Phase 3b Continuation Study of the Safety and Efficacy of Prophylactic BAX 855 in PTPs With Severe Hemophilia A
Verified October 2013 by Baxter Healthcare Corporation
Information provided by (Responsible Party):
Baxter Healthcare Corporation
First received: September 16, 2013
Last updated: October 30, 2013
Last verified: October 2013
To continue the evaluation of the safety and efficacy of prophylaxis with BAX 855 for the prevention and treatment of bleeding episodes in previously treated patients (PTPs) (children and adults from 0 to 75 years of age) with severe hemophilia A.
Biological: PEGylated Recombinant Factor VIII
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
||A Phase 3b Continuation Study of the Safety and Efficacy of PEGylated Recombinant Factor VIII (PEG-rFVIII; BAX 855) in Prophylaxis of Bleeding in Previously Treated Patients With Severe Hemophilia A
Primary Outcome Measures:
Secondary Outcome Measures:
- Total annualized bleed rate (ABR) - Spontaneous and traumatic bleeding episodes [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Rate of success of BAX 855 for treatment of breakthrough bleeding episodes [ Time Frame: 36 months ] [ Designated as safety issue: No ]
A breakthrough bleeding episode (BE) is a BE during prophylaxis
- Number of BAX 855 infusions needed to treat bleeding episodes [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Time intervals between bleeding episodes [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Weight-adjusted consumption of BAX 855 [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Occurrence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 36 months ] [ Designated as safety issue: Yes ]
- Changes in vital signs and clinical laboratory parameters [ Time Frame: 36 months ] [ Designated as safety issue: Yes ]
Clinical laboratory parameters include hematology, clinical chemistry, and lipids
- Immunogenicity: Binding antibodies [ Time Frame: 36 months ] [ Designated as safety issue: Yes ]
- Binding antibodies (IgG and IgM) to Factor VIII, BAX855, and polyethylene glycol (PEG)
- A 72-hour washout period is required
- Immunogenicity: Anti-Chinese hamster ovary (CHO) antibodies [ Time Frame: 36 months ] [ Designated as safety issue: Yes ]
A 72-hour washout period is required
- Change from baseline in patient reported outcomes: Bleed and pain severity [ Time Frame: 36 months ] [ Designated as safety issue: No ]
Bleed and pain severity measured using the Haemo-SYM questionnaire
- Change from baseline in patient reported outcomes: health-related quality of life (HRQoL) [ Time Frame: 36 months ] [ Designated as safety issue: No ]
HRQoL assessed using the Short Form-36 Questionnaire (SF-36)
| Estimated Enrollment:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||May 2017 (Final data collection date for primary outcome measure)
30-80 ±5 IU/kg twice weekly to once per week. After each consecutive 6 months of being treated in this study, the dose and/or frequency may be adjusted, depending on the participant's spontaneous annualized bleed rate (ABR) estimated at those intervals.
Biological: PEGylated Recombinant Factor VIII
Other Name: BAX 855
|Ages Eligible for Study:
||up to 75 Years
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
Main Inclusion Criteria:
- Participants from other BAX 855 studies can be provided with the continuation study informed consent form (ICF) prior to the end of study visit to review and consider participation in this continuation study. These participants will complete any additional screening assessments within 2 weeks of the previous study's end of study visit and will return to the study site within 6 weeks of the previous study end of study visit to confirm eligibility for this continuation study.
- BAX 855 naïve participants who are ≥ 12 years of age can only be enrolled in this continuation study after enrollment in the phase 2/3 pivotal study is closed. BAX 855 naïve participants who are < 12 years of age and in countries where the pediatric PTP study is being conducted can only be enrolled in this continuation study after enrollment in the pediatric PTP study is closed.
- Participants coming from other BAX 855 studies into the continuation study, will be considered enrolled only after they have completed all procedures and assessments at the End of Study Visit in the previous BAX 855 study
- Participant and/or legal representative has/have voluntarily provided signed informed consent
- Participant is from 0 to 75 years of age at screening
- Participant is male with severe hemophilia A (Factor VIII (FVIII) clotting activity < 1%) as confirmed by central laboratory at screening (after at least a 72-hour washout period) or a documented FVIII clotting activity <1% (confirmation is only required for BAX 855 naïve participants)
- Participant has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for ≥150 documented exposure days (EDs)
- Participant is currently receiving prophylaxis or on-demand therapy with FVIII
- Participant has a Karnofsky or Lansky performance score of ≥ 60.
- Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at screening.
- Participant is hepatitis C virus negative (HCV-) by antibody or PCR testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
- Participant is willing and able to comply with the requirements of the protocol
Main Exclusion Criteria:
- Participant has detectable FVIII inhibitory antibodies (≥ 0.4 Bethesda unit (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
- Participant has history of FVIII inhibitory antibodies (≥ 0.4 BU using the Nijmegen modification of the Bethesda assay or ≥ 0.6 BU using the Bethesda assay) at any time prior to screening.
- Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
- Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG), or Tween 80.
- Participant has severe chronic hepatic dysfunction.
- Participant has severe renal impairment.
- Participant has current or recent (<30 days) use of other PEGylated drugs prior to study participation or scheduled use of such drugs during study participation.
- Participant has participated in another clinical study involving an investigational product (IP) other than BAX 855 or 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.
- Participant has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
- Participant is a family member or employee of the investigator.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01945593
|Puget Sound Blood Group
|Seattle, Washington, United States, 98104 |
Baxter Healthcare Corporation
||Brigitt Abbuehl, MD
||Baxter Innovations GmbH
No publications provided
||Baxter Healthcare Corporation
History of Changes
|Other Study ID Numbers:
|Study First Received:
||September 16, 2013
||October 30, 2013
||Australia: Department of Health and Ageing Therapeutic Goods Administration
Austria: Agency for Health and Food Safety
Belgium: Federal Agency for Medicinal Products and Health Products
Bulgaria: Bulgarian Drug Agency
Canada: Health Canada
Czech Republic: State Institute for Drug Control
Hong Kong: Department of Health
Hungary: National Institute of Pharmacy
India: Drugs Controller General of India
Israel: Israeli Health Ministry Pharmaceutical Administration
Italy: The Italian Medicines Agency
Japan: Pharmaceuticals and Medical Devices Agency
Korea: Food and Drug Administration
Lithuania: State Medicine Control Agency - Ministry of Health
Malaysia: Ministry of Health
Netherlands: Medicines Evaluation Board (MEB)
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Romania: National Medicines Agency
Russia: FSI Scientific Center of Expertise of Medical Application
Serbia: Medicines and Medical Devices Agency
Singapore: Health Sciences Authority
Spain: Spanish Agency of Medicines
Sweden: Medical Products Agency
Switzerland: Federal Office of Public Health
Taiwan: Department of Health
Thailand: Food and Drug Administration
Turkey: Ministry of Health
Ukraine: State Pharmacological Center - Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on September 18, 2014
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Coagulation Protein Disorders
Genetic Diseases, Inborn