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

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ClinicalTrials.gov Identifier: NCT03995784
Recruitment Status : Recruiting
First Posted : June 24, 2019
Last Update Posted : June 24, 2019
Sponsor:
Information provided by (Responsible Party):
Catalyst Biosciences

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.

Condition or disease Intervention/treatment Phase
Hemophilia B Biological: Coagulation Factor IX variant Phase 2

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Estimated Primary Completion Date : September 15, 2019
Estimated Study Completion Date : November 15, 2019


Arm Intervention/treatment
Experimental: Intravenous Loading Dose
Coagulation Factor IX variant, 50 IU/kg by intravenous route
Biological: Coagulation Factor IX variant
Single intravenous injection of CB2679d

Experimental: Subcutaneous Dosing
Coagulation Factor IX variant, 100 IU/kg by subcutaneous route
Biological: Coagulation Factor IX variant
Daily subcutaneous injections of CB2679d for 28 days




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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)

  2. 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

  3. 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

  4. 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))

  5. 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.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03995784


Contacts
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Contact: Howard Levy, MD, PhD, MMM +1.650.266.8671 hlevy@catbio.com

Locations
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South Africa
Haemophilia Comprehensive Care Centre Recruiting
Johannesburg, South Africa
Sponsors and Collaborators
Catalyst Biosciences
Investigators
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Study Director: Howard Levy, MD, PhD, MMM Catalyst Biosciences

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Responsible Party: Catalyst Biosciences
ClinicalTrials.gov Identifier: NCT03995784     History of Changes
Other Study ID Numbers: DLZ-201
First Posted: June 24, 2019    Key Record Dates
Last Update Posted: June 24, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
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.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked