Baxalta

Principal Investigator: George Rodgers
Keywords: Hematology , Hemophilia , FVIII Department: Hematology
IRB Number: 00088973 Co Investigator:  
Specialty: Hematology/BMT
Sub Specialties:
Recruitment Status: Recruiting

Contact Information

Renee Rinaldi
renee.rinaldi@hsc.utah.edu
801-585-5341

Brief Summary

This study is being carried out to look at the safety and effectiveness of an investigational drug called BAX 855  in preventing bleeds in patients with hemophilia A.

The study will also look at how long BAX 855 stays in the blood after it has been administered; this is known as pharmacokinetics (PK).

The study drug (BAX 855), is a blood-clotting factor made in the laboratory for use as a medication. It is an investigational Factor VIII medication similar to a medication called ADVATE. The study drug is a form of ADVATE that has been modified to extend the time it circulates in the bloodstream. The modification involves the attachment of polyethylene glycol (PEG) to ADVATE (in a process called pegylation). PEG is a substance that has been used to prolong the circulation time in the blood of many other drugs, of which some are currently available. The idea of adding PEG to ADVATE is to allow for less frequent dosing in the treatment of hemophilia A. The study drug has not yet been approved for commercial use anywhere in the world.

The study drug is administered as an “infusion”, drip into a vein in the patient's arm.

This study will look at the use of two different dosing schedules of the study drug: twice weekly (standard dosing), or once every other day (more frequent dosing). The specific schedule to which patients are assigned will be chosen by randomization. Patients will have an equal chance of being assigned to either of the dosing schedules. This study is open-label, patients will be told which dosing schedule of the study drug they are assigned to.

Inclusion Criteria

Inclusion Criteria for Subjects Transitioning from Another BAX 855 study:

1. Subject has completed the end of study visit of a BAX 855 study or is transitioning from the

ongoing Continuation Study 261302

2. Subject is either receiving on-demand or prophylactic treatment with BAX 855 and had an ABR of

≥ 2 documented and treated during the past 12 months

3. Subject is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+

count ≥ 200 cells/mm3, as confirmed by central laboratory

4. Subject is willing and able to comply with the requirements of the protocol

Inclusion Criteria for Newly Recruited Subjects:

1. Subject is 12 to 65 years old at the time of screening

2. Subject has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by central laboratory

or by historically documented FVIII clotting activity performed by a certified clinical laboratory

and/or a FVIII gene mutation consistent with severe hemophilia A

3. Subject has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII

for ≥ 150 documented exposure days (EDs)

4. Subject is either receiving on-demand treatment or prophylactic treatment and had an annual

bleeding rate of ≥ 2 documented and treated during the past 12 months.

5. Subject has a Karnofsky performance score of ≥ 60 at screening

6. Subject is HIV-; or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm3, as confirmed by

central laboratory at screening

7. Subject is hepatitis C virus negative (HCV-) by antibody (if positive, additional PCR testing will be

performed), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis

8. If female of childbearing potential, subject presents with a negative urine pregnancy test and agrees

to employ adequate birth control measures for the duration of the study

9. Subject is willing and able to comply with the requirements of the protocol

Exclusion Criteria

Exclusion Criteria for Newly Recruited Subjects

1. Subject has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of

the Bethesda assay) as confirmed by central laboratory at screening

2. Subject has a history of confirmed FVIII inhibitors with a titer ≥ 0.6 BU (as determined by the

Nijmegen modification of the Bethesda assay or the assay employed with the respective cut-off in

the local laboratory) at any time prior to screening

3. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A

(eg, qualitative platelet defect or von Willebrand’s disease)

4. The subject’s weight is < 35 kg or > 100 kg.

5. Subject’s platelet count is < 100,000/mL.

6. Subject has known hypersensitivity towards mouse or hamster proteins, PEG or Tween 80

7. Subject has severe chronic hepatic dysfunction [eg, ≥5 times upper limit of normal ALT and/ or

AST, as confirmed by central laboratory at screening, or a documented INR > 1.5]

8. Subject has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal)

9. Subject has current or recent (< 30 days) use of other pegylated drugs prior to study participation or

is scheduled to use such drugs during study participation

10. Subject is scheduled to receive during the course of the study, a systemic immunomodulating drug

(eg, corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or α-

interferon) other than anti-retroviral chemotherapy.

11. Subject has participated in another clinical study involving an IP or investigational device within

30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or

investigational device during the course of this study

12. Subject has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the

opinion of the investigator, would affect subject safety or compliance

13. Subject is a member of the team conducting this study or is in a dependent relationship with one of

the study team members. Dependent relationships include close relatives (ie, children,

partner/spouse, siblings, parents)