NCT03186677

Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients

A Phase 1, Open-label, Multi-center, Dose-escalation Study to Investigate the Safety, Pharmacokinetics and Pharmacodynamics of ISU304 in Previously Treated Hemophilia B Patients

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: ISU Abxis Co., Ltd. · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This study is a phase 1, open-label, multi-center, dose-escalation study to investigate the safety, pharmacokinetics and pharmacodynamics of ISU304/CB2679d in previously treated hemophilia B patients.

Detailed description

This study is a phase 1, open-label, multi-center, dose-escalation study to investigate the safety, pharmacokinetics, and pharmacodynamics of ISU304/CB2679d/Dalcinonacog alfa in previously treated Hemophilia B patients. This study is comprised of 5 cohorts. Each cohort may receive an intravenous administration of 75 IU/kg, with subcutaneous administrations from 75 IU/kg to 150 IU/kg. During the study period, a subject may be hospitalized to facilitate the collection of blood samples for pharmacokinetic (PK)/pharmacodynamic (PD) analysis. The Data Safety Monitoring Board (DSMB) and Data Monitoring Committee (DMC) will be operated after the end of Cohorts 1 to 4. These committees will monitor the PK/PD and safety data from each cohort to determine the continuation of next cohort (Cohorts 2 to 5), target dose, and blood sampling period for PK/PD (including timing of collection). Additional subjects may be enrolled in all cohorts or cohorts may be canceled depending on the results of PK/PD analysis. A cohort of subcutaneous dosing at 300 IU/kg was cancelled as single-dose PK is uninformative.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
BIOLOGICAL	ISU304/CB2679d/Dalcinonacog alfa 75~150 IU/kg	ISU304/CB2679d/Dalcinonacog alfa 75\~150 IU/kg by intravenous or subcutaneous
BIOLOGICAL	BeneFIX	BeneFIX 75 IU/kg, intravenous administration

Timeline

• Start date: 2017-06-03
• Primary completion: 2018-10-10
• Completion: 2019-02-22
• First posted: 2017-06-14
• Last updated: 2020-11-10
• Results posted: 2020-11-10

Locations

3 sites across 1 country: South Korea