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NCT01687608 Clinical Trial

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

Hemophilia B Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT01687608
Other study ID # AskBio009-101
Secondary ID 231401
Status Active, not recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date February 11, 2013
Est. completion date January 17, 2030

Study information
Verified date January 2024
Source Takeda
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical 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.

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.

Recruitment information / eligibility
Status Active, not recruiting
Enrollment 30
Est. completion date January 17, 2030
Est. primary completion date January 17, 2030
Accepts healthy volunteers No
Gender Male
Age group 18 Years to 75 Years
Eligibility 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

Study Design

Related Conditions & MeSH terms

Intervention

Biological:
• AskBio009
Single dose IV injection

Locations
Country Name City State
United States Emory University Atlanta Georgia
United States U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center Aurora Colorado
United States Children's Hospital of Boston Boston Massachusetts
United States Medical University of South Carolina Charleston South Carolina
United States Rush University Medical Center Chicago Illinois
United States Gulf States Hemophilia and Thrombosis Center Houston Texas
United States Children's Hospital Los Angeles Los Angeles California
United States Orthopaedic Hemophilia Treatment Center Los Angeles California
United States BloodCenter of Wisconsin Milwaukee Wisconsin
United States University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute Minneapolis Minnesota
United States Mount Sinai Medical Center New York New York
United States The Hemophilia Center, Oregon Health and Science University Portland Oregon
United States University of California Davis Medical Center Sacramento California
United States University of California at San Diego Medical Center San Diego California
United States Bloodworks Northwest Seattle Washington

Sponsors (1)
Lead Sponsor Collaborator
Baxalta now part of Shire

Country where clinical trial is conducted

United States, 

Outcome
Type Measure Description Time frame Safety issue
Primary Number of patients experiencing treatment-related adverse events by dose group Infusion to Week 3 and Infusion to end of study
Primary Change from baseline in clinical laboratory evaluations Change from baseline at week 3 and change from baseline at the end of study
Secondary 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
Secondary Immune Response to AskBio009 At multiple timepoints from pre-dose through up to 5 years post-dose
Secondary Detection of AskBio009 genomes in blood, saliva, urine, stool, and semen At multiple timepoints from pre-dose through up to 1 years post-dose
See also
  Status Clinical Trial Phase
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Completed NCT02199717 - An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia N/A
Completed NCT01662531 - A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B Phase 3
Completed NCT01335061 - Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B Phase 3
Completed NCT01217255 - Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
Completed NCT00037557 - Study Evaluating rFIX; BeneFIX in Severe Hemophilia B Phase 3
Completed NCT02554773 - An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B Phase 1/Phase 2
Terminated NCT02807753 - The Hemophilia Ultrasound Project
Active, not recruiting NCT03901755 - An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B
Not yet recruiting NCT05980377 - Patterns of Hemophilia Care in Assiut Children Patients
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Terminated NCT03248141 - Understanding Hemophilia A and B Drug Dosage Administration Patterns
Terminated NCT01460147 - Osteoporosis and MRI Study in Hemophilia N/A
Completed NCT03818529 - ATHN 8: Previously Untreated Patients (PUPs) Matter Study
Completed NCT02571569 - A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors Phase 1
Terminated NCT01620801 - Hemophilia B Gene Therapy With AAV8 Vector Phase 1
Completed NCT01233440 - Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B Phase 1
Active, not recruiting NCT04135300 - Gene Therapy for Chinese Hemophilia B N/A
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Terminated NCT00947193 - Study of Ataluren (PTC124) in Hemophilia A and B Phase 2

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