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

Catalyst Biosciences

Status and phase: Completed; Phase 2

Conditions: Hemophilia B

Treatments: Biological: Coagulation Factor IX variant

Study type: Interventional

Funder types: Industry

Identifiers

NCT03995784

DLZ-201

Details and patient eligibility

About

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.

Full 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.

Enrollment: 6 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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6 participants in 2 patient groups

Intravenous Dose

Experimental group

Description:

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

Treatment:

Biological: Coagulation Factor IX variant

Biological: Coagulation Factor IX variant

Subcutaneous Dosing

Experimental group

Description:

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

Treatment:

Biological: Coagulation Factor IX variant

Biological: Coagulation Factor IX variant