Hemophilia APhase 3ASPIRENCT01454739
Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A
A Phase 3 study testing rFVIIIFc in people with Hemophilia A, run by Bioverativ Therapeutics Inc. at 78 sites in 22 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 0 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- rFVIIIFc
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 240 participants
- Where
- 78 sitesin 22 countries
- Study dates
- Dec 2011to Oct 2017
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- Confirmed positive high-titer inhibitor (≥5.00 BU/mL).
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Trial locations and contacts
78 sites in 22 countries.
78 sites in 22 countries
United States22 sites
Research Site
Boston, Massachusetts, 02115
Research Site
Chapel Hill, North Carolina, 27599
Research Site
Cincinnati, Ohio, 45229
Research Site
East Lansing, Michigan, 48823
Research Site
Houston, Texas, 77030
Research Site
Indianapolis, Indiana, 46260
Research Site
Iowa City, Iowa, 52242
Research Site
Las Vegas, Nevada, 89109
Research Site
Los Angeles, California, 90007
Research Site
Los Angeles, California, 90027
Research Site
New Orleans, Louisiana, 70112
Research Site
Orange, California, 92868
Research Site
Philadelphia, Pennsylvania, 19104
Research Site
Philadelphia, Pennsylvania, 19107
Research Site
Pittsburgh, Pennsylvania, 15213
Research Site
Portland, Oregon, 97239
Research Site
Sacramento, California, 95817
Research Site
Salt Lake City, Utah, 84132
Research Site
San Diego, California, 92123
Research Site
Seattle, Washington, 98104
Research Site
St Louis, Missouri, 63104
Research Site
Washington D.C., District of Columbia, 20010
United Kingdom9 sites
Research Site
Basingstoke, Hampshire, RG24 9NA
Research Site
Cambridge, Cambridgeshire, CB2 0QQ
Research Site
Glasgow, Strathclyde, G3 8SJ
Research Site
Glasgow, Strathclyde, G4 0SF
Research Site
Hamstead, London, NW3 2QG
Research Site
London, Greater London, E1 1BB
Research Site
London, Greater London, SE1 7EH
Research Site
London, Greater London, WC1N 3JH
Research Site
London, SE1 7EH
Australia7 sites
Research Site
Adelaide, South Australia, 5000
Research Site
Camperdown, New South Wales, 2050
Research Site
Melbourne, Victoria, 3052
Research site
Melbourne, Victoria, 3181
Research Site
Murdoch, Western Australia, 6150
Research Site
South Brisbane, Queensland, 4101
Research Site
Subiaco, Western Australia, 6008
Japan6 sites
Research Site
Kashihara-shi, Nara, 634-8522
Research Site
Kawasaki, Kanagawa, 216-8511
Research Site
Kitakyushu, Fukuoka, 807-8556
Research Site
Nagoya, Aichi-ken, 466-8560
Research Site
Shinjuku-ku, Tokyo-To, 160-0023
Research Site
Tokyo, Tokyo-To, 167-8515
India5 sites
Research Site
Bangalore, Karnataka, 560034
Research Site
Ludhiana, Punjab, 141008
Research Site
New Delhi, National Capital Territory of Delhi, 110002
Research Site
Pune, Maharashtra, 411004
Research Site
Vellore, Tamil Nadu, 632004
New Zealand5 sites
Research Site
Auckland, 1023
Research Site
Christchurch, 8011
Research site
Hamilton, 3200
Research Site
Palmerston North, 4410
Research site
Wellington, 6021
Hong Kong3 sites
Children Cancer Centre
Hong Kong, New Territories
Sir Yue Kong Pao Center for Cancer
Hong Kong, New Territories
Research Site
Hong Kong
Italy3 sites
Research Site
Florence, 50134
Research Site
Milan, 20122
Research Site
Vicenza, 36100
Canada2 sites
Research Site
Toronto, Ontario, M5G 1X8
Research Site
Vancouver, British Columbia, V6Z 1Y6
Germany2 sites
Research Site
Berlin, 10249
Research Site
Bonn, North Rhine-Westphalia, 53127
South Africa2 sites
Research Site
Cape Town, Western Cape, 7925
Research Site
Johannesburg, Gauteng, 2193
About this study
Official 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
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.
Read the detailed description
Participant will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the participant in the preceding studies A-LONG - 997HA301 (NCT01181128), pediatric study 8HA02PED (NCT01458106), 997HA307 (NCT02083965) and 997HA309 (NCT02502149).
- Conditions
- Hemophilia A
- Being tested
- rFVIIIFc
- Keywords
- rFVIIIFc, A-LONG Extension
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rFVIIIFcDrug
Also called Eloctate, recombinant coagulation factor VIII Fc fusion protein, BIIB031, antihemophilic factor (recombinant) Fc fusion protein
Administered as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| On-Demand | Experimental | 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. Receives: Drug: rFVIIIFc |
| Prophylaxis | Experimental | Tailored prophylaxis, Weekly prophylaxis or Personalized prophylaxis available. Receives: Drug: rFVIIIFc |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Any Positive Inhibitor Development
An inhibitor test result greater than or equal to (>=) 0.6 Bethesda units per milliliter (BU/mL), identified and confirmed by re-testing of a second sample obtained within 2 to 4 weeks, was considered positive. Both tests were to be performed using the Nijmegen-modified Bethesda Assay by the central laboratory. Data was summarized by treatment regimen for participants from 997HA301/997HA307/997HA309 combined and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from 8HA02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Measured: Approximately 5 years
Secondary outcomes (6)
Annualized Bleeding Rate (ABR)
ABR is annualized number of bleeding episodes per participant per year. Bleeding episodes were classified as spontaneous if participant records bleeding event when there is no known contributing factor such as definite trauma/antecedent strenuous activity and as traumatic if participant records bleeding event when there is known reason for bleed. ABR=(Number of bleeding episodes during efficacy period (EP)/number of days during EP)*365.25. EP reflects sum of all intervals of time during which participants were treated with rFVIIIFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. ABR was summarized by treatment regimen for participants from studies 997HA301/997HA307/997HA309 combined and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from Study 8HA02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Measured: Approximately 5 years
Annualized Spontaneous Joint Bleeding Episodes
Bleeding episodes were classified as spontaneous if participant records a bleeding event when there is no known contributing factor such as definite trauma/antecedent strenuous activity. In addition, location of bleed (joint, internal, skin/mucosa or muscle) were collected. Annualized spontaneous joint bleeding episodes=(Number of spontaneous joint bleeding episodes during efficacy period (EP)/number of days during EP)*365.25. EP reflects sum of all intervals of time during which participants were treated with rFVIIIFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. Bleeding episodes were summarized by treatment regimen for participants from studies 997HA301/997HA307/997HA309 combined and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from Study 8HA02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Measured: Approximately 5 years
Total Number of Exposure Days (EDs)
An exposure day is a 24-hour period in which one or more rFVIIIFc injections are given. The total number of days of exposure to rFVIIIFc were summarized by treatment regimen for participants from studies 997HA301/997HA307/997HA309 combined and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from Study 8HA02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Measured: Approximately 5 years
Annualized rFVIIIFc Consumption (International Units Per Kilogram [IU/kg])
Annualized consumption = (total international unit per kilogram [IU/kg] of study treatment received during the efficacy period / total number of days during the efficacy period) multiplied by 365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFVIIIFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. Annualized consumption was summarized by treatment regimen for participants from studies 997HA301/997HA307/997HA309 combined and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from Study 8HA02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Measured: Approximately 5 years
Physicians' Global Assessment of Participant's Response to rFVIIIFc Regimen Using a 4-Point Scale
Participants were assessed for response to their rFVIIIFc regimen using following 4-point scale: 1=Excellent:bleeding episodes responded to less than or equal to (<=)usual number of injections/dose of rFVIIIFc or rate of breakthrough bleeding during prophylaxis was <= that usually observed; 2=Effective: most bleeding episodes responded to same number of injections and dose, but some required more injections or higher doses, or there was minor increase in rate of breakthrough; 3=Partially Effective: bleeding episodes most often required more injections and/or higher doses than expected or adequate breakthrough bleeding prevention during prophylaxis required more frequent injections and/or higher doses and 4=Ineffective: routine failure to control hemostasis/hemostatic control require additional agents. Total number of scale responses =total count of scale responses for all participants; multiple responses per participant including those at scheduled and unscheduled visits are counted.
Measured: Approximately 5 years
Participant's Assessment of Response (Excellent or Good Response) to rFVIIIFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
Using eDiary, participant received rating for treatment response to any bleeding episode (BE) using 4-point scale- 1=Excellent: Abrupt pain relief and/or improvement in signs of bleeding within approximately (approx.) 8 hours (h) after initial injection (inj.); 2=Good: Definite pain relief and/or improvement in signs of bleeding within approx. 8h after an injection, but possibly requiring more than 1 injection after 24-48h for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8h after initial injection and requires more than 1 injection and 4=None: No improvement, or condition worsens within approx. 8h after initial injection. This assessment was to be made approx. 8 to 12h from time the injection was given to treat BE and prior to any additional doses of rFVIIIFc given for same bleeding episode. Percentages are based on the number of bleeding episodes for which a response (excellent or good) was provided for the first injection during the efficacy period.
Measured: Approximately 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2011 (estimated)
- Study startDec 2011
- Primary completionOct 2017
- Study completionOct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 240 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioverativ Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Bioverativ Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01454739
- Sponsor's protocol ID
- 8HA01EXT
- Other IDs
- 2011-003072-37
- Acronym
- ASPIRE
- Status last verified
- Nov 2018
- First submitted
- 29 Sep 2011
- Last updated
- 19 Dec 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Raheja P, Kragh N, Bystricka L, Eriksson D, Aroui K, Mezghani M, Barbier S, Linari S. Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A: post hoc analysis of phase III trials using patient-reported outcomes. Ther Adv Hematol. 2024 Jul 30;15:20406207241257917. doi: 10.1177/20406207241257917. eCollection 2024.
PubMed 39091324Nolan 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.
PubMed 32227570Nolan 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.
PubMed 26218032
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT01454739
- Acronym / sponsor ID
- ASPIRE / 8HA01EXT
- Conditions
- Hemophilia A
- Keywords
- rFVIIIFc, A-LONG Extension
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 240 participants (actual)
- Lead sponsor
- Bioverativ Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- Dec 2011
- Primary completion
- Oct 2017 (actual)
- Study completion
- Oct 2017 (actual)
- First posted
- 19 Oct 2011 (estimated)
- Results
- Posted 23 Nov 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Participant will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the participant in the preceding studies A-LONG - 997HA301 (NCT01181128), pediatric study 8HA02PED (NCT01458106), 997HA307 (NCT02083965) and 997HA309 (NCT02502149).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| On-Demand | Experimental | Drug: rFVIIIFcThe 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. |
| Prophylaxis | Experimental | Drug: rFVIIIFcTailored prophylaxis, Weekly prophylaxis or Personalized prophylaxis available. |
Who may qualify
- Ages
- 0 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- 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 exclusion criteria (2 listed)
- Confirmed positive high-titer inhibitor (≥5.00 BU/mL).
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rFVIIIFcAlso: Eloctate, recombinant coagulation factor VIII Fc fusion protein, BIIB031, antihemophilic factor (recombinant) Fc fusion proteinAdministered as specified in the treatment arm. | Drug | On-Demand, Prophylaxis |
Primary outcome
Number of Participants With Any Positive Inhibitor Development
Time frame: Approximately 5 years
An inhibitor test result greater than or equal to (>=) 0.6 Bethesda units per milliliter (BU/mL), identified and confirmed by re-testing of a second sample obtained within 2 to 4 weeks, was considered positive. Both tests were to be performed using the Nijmegen-modified Bethesda Assay by the central laboratory. Data was summarized by treatment regimen for participants from 997HA301/997HA307/997HA309 combined and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from 8HA02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
6 secondary outcomes
Annualized Bleeding Rate (ABR)
Time frame: Approximately 5 years
Annualized Spontaneous Joint Bleeding Episodes
Time frame: Approximately 5 years
Total Number of Exposure Days (EDs)
Time frame: Approximately 5 years
Annualized rFVIIIFc Consumption (International Units Per Kilogram [IU/kg])
Time frame: Approximately 5 years
Physicians' Global Assessment of Participant's Response to rFVIIIFc Regimen Using a 4-Point Scale
Time frame: Approximately 5 years
Participant's Assessment of Response (Excellent or Good Response) to rFVIIIFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
Time frame: Approximately 5 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39091324Raheja P, Kragh N, Bystricka L, Eriksson D, Aroui K, Mezghani M, Barbier S, Linari S. Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A: post hoc analysis of phase III trials using patient-reported outcomes. Ther Adv Hematol. 2024 Jul 30;15:20406207241257917. doi: 10.1177/20406207241257917. eCollection 2024.
- Derived from this study: PubMed 32227570Nolan 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.
- Derived from this study: PubMed 26218032Nolan 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Bioverativ Therapeutics Inc.
78 sites in 22 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing rFVIIIFc in people with Hemophilia A, run by Bioverativ Therapeutics Inc. at 78 sites in 22 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 78 sites in United States, United Kingdom, Australia, Japan, India and 17 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Nov 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Any Positive Inhibitor Development (time frame: Approximately 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01454739, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Dec 2020.