Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
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
2 other identifiers
interventional
30
1 country
15
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Feb 2013
Longer than P75 for phase_1
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 27, 2012
CompletedFirst Posted
Study publicly available on registry
September 19, 2012
CompletedStudy Start
First participant enrolled
February 11, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 17, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
January 17, 2030
March 20, 2026
March 1, 2026
16.9 years
August 27, 2012
March 18, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Number of patients experiencing treatment-related adverse events by dose group
Infusion to Week 3 and Infusion to end of study
Change from baseline in clinical laboratory evaluations
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
At multiple timepoints from pre-dose through up to 5 years post-dose
Immune Response to AskBio009
At multiple timepoints from pre-dose through up to 5 years post-dose
Detection of AskBio009 genomes in blood, saliva, urine, stool, and semen
At multiple timepoints from pre-dose through up to 1 years post-dose
Study Arms (1)
AskBio009 Dose Escalation
EXPERIMENTALSingle Dose of a Self-Complementing Optimized Adeno-associated Virus (AAV) Serotype 8 Factor IX Gene Therapy
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
Orthopaedic Hemophilia Treatment Center
Los Angeles, California, 90007, United States
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
University of California Davis Medical Center
Sacramento, California, 95817, United States
University of California at San Diego Medical Center
San Diego, California, 92103-8651, United States
U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center
Aurora, Colorado, 80045, United States
Emory University
Atlanta, Georgia, 30322, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
Children's Hospital of Boston
Boston, Massachusetts, 022105, United States
University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute
Minneapolis, Minnesota, 55455, United States
Mount Sinai Medical Center
New York, New York, 10029, United States
The Hemophilia Center, Oregon Health and Science University
Portland, Oregon, 97239, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Gulf States Hemophilia and Thrombosis Center
Houston, Texas, 77030, United States
Bloodworks Northwest
Seattle, Washington, 98104, United States
BloodCenter of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Related Publications (2)
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.
PMID: 33067633DERIVEDWeber 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.
PMID: 30003118DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
Shire
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 27, 2012
First Posted
September 19, 2012
Study Start
February 11, 2013
Primary Completion (Estimated)
January 17, 2030
Study Completion (Estimated)
January 17, 2030
Last Updated
March 20, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR
- Access Criteria
- IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
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.