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Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03995784
Recruitment Status : Completed
First Posted : June 24, 2019
Last Update Posted : July 7, 2020
Sponsor:
Information provided by (Responsible Party):
Catalyst Biosciences

Tracking Information
First Submitted Date  ICMJE June 19, 2019
First Posted Date  ICMJE June 24, 2019
Last Update Posted Date July 7, 2020
Actual Study Start Date  ICMJE June 18, 2019
Actual Primary Completion Date February 28, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 20, 2019)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 20, 2019)
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B
Official Title  ICMJE 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
Brief Summary Phase 2b, single-center, open-label study designed to evaluate the pharmacokinetics, pharmacodynamics, efficacy and safety of subcutaneous (SC) prophylaxis treatment regimens with CB2679d in 6 adult, male subjects with severe congenital hemophilia B.
Detailed Description

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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophilia B
Intervention  ICMJE
  • Biological: Coagulation Factor IX variant
    Single intravenous injection of CB2679d/Dalcinonacog alfa
  • Biological: Coagulation Factor IX variant
    Daily subcutaneous injections of CB2679d/Dalcinonacog alfa for 28 days
Study Arms  ICMJE
  • Experimental: Intravenous Loading Dose
    Coagulation Factor IX variant, 50 IU/kg by intravenous route
    Intervention: Biological: Coagulation Factor IX variant
  • Experimental: Subcutaneous Dosing
    Coagulation Factor IX variant, 100 IU/kg by subcutaneous route
    Intervention: Biological: Coagulation Factor IX variant
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 20, 2019)
6
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE April 30, 2020
Actual Primary Completion Date February 28, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Confirmed diagnosis of severe (<2%) congenital hemophilia B.
  • Male, age 18 or older.
  • Agreement to use highly effective birth control throughout the study.
  • Affirmation of informed consent with signature confirmation before any trial-related activities.
  • Stated willingness to comply with all study procedures and availability for the duration of the study.

Exclusion Criteria:

  • History or a family history of FIX inhibitors.
  • Positive antibody to FIX detected by central laboratory at screening.
  • Previous participation in and subsequent treatment in a clinical trial within the previous 30 days or 3-half-lives, whichever is longer, or absence of clinical effect.
  • Have a coagulation disorder other than congenital hemophilia B.
  • Factor IX gene mutation 128G>A.
  • Significant contraindication to participation.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE South Africa
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03995784
Other Study ID Numbers  ICMJE DLZ-201
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Plan Description: This is a single-center, open label study so the investigator will have full access to all study subject data.
Responsible Party Catalyst Biosciences
Study Sponsor  ICMJE Catalyst Biosciences
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Howard Levy, MD, PhD, MMM Catalyst Biosciences
PRS Account Catalyst Biosciences
Verification Date July 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP