BAX 855 Dose-Escalation Safety Study

Baxalta

Status and phase: Completed; Phase 1

Conditions: Hemophilia A

Treatments: Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method; Biological: PEGylated Recombinant Factor VIII

Study type: Interventional

Funder types: Industry

Identifiers

NCT01599819

261101

2011-002011-28 (EudraCT Number)

Details and patient eligibility

About

The objectives of this study are to assess the tolerability and safety after single dose treatments of BAX 855 in previously treated patients (PTPs) with severe hemophilia A, to determine the pharmacokinetic (PK) parameters of BAX 855 compared in crossover with ADVATE, and to evaluate the impact of anti-polyethylene glycol (PEG) antibodies on PK parameters.

Enrollment: 19 patients

Sex: Male

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• The subject is male and is 18 to 65 years of age at the time of screening
• The subject has severe hemophilia A (factor VIII level < 1%)
• The subject was previously treated with plasma-derived factor VIII (FVIII) concentrates or recombinant FVIII for at least 150 exposure days (EDs)

Exclusion criteria

• The subject has a detectable FVIII inhibitor at screening, with a titer >= 0.6 BU
• The subject has a documented history of FVIII inhibitors with a titer >= 0.4 BU at any time prior to screening
• The subject has a known hypersensitivity towards mouse or hamster proteins or to polyethylene glycol (PEG)

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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19 participants in 2 patient groups

Cohort 1

Experimental group

Description:

Low dose of ADVATE followed by low dose of BAX 855

Treatment:

Biological: PEGylated Recombinant Factor VIII

Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method

Cohort 2

Experimental group

Description:

High dose of ADVATE followed by high dose of BAX 855

Treatment:

Biological: PEGylated Recombinant Factor VIII

Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method