Brief Title
Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B
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
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 Phase
Phase 2
Study Type
Interventional
Primary Outcome
Number of Subjects Who Achieved FIX Level ≥12%
Secondary Outcome
FIX Activity Levels (Actual and Change From Baseline) in All Subjects
Condition
Hemophilia B
Intervention
Coagulation Factor IX variant
Study Arms / Comparison Groups
Intravenous Dose
Description: Coagulation Factor IX variant, 50 IU/kg by intravenous route
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
6
Start Date
June 18, 2019
Completion Date
April 30, 2020
Primary Completion Date
February 28, 2020
Eligibility 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.
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Howard Levy, MD, PhD, MMM, ,
Location Countries
South Africa
Location Countries
South Africa
Administrative Informations
NCT ID
NCT03995784
Organization ID
DLZ-201
Responsible Party
Sponsor
Study Sponsor
Catalyst Biosciences
Study Sponsor
Howard Levy, MD, PhD, MMM, Study Director, Catalyst Biosciences
Verification Date
August 2021