Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B
|ClinicalTrials.gov Identifier: NCT03995784|
Recruitment Status : Completed
First Posted : June 24, 2019
Last Update Posted : July 7, 2020
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia B||Biological: Coagulation Factor IX variant||Phase 2|
Single-center, open-label Phase 2b study will evaluate the PK, PD, efficacy and safety parameters of SC prophylaxis treatment regimens with CB2679d in adult subjects with hemophilia B. The study will enroll and dose subcutaneously, a total of 6 adult male subjects with severe congenital hemophilia B.
During the Treatment Period, the subject will receive an IV dose of 50 IU/kg followed 35 ± 5 minutes later by a SC dose of 100 IU/kg. Daily SC doses of 100 IU/kg will be administered until Day 28 (28 total SC doses). On Day 1, PK, PD, and safety assessments will be done at pre-IV dose and repeated 35 (± 5) minutes later prior to the SC dose. Subsequent PK, PD and safety assessments will be performed pre-dose on days 2, 3, 7, 14, 21 and 28.
During the Washout Period, PK, PD, and safety assessments will be done on Days 29, 30, 31, 32 and 33. Daily FIX activity levels will be measured, unless FIX activity level is known to be < 5%, as measured by local laboratory.
An End of Study visit will occur 30 days (± 2 days) after the last dose of study drug.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase 2b Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of a Subcutaneous Prophylaxis Treatment Regimen of CB2679d, in Adult Subjects With Hemophilia B|
|Actual Study Start Date :||June 18, 2019|
|Actual Primary Completion Date :||February 28, 2020|
|Actual Study Completion Date :||April 30, 2020|
Experimental: Intravenous Loading Dose
Coagulation Factor IX variant, 50 IU/kg by intravenous route
Biological: Coagulation Factor IX variant
Single intravenous injection of CB2679d/Dalcinonacog alfa
Experimental: Subcutaneous Dosing
Coagulation Factor IX variant, 100 IU/kg by subcutaneous route
Biological: Coagulation Factor IX variant
Daily subcutaneous injections of CB2679d/Dalcinonacog alfa for 28 days
- Evaluate the dose required to achieve steady-state FIX levels >12% [ Time Frame: From date of first dose of CB2679d assessed up to treatment Day 28 ]The dose required to achieve steady-state levels >12% will be reported for daily SC dosing.
- Occurrence of antibody formation [ Time Frame: From date of first dose of CB2679d until date of first occurrence of clinical event, assessed up to treatment Day 28. ]Rate of occurrence of antibody formation resulting in a decreased endogenous level of coagulation Factor IX (FIX)
- Occurrence of clinical thrombotic event [ Time Frame: From date of first dose of CB2679d until date of first occurrence of clinical event, assessed up to treatment Day 28 ]Rate of occurrence of clinical thrombotic event not attributable to another cause
- Occurrence of an antibody response [ Time Frame: From date of first dose of CB2679d until date of first occurrence of clinical event, assessed up to treatment Day 28 ]Rate of occurrence of an antibody response to CB2679d and cross-reactive with wild-type recombinant coagulation FIX
- Thrombogenicity assessment [ Time Frame: From date of first dose of CB2679d until date of first occurrence of clinical event, assessed up to treatment Day 28 ]Patients with clinically significant level of thrombogenicity markers (D-dimer, Prothrombin fragment 1+2 (F1+2), and thrombin-antithrombin complex (TAT))
- FIX activity levels [ Time Frame: From date of IV pre-dose to 35 minutes post dose. From date SC pre-dose Day 1 of dose up to treatment Day 28, and was-out period ]Patients with a change in FIX activity levels from pre-dose. The frequencies of these events will be summarized as proportions and counts.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03995784
|Haemophilia Comprehensive Care Centre|
|Johannesburg, South Africa|
|Study Director:||Howard Levy, MD, PhD, MMM||Catalyst Biosciences|