Overview

This trial is active, not recruiting.

Condition hemophilia b
Treatment askbio009
Phase phase 1/phase 2
Sponsor Baxalta US Inc.
Start date September 2012
End date November 2030
Trial size 30 participants
Trial identifier NCT01687608, AskBio009-101

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Single Dose of a Self-Complementing Optimized Adeno-associated Virus (AAV) Serotype 8 Factor IX Gene Therapy
askbio009 BAX 335
Single dose IV injection

Primary Outcomes

Measure
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

Secondary Outcomes

Measure
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

Eligibility Criteria

Male participants from 18 years up to 75 years old.

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

Additional Information

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
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.
Trial information was received from ClinicalTrials.gov and was last updated in September 2016.
Information provided to ClinicalTrials.gov by Baxalta US Inc..