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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01945593




Registration number
NCT01945593
Ethics application status
Date submitted
16/09/2013
Date registered
18/09/2013

Titles & IDs
Public title
BAX 855 Continuation
Scientific title
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
Secondary ID [1] 0 0
2013-002236-24
Secondary ID [2] 0 0
261302
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - BAX855

Experimental: Fixed BAX855 prophylaxis - 45-80 IU/kg twice weekly to once per week.

Experimental: Pharmacokinetic (PK)-tailored BAX 855 prophylaxis - PK-tailored prophylactic BAX855 regimen based on participant's individual PK profile to maintain a Factor VIII (FVIII) trough level


Treatment: Other: BAX855
Antihemophilic Factor (Recombinant), PEGylated
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII)
Timepoint [1] 0 0
Baseline through end of study (53 months)
Primary outcome [2] 0 0
Annualized Bleed Rate (ABR) - Spontaneous Bleeds
Timepoint [2] 0 0
Baseline through end of study (53 months)
Secondary outcome [1] 0 0
Total Annualized Bleed Rate (ABR)
Timepoint [1] 0 0
Baseline through end of study (53 months)
Secondary outcome [2] 0 0
Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes
Timepoint [2] 0 0
Baseline through end of study (53 months)
Secondary outcome [3] 0 0
BAX 855 Infusions Needed to Treat Bleeding Episodes
Timepoint [3] 0 0
Baseline through end of study (53 months)
Secondary outcome [4] 0 0
Total Time Intervals Between Bleeding Episodes
Timepoint [4] 0 0
Baseline through end of study (53 months)
Secondary outcome [5] 0 0
Average Dose of BAX 855 Per Prophylactic Infusion
Timepoint [5] 0 0
Baseline through end of study (53 months)
Secondary outcome [6] 0 0
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [6] 0 0
Baseline through end of study (53 months)
Secondary outcome [7] 0 0
Change From Baseline in Body Temperature
Timepoint [7] 0 0
Baseline, end of study (53 months)
Secondary outcome [8] 0 0
Change From Baseline in Pulse Rate
Timepoint [8] 0 0
Baseline, end of study (53 months)
Secondary outcome [9] 0 0
Change From Baseline in Respiratory Rate
Timepoint [9] 0 0
Baseline, end of study (53 months)
Secondary outcome [10] 0 0
Change From Baseline in Blood Pressure
Timepoint [10] 0 0
Baseline, end of study (53 months)
Secondary outcome [11] 0 0
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Timepoint [11] 0 0
Baseline through end of study (53 months)
Secondary outcome [12] 0 0
Number of Participants With Shifts in Hematology Laboratory Assessments
Timepoint [12] 0 0
Baseline through end of study (53 months)
Secondary outcome [13] 0 0
Number of Participants With Shifts in Lipid Panel Assessments
Timepoint [13] 0 0
Baseline through end of study (53 months)
Secondary outcome [14] 0 0
Number of Participants With Binding Antibodies
Timepoint [14] 0 0
Baseline through end of study (53 months)
Secondary outcome [15] 0 0
Number of Participants With Anti-Chinese Hamster Ovary (CHO) Antibodies
Timepoint [15] 0 0
Baseline through end of study (53 months)
Secondary outcome [16] 0 0
Change From Baseline in Bleed Severity
Timepoint [16] 0 0
Baseline, end of study (53 months)
Secondary outcome [17] 0 0
Change From Baseline in Pain Severity
Timepoint [17] 0 0
Baseline, end of study (53 months)
Secondary outcome [18] 0 0
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
Timepoint [18] 0 0
Baseline, end of study (53 months)
Secondary outcome [19] 0 0
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
Timepoint [19] 0 0
Baseline, end of study (53 months)

Eligibility
Key inclusion criteria
INCLUSION CRITERIA

Participants Transitioning from Other BAX 855 Studies:

Participants transitioning 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 (± 1) weeks of the previous study end of study visit to confirm eligibility for this continuation study.

* Participants transitioning from other BAX 855 studies who meet ALL of the following criteria are eligible for this study:

1. Participant has completed a previous BAX 855 study and is willing to immediately transition into this continuation study.
2. Participant is =75 years of age at screening of the previous BAX 855 study.
3. Participant continues to have a Karnofsky (for participants aged = 16 years) or Lansky (for participants aged < 16 years) performance score of = 60.
4. 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.
5. Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
6. If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
7. Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.
* BAX 855 Naïve Participants:

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 can only be enrolled in this continuation study after enrollment in the pediatric previously treated patient (PTP) study is closed.

- Enrolment of BAX 855 naïve participants will only start once the sponsor has notified the study sites accordingly.

BAX 855 naïve participants who meet ALL of the following criteria are eligible for this study:

1. Participant is =75 years of age at screening.
2. Participant is naïve to BAX 855.
3. Participant has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by central laboratory at screening after at least a 72-hour washout period.
4. Participant aged = 6 years has documented previous treatment with plasma-derived FVIII or rFVIII for = 150 exposure days (EDs).
5. Participant aged < 6 years has documented previous treatment with plasma-derived FVIII concentrates or rFVIII for = 50 EDs.
6. Participant is currently receiving prophylaxis or on-demand therapy with FVIII.
7. Participant has a Karnofsky (for participants aged = 16 years) or Lansky (for participants aged < 16 years) performance score of = 60.
8. Participant is HIV-; or HIV+ with stable disease and CD4+ count = 200 cells/mm^3, as confirmed by central laboratory at screening.
9. Participant is 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.
10. If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
11. Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.

EXCLUSION CRITERA

- Participants Transitioning from Other BAX 855 Studies:

Participants transitioning from other BAX 855 studies who meet ANY of the following criteria are not eligible for this study:

1. Participant had detectable factor VIII (FVIII) inhibitory antibodies (= 0.6 Bethesda unit (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
2. Participant has developed FVIII inhibitory antibodies (= 0.6 BU using the Nijmegen modification of the Bethesda assay as determined at central laboratory in a previous BAX 855 study).
3. Participant has acquired a hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease) in a previous BAX 855 study.
4. Participant has severe chronic hepatic dysfunction (eg, = 5 times upper limit of normal alanine aminotransferase [ALT], as confirmed by central laboratory at screening).
5. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
6. Participant experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.
7. Participant is scheduled to use other PEGylated drugs during study participation.
8. Participant is planning to take part in any other clinical study during the course of the continuation study, with the exception of any other parallel BAX 855 study.
9. 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.
10. Participant is a family member or employee of the investigator.

* BAX 855 Naïve Participants:

BAX 855 naïve participants who meet ANY of the following criteria are not eligible for this study:

1. Participant has detectable FVIII inhibitory antibodies (= 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
2. Participant has history of FVIII inhibitory antibodies (= 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening.
3. Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
4. Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG), or Tween 80.
5. Participant has severe chronic hepatic dysfunction eg, = 5 times upper limit of normal ALT, as confirmed by central laboratory at screening).
6. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
7. Participant experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.
8. 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.
9. Participant has participated in another clinical study involving an IP other than BAX 855 or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an investigational product (IP) or investigational device during the course of this study.
10. 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.
11. Participant is a family member or employee of the investigator.
Minimum age
No limit
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Prevention
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/10/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
2/03/2018
Sample size
Target
Accrual to date
Final
218
Recruitment in Australia
Recruitment state(s)
SA,VIC,WA
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [3] 0 0
Fremantle Hospital - Fremantle
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment postcode(s) [3] 0 0
6160 - Fremantle
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Kentucky
Country [6] 0 0
United States of America
State/province [6] 0 0
Louisiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Oklahoma
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
South Carolina
Country [14] 0 0
United States of America
State/province [14] 0 0
Utah
Country [15] 0 0
United States of America
State/province [15] 0 0
Washington
Country [16] 0 0
Austria
State/province [16] 0 0
Linz
Country [17] 0 0
Austria
State/province [17] 0 0
Vienna
Country [18] 0 0
Bulgaria
State/province [18] 0 0
Plovdiv
Country [19] 0 0
Bulgaria
State/province [19] 0 0
Sofia
Country [20] 0 0
Bulgaria
State/province [20] 0 0
Varna
Country [21] 0 0
Czechia
State/province [21] 0 0
Brno
Country [22] 0 0
Czechia
State/province [22] 0 0
Olomouc
Country [23] 0 0
Czechia
State/province [23] 0 0
Praha 5
Country [24] 0 0
Germany
State/province [24] 0 0
Niedersachsen
Country [25] 0 0
Germany
State/province [25] 0 0
Nordrhein Westfalen
Country [26] 0 0
Germany
State/province [26] 0 0
Berlin
Country [27] 0 0
Germany
State/province [27] 0 0
Hamburg
Country [28] 0 0
Hong Kong
State/province [28] 0 0
Shatin
Country [29] 0 0
Israel
State/province [29] 0 0
Haifa
Country [30] 0 0
Israel
State/province [30] 0 0
Ramat-Gan
Country [31] 0 0
Japan
State/province [31] 0 0
Aichi-Ken
Country [32] 0 0
Japan
State/province [32] 0 0
Fukuoka-Ken
Country [33] 0 0
Japan
State/province [33] 0 0
Hiroshima-Ken
Country [34] 0 0
Japan
State/province [34] 0 0
Kanagawa-Ken
Country [35] 0 0
Japan
State/province [35] 0 0
Nara-Ken
Country [36] 0 0
Japan
State/province [36] 0 0
Tokyo-To
Country [37] 0 0
Japan
State/province [37] 0 0
Tokyo
Country [38] 0 0
Korea, Republic of
State/province [38] 0 0
Jeollanam-do
Country [39] 0 0
Korea, Republic of
State/province [39] 0 0
Busan
Country [40] 0 0
Korea, Republic of
State/province [40] 0 0
Daejeon
Country [41] 0 0
Korea, Republic of
State/province [41] 0 0
Seoul
Country [42] 0 0
Korea, Republic of
State/province [42] 0 0
Ulsan
Country [43] 0 0
Lithuania
State/province [43] 0 0
Vilnius
Country [44] 0 0
Malaysia
State/province [44] 0 0
Penang
Country [45] 0 0
Malaysia
State/province [45] 0 0
Sarawak
Country [46] 0 0
Malaysia
State/province [46] 0 0
Selangor
Country [47] 0 0
Malaysia
State/province [47] 0 0
Kuala Lumpur
Country [48] 0 0
Malaysia
State/province [48] 0 0
Pulau Pinang
Country [49] 0 0
Netherlands
State/province [49] 0 0
Amsterdam
Country [50] 0 0
Poland
State/province [50] 0 0
Gdansk
Country [51] 0 0
Poland
State/province [51] 0 0
Lodz
Country [52] 0 0
Romania
State/province [52] 0 0
Bucuresti
Country [53] 0 0
Russian Federation
State/province [53] 0 0
Kirov
Country [54] 0 0
Russian Federation
State/province [54] 0 0
Krasnoyarsk
Country [55] 0 0
Spain
State/province [55] 0 0
Baleares
Country [56] 0 0
Spain
State/province [56] 0 0
La Coruña
Country [57] 0 0
Spain
State/province [57] 0 0
Málaga
Country [58] 0 0
Spain
State/province [58] 0 0
Madrid
Country [59] 0 0
Spain
State/province [59] 0 0
Valencia
Country [60] 0 0
Sweden
State/province [60] 0 0
Malmo
Country [61] 0 0
Sweden
State/province [61] 0 0
Stockholm
Country [62] 0 0
Switzerland
State/province [62] 0 0
Zuerich
Country [63] 0 0
Taiwan
State/province [63] 0 0
Taichung
Country [64] 0 0
Taiwan
State/province [64] 0 0
Taipei
Country [65] 0 0
Turkey
State/province [65] 0 0
Ankara
Country [66] 0 0
Turkey
State/province [66] 0 0
Antalya
Country [67] 0 0
Turkey
State/province [67] 0 0
Istanbul
Country [68] 0 0
Ukraine
State/province [68] 0 0
Donetsk
Country [69] 0 0
Ukraine
State/province [69] 0 0
Lviv
Country [70] 0 0
United Kingdom
State/province [70] 0 0
Avon
Country [71] 0 0
United Kingdom
State/province [71] 0 0
Greater London
Country [72] 0 0
United Kingdom
State/province [72] 0 0
Greater Manchester
Country [73] 0 0
United Kingdom
State/province [73] 0 0
Hampshire
Country [74] 0 0
United Kingdom
State/province [74] 0 0
Leicestershire
Country [75] 0 0
United Kingdom
State/province [75] 0 0
West Midlands
Country [76] 0 0
United Kingdom
State/province [76] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Baxalta now part of Shire
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Baxalta Innovations GmbH, now part of Shire
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Takeda
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
When will data be available (start and end dates)?
Available to whom?
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/takeda/


What supporting documents are/will be available?

TypeOther DetailsAttachment
Study protocol Study Protocol: Protocol https://cdn.clinicaltrials.gov/large-docs/93/NCT01945593/Prot_000.pdf
Study protocol Study Protocol: Protocol Amendment 1 https://cdn.clinicaltrials.gov/large-docs/93/NCT01945593/Prot_001.pdf
Study protocol Study Protocol: Protocol Amendment 4 https://cdn.clinicaltrials.gov/large-docs/93/NCT01945593/Prot_002.pdf
Study protocol Study Protocol: Protocol Amendment 7 https://cdn.clinicaltrials.gov/large-docs/93/NCT01945593/Prot_003.pdf
Statistical analysis plan https://cdn.clinicaltrials.gov/large-docs/93/NCT01945593/SAP_004.pdf
Study protocol Study Protocol: Protocol https://cdn.clinicaltrials.gov/large-docs/93/NCT01945593/Prot_000.pdf
Study protocol Study Protocol: Protocol Amendment 1 https://cdn.clinicaltrials.gov/large-docs/93/NCT01945593/Prot_001.pdf
Study protocol Study Protocol: Protocol Amendment 4 https://cdn.clinicaltrials.gov/large-docs/93/NCT01945593/Prot_002.pdf
Study protocol Study Protocol: Protocol Amendment 7 https://cdn.clinicaltrials.gov/large-docs/93/NCT01945593/Prot_003.pdf
Statistical analysis plan https://cdn.clinicaltrials.gov/large-docs/93/NCT01945593/SAP_004.pdf



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT01945593