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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-06-18

Study Completion Date

2020-04-30

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Hemophilia B

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Intravenous Dose

Coagulation Factor IX variant, 50 IU/kg by intravenous route

Group Type EXPERIMENTAL

Coagulation Factor IX variant

Intervention Type BIOLOGICAL

Single intravenous injection of CB2679d/Dalcinonacog alfa

Subcutaneous Dosing

Coagulation Factor IX variant, 100 IU/kg by subcutaneous route

Group Type EXPERIMENTAL

Coagulation Factor IX variant

Intervention Type BIOLOGICAL

Daily subcutaneous injections of CB2679d/Dalcinonacog alfa for 28 days

Interventions

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Coagulation Factor IX variant

Single intravenous injection of CB2679d/Dalcinonacog alfa

Intervention Type BIOLOGICAL

Coagulation Factor IX variant

Daily subcutaneous injections of CB2679d/Dalcinonacog alfa for 28 days

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No