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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01454739




Registration number
NCT01454739
Ethics application status
Date submitted
29/09/2011
Date registered
19/10/2011
Date last updated
19/12/2020

Titles & IDs
Public title
Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A
Scientific title
An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A
Secondary ID [1] 0 0
2011-003072-37
Secondary ID [2] 0 0
8HA01EXT
Universal Trial Number (UTN)
Trial acronym
ASPIRE
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: Drugs - rFVIIIFc

Experimental: On-Demand - The individual dose of rFVIIIFc to treat bleeding episodes will be based on participant's clinical condition, type and severity of the bleeding event, and if indicated, Factor VIII (FVIII) levels.

Experimental: Prophylaxis - Tailored prophylaxis, Weekly prophylaxis or Personalized prophylaxis available.


Treatment: Drugs: rFVIIIFc
Administered as specified in the treatment arm.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Any Positive Inhibitor Development
Timepoint [1] 0 0
Approximately 5 years
Secondary outcome [1] 0 0
Annualized Bleeding Rate (ABR)
Timepoint [1] 0 0
Approximately 5 years
Secondary outcome [2] 0 0
Annualized Spontaneous Joint Bleeding Episodes
Timepoint [2] 0 0
Approximately 5 years
Secondary outcome [3] 0 0
Total Number of Exposure Days (EDs)
Timepoint [3] 0 0
Approximately 5 years
Secondary outcome [4] 0 0
Annualized rFVIIIFc Consumption (International Units Per Kilogram [IU/kg])
Timepoint [4] 0 0
Approximately 5 years
Secondary outcome [5] 0 0
Physicians' Global Assessment of Participant's Response to rFVIIIFc Regimen Using a 4-Point Scale
Timepoint [5] 0 0
Approximately 5 years
Secondary outcome [6] 0 0
Participant's Assessment of Response (Excellent or Good Response) to rFVIIIFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
Timepoint [6] 0 0
Approximately 5 years

Eligibility
Key inclusion criteria
Key

* Subjects who have completed previous rFVIIIFc studies (NCT01181128, NCT02083965, NCT01458106 and NCT02502149)
* Ability to understand purposes and risks of the study and to provide signed and dated informed consent (or assent, as applicable).

Key
Minimum age
0 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Confirmed positive high-titer inhibitor (=5.00 BU/mL).

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Study design
Purpose of the study
Treatment
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
1/12/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/10/2017
Sample size
Target
Accrual to date
Final
240
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Research Site - Camperdown
Recruitment hospital [2] 0 0
Research Site - South Brisbane
Recruitment hospital [3] 0 0
Research Site - Adelaide
Recruitment hospital [4] 0 0
Research Site - Melbourne
Recruitment hospital [5] 0 0
Research site - Melbourne
Recruitment hospital [6] 0 0
Research Site - Murdoch
Recruitment hospital [7] 0 0
Research Site - Subiaco
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
5000 - Adelaide
Recruitment postcode(s) [4] 0 0
3052 - Melbourne
Recruitment postcode(s) [5] 0 0
3181 - Melbourne
Recruitment postcode(s) [6] 0 0
6150 - Murdoch
Recruitment postcode(s) [7] 0 0
6008 - Subiaco
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Indiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Iowa
Country [5] 0 0
United States of America
State/province [5] 0 0
Louisiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
Nevada
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Oregon
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
United States of America
State/province [16] 0 0
Washington
Country [17] 0 0
Austria
State/province [17] 0 0
Vienna
Country [18] 0 0
Belgium
State/province [18] 0 0
Brussels
Country [19] 0 0
Brazil
State/province [19] 0 0
Sao Paulo
Country [20] 0 0
Canada
State/province [20] 0 0
British Columbia
Country [21] 0 0
Canada
State/province [21] 0 0
Ontario
Country [22] 0 0
France
State/province [22] 0 0
Rhone
Country [23] 0 0
Germany
State/province [23] 0 0
Nordrhein Westfalen
Country [24] 0 0
Germany
State/province [24] 0 0
Berlin
Country [25] 0 0
Hong Kong
State/province [25] 0 0
New Territories
Country [26] 0 0
Hong Kong
State/province [26] 0 0
Hong Kong
Country [27] 0 0
India
State/province [27] 0 0
Delhi
Country [28] 0 0
India
State/province [28] 0 0
Karnataka
Country [29] 0 0
India
State/province [29] 0 0
Maharashtra
Country [30] 0 0
India
State/province [30] 0 0
Punjab
Country [31] 0 0
India
State/province [31] 0 0
Tamilnadu
Country [32] 0 0
Ireland
State/province [32] 0 0
Dublin
Country [33] 0 0
Israel
State/province [33] 0 0
Ramat Gan
Country [34] 0 0
Italy
State/province [34] 0 0
Firenze
Country [35] 0 0
Italy
State/province [35] 0 0
Milano
Country [36] 0 0
Italy
State/province [36] 0 0
Vicenza
Country [37] 0 0
Japan
State/province [37] 0 0
Aichi-Ken
Country [38] 0 0
Japan
State/province [38] 0 0
Fukuoka-Ken
Country [39] 0 0
Japan
State/province [39] 0 0
Kanagawa-Ken
Country [40] 0 0
Japan
State/province [40] 0 0
Nara-Ken
Country [41] 0 0
Japan
State/province [41] 0 0
Tokyo-To
Country [42] 0 0
Netherlands
State/province [42] 0 0
Groningen
Country [43] 0 0
New Zealand
State/province [43] 0 0
Auckland
Country [44] 0 0
New Zealand
State/province [44] 0 0
Christchurch
Country [45] 0 0
New Zealand
State/province [45] 0 0
Hamilton
Country [46] 0 0
New Zealand
State/province [46] 0 0
Palmerston North
Country [47] 0 0
New Zealand
State/province [47] 0 0
Wellington
Country [48] 0 0
Poland
State/province [48] 0 0
Lublin
Country [49] 0 0
South Africa
State/province [49] 0 0
Gauteng
Country [50] 0 0
South Africa
State/province [50] 0 0
Western Cape
Country [51] 0 0
Spain
State/province [51] 0 0
Barcelona
Country [52] 0 0
Spain
State/province [52] 0 0
Madrid
Country [53] 0 0
Sweden
State/province [53] 0 0
Göteborg
Country [54] 0 0
Switzerland
State/province [54] 0 0
Zuerich
Country [55] 0 0
United Kingdom
State/province [55] 0 0
Cambridgeshire
Country [56] 0 0
United Kingdom
State/province [56] 0 0
Greater London
Country [57] 0 0
United Kingdom
State/province [57] 0 0
Hampshire
Country [58] 0 0
United Kingdom
State/province [58] 0 0
London
Country [59] 0 0
United Kingdom
State/province [59] 0 0
Strathclyde

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Bioverativ Therapeutics Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A. The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in participants with hemophilia A.
Trial website
https://clinicaltrials.gov/study/NCT01454739
Trial related presentations / publications
Nolan B, Mahlangu J, Pabinger I, Young G, Konkle BA, Barnes C, Nogami K, Santagostino E, Pasi KJ, Khoo L, Winding B, Yuan H, Fruebis J, Rudin D, Oldenburg J. Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study. Haemophilia. 2020 May;26(3):494-502. doi: 10.1111/hae.13953. Epub 2020 Mar 30.
Nolan B, Mahlangu J, Perry D, Young G, Liesner R, Konkle B, Rangarajan S, Brown S, Hanabusa H, Pasi KJ, Pabinger I, Jackson S, Cristiano LM, Li X, Pierce GF, Allen G. Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A. Haemophilia. 2016 Jan;22(1):72-80. doi: 10.1111/hae.12766. Epub 2015 Jul 27.
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Bioverativ Therapeutics Inc.
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01454739