocol.
- Subjects must have a normal protime (PT).
- Hepatitis C infected subjects will be evaluated for liver fibrosis based on liver biopsy data graded on a scale of 0-4 (Poynard et. al., 1997). Subjects who are Hepatitis C antibody and RNA positive and have not had a liver biopsy within the last 36 months will be required to have one.
- Subjects must have low AAV titer.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Stanford University
Palo Alto, California, 94305
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
The Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213
About this study
Official title: A Phase I Safety Study in Subjects With Severe Hemophilia B (Factor IX Deficiency) Using Adeno-Associated Viral Vector to Deliver the Gene for Human Factor IX Into the Liver
In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector). Gene therapy is a very new medical technique being used in a number of clinical studies for diseases such as cancer and cystic fibrosis. At this time, the U.S. Food and Drug Administration has approved no gene transfer products for commercial use. To date, 8 subjects have received AAV vector in the muscle for a hemophilia B trial by intramuscular injection, and, to date, 6 subjects have been treated with AAV vector in the current hemophilia B liver trial. Eleven cystic fibrosis subjects have received AAV vector into their nasal sinuses or lungs to date. In this study, AAV human Factor IX vector will be injected into the liver using a catheter inserted into a large blood vessel (called the proper hepatic artery or the right hepatic artery).
- Conditions
- Hemophilia B
- Being tested
- Adeno-Associated Viral with Human Factor IX
- Keywords
- Hemophilia B, Factor IX, Gene Transfer, Adeno-Associated Virus (AAV)
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Adeno-Associated Viral with Human Factor IXGenetic
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2004 (estimated)
- Study startJan 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants
Sponsor and investigators
- Lead sponsor
- Avigen
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Stanford University, Children's Hospital of Philadelphia, The Hemophilia Center of Western Pennsylvania, University of Washington, The University of Texas Health Science Center, Houston, University of Campinas, Brazil, Christian Medical College, Vellore, India, Royal Prince Alfred Hospital, Sydney, Australia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00076557
- Sponsor's protocol ID
- BB IND 9398
- Status last verified
- Jan 2004
- First submitted
- 26 Jan 2004
- Last updated
- 4 Apr 2007
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Apr 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Safety Study in Subjects With Severe Hemophilia B (Factor IX Deficiency) Using Adeno-Associated Viral Vector to Deliver the Gene for Human Factor IX Into the Liver
- ClinicalTrials.gov ID
- NCT00076557
- Acronym / sponsor ID
- BB IND 9398
- Conditions
- Hemophilia B
- Keywords
- Hemophilia B, Factor IX, Gene Transfer, Adeno-Associated Virus (AAV)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 15 participants
- Lead sponsor
- AvigenIndustry (pharma, biotech, devices)
- Collaborators
- Stanford University, Children's Hospital of Philadelphia, The Hemophilia Center of Western Pennsylvania, University of Washington, The University of Texas Health Science Center, Houston, University of Campinas, Brazil, Christian Medical College, Vellore, India, Royal Prince Alfred Hospital, Sydney, Australia
- Study start
- Jan 2004
- First posted
- 28 Jan 2004 (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.
In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector). Gene therapy is a very new medical technique being used in a number of clinical studies for diseases such as cancer and cystic fibrosis. At this time, the U.S. Food and Drug Administration has approved no gene transfer products for commercial use. To date, 8 subjects have received AAV vector in the muscle for a hemophilia B trial by intramuscular injection, and, to date, 6 subjects have been treated with AAV vector in the current hemophilia B liver trial. Eleven cystic fibrosis subjects have received AAV vector into their nasal sinuses or lungs to date. In this study, AAV human Factor IX vector will be injected into the liver using a catheter inserted into a large blood vessel (called the proper hepatic artery or the right hepatic artery).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| Adeno-Associated Viral with Human Factor IX | Genetic | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Apr 2007.
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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.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Adeno-Associated Viral with Human Factor IX in people with Hemophilia B, run by Avigen at 3 sites 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?
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: stopped early, as of the sponsor's last update on 4 Apr 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early. 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 4 Apr 2007.