BAX 826 Dose-Escalation Safety Study

Baxalta

Status and phase

Completed

Phase 1

Conditions

Hemophilia A

Treatments

Biological: Octocog alfa

Biological: BAX 826

Study type

Interventional

Funder types

Industry

Identifiers

NCT02716194

291501

2015-004079-60 (EudraCT Number)

Details and patient eligibility

About

To assess tolerability and safety of BAX 826 after a single infusion in previously treated patients (PTPs) with severe hemophilia A
To determine the pharmacokinetic (PK) parameters of BAX 826 compared to ADVATE
To evaluate immunogenicity of polysialic acid linked to Factor VIII (FVIII)

Enrollment

40 patients

Sex

Male

Ages

18 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Previously treated male participants aged 18 to 65 years (inclusive) at the time of screening
Diagnosis of severe hemophilia A (Factor VIII level <1%)
Previously treated with FVIII concentrates for ≥150 documented Exposure Days (EDs)
Karnofsky performance score of ≥60
Human immunodeficiency virus negative (HIV-); or HIV+ with stable disease
Hepatitis C virus negative (HCV-); or HCV+ with chronic stable hepatitis as assessed by the investigator
Able to understand and have provided written informed consent including signature on an informed consent form (ICF) approved by an ethics committee (EC)
Have provided written authorization for use and disclosure of protected health information
Agree to abide by the study schedule and to return for the required assessments
Willing and able to comply with the requirements of the protocol

Exclusion criteria

Detectable FVIII inhibitor at screening, with a titer ≥0.6 Bethesda Unit (BU)
Documented history of FVIII inhibitors with a titer ≥0.4 BU at any time prior to screening
Known clinical hypersensitivity towards mouse or hamster proteins or to polysialic acid (PSA)
Scheduled elective surgery during study participation
Severe chronic hepatic dysfunction
Severe renal impairment
Currently receiving, or has recently received (less than 3 months prior to study participation), or is scheduled to receive during the course of the study, other PSA-ylated drugs
Have received another investigational drug within 30 days prior to study entry and/or is scheduled to receive additional investigational drug during the course of the study in the context of another investigational drug study
Diagnosis of an inherited or acquired hemostatic defect other than hemophilia A
Currently receiving, or scheduled to receive during the course of the study, an immune-modulating drug other than antiretroviral chemotherapy
Has a clinically significant medical, psychiatric or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect the safety or compliance of the participant during the study
Is a family member or employee of the investigator

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Crossover Assignment

Masking

None (Open label)

40 participants in 3 patient groups

Cohort 1 - Low dose

Experimental group

Description:

The study is comprised of 3 dose cohorts and two dose escalation steps \[Cohort 1: 10 evaluable participants; Cohort 2: 10 evaluable participants; Cohort 3: 10 evaluable participants\]. Participants will be recruited to the next dose level only after short-term safety has been reviewed and subject to approval by a Safety Review Committee at the preceding dose level.

Treatment:

Biological: BAX 826

Biological: Octocog alfa

Cohort 2 - Medium dose

Experimental group

Description:

The study is comprised of 3 dose cohorts and two dose escalation steps \[Cohort 1: 10 evaluable participants; Cohort 2: 10 evaluable participants; Cohort 3: 10 evaluable participants\]. Participants will be recruited to the next dose level only after short-term safety has been reviewed and subject to approval by a Safety Review Committee at the preceding dose level.

Treatment:

Biological: BAX 826

Biological: Octocog alfa

Cohort 3 - High dose

Experimental group

Description:

The study is comprised of 3 dose cohorts and two dose escalation steps \[Cohort 1: 10 evaluable participants; Cohort 2: 10 evaluable participants; Cohort 3: 10 evaluable participants\]. Participants will be recruited to the next dose level only after short-term safety has been reviewed and subject to approval by a Safety Review Committee at the preceding dose level.

Treatment:

Biological: BAX 826

Biological: Octocog alfa

Trial contacts and locations

28

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