Hemophilia BPhase 1/2NCT01687608
Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
A Phase 1/2 study testing AskBio009 in people with Hemophilia B, run by Baxalta now part of Shire at 15 sites in United States.
Trial summary
The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B. Read more about this study
- Who is it for?
- Ages 18 to 75 years, men only
- Conditions the study is for
- Hemophilia B
- What is being tested?
- AskBio009
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 15 sitesUnited States
- Study dates
- 11 Feb 2013to 17 Jan 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 to 75 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males age 18-75 years, inclusive
- Established hemophilia B with ≥3 hemorrhages per year requiring treatment with exogenous FIX OR use of FIX prophylaxis because of history of frequent bleeding episodes
- Plasma FIX activity ≤2% (<1% for first cohort; then per protocol)
- Negative for active Hepatitis C virus (HCV), defined as Hepatitis C virus antibody negative and negative (undetectable) PCR test for plasma Hepatitis C virus ribonucleic acid (RNA) OR if Hepatitis C virus antibody positive must have ≥2 consecutive negative (undetectable) PCR tests for plasma HCV RNA at least 3 months apart, and negative at screening
Who cannot join
- Family history of inhibitor to FIX protein or personal laboratory evidence of having developed inhibitors to FIX protein at any time (>0.6 Bethesda Units on any single test)
- Documented prior allergic reaction to any FIX product
- Detectable AAV8 neutralizing antibodies
- Markers of hepatic inflammation or overt or occult cirrhosis as evidenced by one or more of the following:
- Platelet count <175,000/μL
- Albumin ≤3.5 g/dL
Show all 11Show fewer
- Total bilirubin >1.5 x ULN and direct bilirubin ≥0.5 mg/dL
- Alkaline phosphatase >2.0 x ULN
- ALT or AST >2.0 x ULN (except for subjects who are HIV infected)
- Liver biopsy in the past indicating moderate or severe fibrosis (Metavir staging of 2 or greater)
- History of ascites, varices, variceal hemorrhage or hepatic encephalopathy
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
Emory University
Atlanta, Georgia, 30322
U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center
Aurora, Colorado, 80045
Children's Hospital of Boston
Boston, Massachusetts, 022105
Medical University of South Carolina
Charleston, South Carolina, 29425
Rush University Medical Center
Chicago, Illinois, 60612
Gulf States Hemophilia and Thrombosis Center
Houston, Texas, 77030
Orthopaedic Hemophilia Treatment Center
Los Angeles, California, 90007
Children's Hospital Los Angeles
Los Angeles, California, 90027
BloodCenter of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute
Minneapolis, Minnesota, 55455
Mount Sinai Medical Center
New York, New York, 10029
The Hemophilia Center, Oregon Health and Science University
Portland, Oregon, 97239
University of California Davis Medical Center
Sacramento, California, 95817
University of California at San Diego Medical Center
San Diego, California, 92103-8651
Bloodworks Northwest
Seattle, Washington, 98104
About this study
Official title: A Phase 1/2 Open-Label, Single Ascending Dose Trial of a Self-Complementing Optimized Adeno-associated Virus Serotype 8 Factor IX Gene Therapy (AskBio009) in Adults With Hemophilia B
The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B.
Read the detailed description
Hemophilia B is a genetic X-linked bleeding disorder caused by a deficiency in blood-clotting Factor IX (FIX) activity. FIX is synthesized in the liver and circulates in the blood as a proenzyme. Current treatment for hemophilia B is based on replacement of the deficient FIX with IV injections of recombinant FIX protein prophylactically or as needed to treat bleeding episodes. This clinical program will test a gene transfer approach involving the use of a gene delivery vector carrying a FIX gene. This first-in-humans study is intended to evaluate the safety, kinetics, and if possible, the dose of AskBio009 required to achieve stable plasma FIX activity between 10% and 40% of normal activity.
- Conditions
- Hemophilia B
- Being tested
- AskBio009
- Keywords
- Hemophilia B, factor IX deficiency, gene therapy
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AskBio009Biological
Also called BAX 335
Single dose IV injection
| Study group | Role | What they receive |
|---|---|---|
| AskBio009 Dose Escalation | Experimental | Single Dose of a Self-Complementing Optimized Adeno-associated Virus (AAV) Serotype 8 Factor IX Gene Therapy Receives: Biological: AskBio009 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients experiencing treatment-related adverse events by dose group
Measured: Infusion to Week 3 and Infusion to end of study
Change from baseline in clinical laboratory evaluations
Measured: Change from baseline at week 3 and change from baseline at the end of study
Secondary outcomes (3)
Changes from Baseline in FIX activity levels, FIX protein levels, and Bleeding Episode Severity & Frequency
Measured: At multiple timepoints from pre-dose through up to 5 years post-dose
Immune Response to AskBio009
Measured: At multiple timepoints from pre-dose through up to 5 years post-dose
Detection of AskBio009 genomes in blood, saliva, urine, stool, and semen
Measured: At multiple timepoints from pre-dose through up to 1 years post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2012 (estimated)
- Study start11 Feb 2013
- Primary completion17 Jan 2030 (estimated)
- Study completion17 Jan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Shire
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01687608
- Sponsor's protocol ID
- AskBio009-101
- Other IDs
- 231401
- Status last verified
- Mar 2026
- First submitted
- 27 Aug 2012
- Last updated
- 20 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Publications
Konkle BA, Walsh CE, Escobar MA, Josephson NC, Young G, von Drygalski A, McPhee SWJ, Samulski RJ, Bilic I, de la Rosa M, Reipert BM, Rottensteiner H, Scheiflinger F, Chapin JC, Ewenstein B, Monahan PE. BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression. Blood. 2021 Feb 11;137(6):763-774. doi: 10.1182/blood.2019004625.
PubMed 33067633Weber A, Engelmaier A, Voelkel D, Pachlinger R, Scheiflinger F, Monahan PE, Rottensteiner H. Development of Methods for the Selective Measurement of the Single Amino Acid Exchange Variant Coagulation Factor IX Padua. Mol Ther Methods Clin Dev. 2018 Jun 28;10:29-37. doi: 10.1016/j.omtm.2018.05.004. eCollection 2018 Sep 21.
PubMed 30003118
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Open-Label, Single Ascending Dose Trial of a Self-Complementing Optimized Adeno-associated Virus Serotype 8 Factor IX Gene Therapy (AskBio009) in Adults With Hemophilia B
- ClinicalTrials.gov ID
- NCT01687608
- Acronym / sponsor ID
- AskBio009-101
- Conditions
- Hemophilia B
- Keywords
- Hemophilia B, factor IX deficiency, gene therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 11 Feb 2013 (actual)
- Primary completion
- 17 Jan 2030 (estimated)
- Study completion
- 17 Jan 2030 (estimated)
- First posted
- 19 Sep 2012 (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.
The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B.
Detailed description
Hemophilia B is a genetic X-linked bleeding disorder caused by a deficiency in blood-clotting Factor IX (FIX) activity. FIX is synthesized in the liver and circulates in the blood as a proenzyme. Current treatment for hemophilia B is based on replacement of the deficient FIX with IV injections of recombinant FIX protein prophylactically or as needed to treat bleeding episodes. This clinical program will test a gene transfer approach involving the use of a gene delivery vector carrying a FIX gene. This first-in-humans study is intended to evaluate the safety, kinetics, and if possible, the dose of AskBio009 required to achieve stable plasma FIX activity between 10% and 40% of normal activity.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AskBio009 Dose Escalation | Experimental | Biological: AskBio009Single Dose of a Self-Complementing Optimized Adeno-associated Virus (AAV) Serotype 8 Factor IX Gene Therapy |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Males age 18-75 years, inclusive
- Established hemophilia B with ≥3 hemorrhages per year requiring treatment with exogenous FIX OR use of FIX prophylaxis because of history of frequent bleeding episodes
- Plasma FIX activity ≤2% (<1% for first cohort; then per protocol)
- Negative for active Hepatitis C virus (HCV), defined as Hepatitis C virus antibody negative and negative (undetectable) PCR test for plasma Hepatitis C virus ribonucleic acid (RNA) OR if Hepatitis C virus antibody positive must have ≥2 consecutive negative (undetectable) PCR tests for plasma HCV RNA at least 3 months apart, and negative at screening
Key exclusion criteria (11 listed)
- Family history of inhibitor to FIX protein or personal laboratory evidence of having developed inhibitors to FIX protein at any time (>0.6 Bethesda Units on any single test)
- Documented prior allergic reaction to any FIX product
- Detectable AAV8 neutralizing antibodies
- Markers of hepatic inflammation or overt or occult cirrhosis as evidenced by one or more of the following:
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 |
|---|---|---|
| AskBio009Also: BAX 335Single dose IV injection | Biological | AskBio009 Dose Escalation |
Primary outcome
Number of patients experiencing treatment-related adverse events by dose group
Time frame: Infusion to Week 3 and Infusion to end of study
Change from baseline in clinical laboratory evaluations
Time frame: Change from baseline at week 3 and change from baseline at the end of study
3 secondary outcomes
Changes from Baseline in FIX activity levels, FIX protein levels, and Bleeding Episode Severity & Frequency
Time frame: At multiple timepoints from pre-dose through up to 5 years post-dose
Immune Response to AskBio009
Time frame: At multiple timepoints from pre-dose through up to 5 years post-dose
Detection of AskBio009 genomes in blood, saliva, urine, stool, and semen
Time frame: At multiple timepoints from pre-dose through up to 1 years post-dose
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2026.
- Derived from this study: PubMed 33067633Konkle BA, Walsh CE, Escobar MA, Josephson NC, Young G, von Drygalski A, McPhee SWJ, Samulski RJ, Bilic I, de la Rosa M, Reipert BM, Rottensteiner H, Scheiflinger F, Chapin JC, Ewenstein B, Monahan PE. BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression. Blood. 2021 Feb 11;137(6):763-774. doi: 10.1182/blood.2019004625.
- Derived from this study: PubMed 30003118Weber A, Engelmaier A, Voelkel D, Pachlinger R, Scheiflinger F, Monahan PE, Rottensteiner H. Development of Methods for the Selective Measurement of the Single Amino Acid Exchange Variant Coagulation Factor IX Padua. Mol Ther Methods Clin Dev. 2018 Jun 28;10:29-37. doi: 10.1016/j.omtm.2018.05.004. eCollection 2018 Sep 21.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Hemophilia
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.
Study Director study director
Shire
15 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 AskBio009 in people with Hemophilia B, run by Baxalta now part of Shire at 15 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?
The sponsor lists 4 inclusion and 11 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: under way, not looking for participants, as of the sponsor's last update on 20 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 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?
This study is still under way. Results are not available in the source record yet.
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.
What does the primary outcome measure?
Number of patients experiencing treatment-related adverse events by dose group (time frame: Infusion to Week 3 and Infusion to end of study). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01687608, which also lists 2 linked publications.
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 20 Mar 2026.