Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01687608 |
Recruitment Status
:
Active, not recruiting
First Posted
: September 19, 2012
Last Update Posted
: November 8, 2017
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Hemophilia B | Biological: AskBio009 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 30 participants |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | January 28, 2013 |
Estimated Primary Completion Date : | October 28, 2030 |
Estimated Study Completion Date : | October 28, 2030 |

Arm | Intervention/treatment |
---|---|
Experimental: AskBio009 Dose Escalation
Single Dose of a Self-Complementing Optimized Adeno-associated Virus (AAV) Serotype 8 Factor IX Gene Therapy
|
Biological: AskBio009
Single dose IV injection
Other Name: BAX 335
|
- 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 ]
- 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 ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years to 75 Years (Adult, Senior) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
- 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01687608
United States, California | |
Orthopaedic Hemophilia Treatment Center | |
Los Angeles, California, United States, 90007 | |
Children's Hospital Los Angeles | |
Los Angeles, California, United States, 90027 | |
University of California Davis Medical Center | |
Sacramento, California, United States, 95817 | |
University of California at San Diego Medical Center | |
San Diego, California, United States, 92103-8651 | |
United States, Colorado | |
U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center | |
Aurora, Colorado, United States, 80045 | |
United States, Georgia | |
Emory University | |
Atlanta, Georgia, United States, 30322 | |
United States, Illinois | |
Rush University Medical Center | |
Chicago, Illinois, United States, 60612 | |
United States, Massachusetts | |
Children's Hospital of Boston | |
Boston, Massachusetts, United States, 022105 | |
United States, Minnesota | |
University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute | |
Minneapolis, Minnesota, United States, 55455 | |
United States, New York | |
Mount Sinai Medical Center | |
New York, New York, United States, 10029 | |
United States, Oregon | |
The Hemophilia Center, Oregon Health and Science University | |
Portland, Oregon, United States, 97239 | |
United States, South Carolina | |
Medical University of South Carolina | |
Charleston, South Carolina, United States, 29425 | |
United States, Texas | |
Gulf States Hemophilia and Thrombosis Center | |
Houston, Texas, United States, 77030 | |
United States, Washington | |
Bloodworks Northwest | |
Seattle, Washington, United States, 98104 | |
United States, Wisconsin | |
BloodCenter of Wisconsin | |
Milwaukee, Wisconsin, United States, 53226 |
Study Director: | John Chapin, MD | Baxalta now part of Shire |
Responsible Party: | Baxalta now part of Shire |
ClinicalTrials.gov Identifier: | NCT01687608 History of Changes |
Other Study ID Numbers: |
AskBio009-101 |
First Posted: | September 19, 2012 Key Record Dates |
Last Update Posted: | November 8, 2017 |
Last Verified: | November 2017 |
Studies a U.S. FDA-regulated Drug Product: | Yes | |
Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Shire ( Baxalta now part of Shire ):
Hemophilia B factor IX deficiency gene therapy |
Additional relevant MeSH terms:
Hemophilia A Hemophilia B Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked |