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

NCT03995784 | Completed Phase 2 Results

• 1 other identifier: DLZ-201
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (1)

• Number of Subjects Who Achieved FIX Level ≥12%

  Subjects who achieved a FIX activity level ≥12% during the treatment period in the PK Population

  Days 7, 14, 21, 28, 29

Secondary Outcomes (12)

• FIX Activity Levels (Actual and Change From Baseline) in All Subjects

  Screening, Day 1 (IV Pre-dose, SC Dose), Day 2, 3, 7, 14, 21, 28, 29, 30, 31, 32, 33, and End of Study. End of Study is the average of each subject's last recorded assessment (between Days 29 to 33).

• Pharmacokinetic (PK) Analysis - AUC

  From date of first dose of CB2679d until date of first occurrence of clinical event, assessed up to treatment Day 28. PK sampling was conducted on Day 1 (IV pre-dose -5 min, SC 30 min post IV dose, hour 7 +/- 1 hour) and Day 2 (hour 24 +/- 1 hour).

• PK Analysis - Clearance

  From date of first dose of CB2679d until date of first occurrence of clinical event, assessed up to treatment Day 28. PK sampling was conducted on Day 1 (IV pre-dose -5 min, SC 30 min post IV dose, hour 7 +/- 1 hour) and Day 2 (hour 24 +/- 1 hour).

• PK Analysis - Maximum Concentration During SC Dosing

  From date of first dose of CB2679d until date of first occurrence of clinical event, assessed up to treatment Day 28. PK sampling was conducted on Day 1 (IV pre-dose -5 min, SC 30 min post IV dose, hour 7 +/- 1 hour) and Day 2 (hour 24 +/- 1 hour).

• PK Analysis - Half-Life and Residence Time

  From date of first dose of CB2679d until date of first occurrence of clinical event, assessed up to treatment Day 28. PK sampling was conducted on Day 1 (IV pre-dose -5 min, SC 30 min post IV dose, hour 7 +/- 1 hour) and Day 2 (hour 24 +/- 1 hour).

Study Arms (2)

Intravenous Dose

EXPERIMENTAL

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

Biological: Coagulation Factor IX variant

Subcutaneous Dosing

EXPERIMENTAL

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

Biological: Coagulation Factor IX variant

Interventions

Coagulation Factor IX variant BIOLOGICAL

Single intravenous injection of CB2679d/Dalcinonacog alfa

Intravenous Dose

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Catalyst Biosciences: lead

Study Sites (1)

Haemophilia Comprehensive Care Centre

Johannesburg, South Africa

Location

Related Publications (1)

• Faraj A, Le Moan N, Gorina E, Blouse GE, Knudsen T, Simonsson USH. Model-Informed Support of Dose Selection for Prophylactic Treatment with Dalcinonacog Alfa in Adult and Paediatric Hemophilia B Patients. Adv Ther. 2023 Sep;40(9):3739-3750. doi: 10.1007/s12325-023-02570-6. Epub 2023 Jun 21.

  PMID: 37341915 DERIVED

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Results Point of Contact

• Title: Howard Levy, Chief Medical Officer
• Organization: Catalyst Biosciences

hlevy@catbio.com

+1.650.266.6871

Study Officials

• Howard Levy, MD, PhD, MMM

  Catalyst Biosciences

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 19, 2019
• First Posted: June 24, 2019
• Study Start: June 18, 2019
• Primary Completion: February 28, 2020
• Study Completion: April 30, 2020
• Last Updated: August 9, 2021
• Results First Posted: August 9, 2021

Record last verified: 2021-08

Data Sharing

• IPD Sharing: Will not share

Locations

ZA (1)