Bleeder's disease Christmas disease
Conditions
Interventions
Subjects will receive a single infusion of CSL222 (formerly AMT-061). CSL222
(formerly AMT-061) will be administered at a dose of 2 x 10^13 gc/kg as a
one-time infusion in a peripheral vein, subject
Sponsors
CSL Behring LCC
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Male 2. Age >=18 years 3. Subjects with congenital hemophilia B with known severe or moderately severe FIX deficiency (150 previous exposure days of treatment with FIX protein 5. Have been on stable prophylaxis for at least 2 months prior to screening 6. Have demonstrated capability to independently, accurately and in a timely manner complete the diary during the lead-in phase as judged by the investigator 7. Acceptance to use a condom during sexual intercourse in the period from IMP administration until AAV5 has been cleared from semen, as evidenced by the central laboratory from negative analysis results for at least three consecutively collected semen samples (this criterion is applicable also for subjects who are surgically sterilized) 8. Able to provide informed consent following receipt of verbal and written information about the trial, *Continuous routine prophylaxis is defined as the intent of treating with an a priori defined frequency of infusions (e.g., twice weekly, once every two weeks, etc.) as documented in the medical records
Exclusion criteria
Exclusion criteria: 1. History of FIX inhibitors 2. Positive FIX inhibitor test at screening and Visit L-Final (based on local laboratory results) 3. Screening and Visit L-Final laboratory values (based on central laboratory results): a. ALT >2 times ULN b. Aspartate aminotransferase (AST) >2 times ULN c. Total bilirubin >2 times ULN (except if this is caused by Gilbert disease) d. Alkaline phosphatase (ALP) >2 times ULN e. Creatinine >2 times ULN limit 4. Positive human immunodeficiency virus (HIV) serological test at screening and Visit L-Final, not controlled with anti-viral therapy as shown by CD4+ counts =9 kPa is considered equivalent) 10. Known history of an allergic reaction or anaphylaxis to factor IX products 11. Known history of allergy to corticosteroids 12. Known uncontrolled allergic conditions or allergy/hypersensitivity to any component of the IMP excipients 13. Known medical condition that would require chronic administration of steroids 14. Previous gene therapy treatment 15. Receipt of an experimental agent within 60 days prior to screening 16. Current participation or anticipated participation within one year after IMP administration in this trial in any other interventional clinical trial involving drugs or devices.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary efficacy endpoints - ABR comparison between CSL222 (formerly AMT-061) and prophylaxis for non-inferiority between the lead-in phase and the 52 weeks following stable factor IX expression (months 6-18 post treatment) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary efficacy endpoints - Endogenous factor IX activity at 6 months after CSL222 dosing - Endogenous factor IX activity at 12 months after CSL222 dosing - Endogenous factor IX activity at 18 months after CSL222 dosing - Annualized consumption of factor IX replacement therapy during the 52 weeks following stable factor IX expression (months 6-18 post-treatment), excluding factor IX replacement for invasive procedures, compared to the lead-in phase - Annualized infusion rate of factor IX replacement therapy during the 52 weeks following stable factor IX expression (months 6-18 post-treatment), excluding factor IX replacement for invasive procedures, compared to the lead-in phase - Proportion of subjects remaining free of previous continuous routine prophylaxis during the 52 weeks following stable factor IX expression (months 6-18 post-treatment) - Comparison of the percentage of subjects with trough factor IX activity | — |
Countries
Netherlands
Outcome results
None listed