Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A (ASPIRE)

December 16, 2020 updated by: Bioverativ Therapeutics Inc.

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
240

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Camperdown, New South Wales, Australia, 2050
        • Research Site
    • Queensland
      • South Brisbane, Queensland, Australia, 4101
        • Research Site
    • South Australia
      • Adelaide, South Australia, Australia, 5000
        • Research Site
    • Victoria
      • Melbourne, Victoria, Australia, 3052
        • Research Site
      • Melbourne, Victoria, Australia, 3181
        • Research Site
    • Western Australia
      • Murdoch, Western Australia, Australia, 6150
        • Research Site
      • Subiaco, Western Australia, Australia, 6008
        • Research Site
  • Austria
      • Vienna, Austria, 1090
        • Research Site
  • Belgium
    • Brussels
      • Bruxelles, Brussels, Belgium, 1200
        • Research Site
  • Brazil
    • Sao Paulo
      • Campinas, Sao Paulo, Brazil, 13083-878
        • Research Site
  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V6Z 1Y6
        • Research Site
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Research Site
  • France
    • Rhone
      • Bron cedex, Rhone, France, 69677
        • Research Site
  • Germany
      • Berlin, Germany, 10249
        • Research Site
    • Nordrhein Westfalen
      • Bonn, Nordrhein Westfalen, Germany, 53127
        • Research Site
  • Hong Kong
      • Hong Kong, Hong Kong
        • Research Site
    • New Territories
      • Hong Kong, New Territories, Hong Kong
        • Children Cancer Centre
      • Hong Kong, New Territories, Hong Kong
        • Sir Yue Kong Pao Center for Cancer
  • India
    • Delhi
      • New Delhi, Delhi, India, 110002
        • Research Site
    • Karnataka
      • Bangalore, Karnataka, India, 560034
        • Research Site
    • Maharashtra
      • Pune, Maharashtra, India, 411004
        • Research Site
    • Punjab
      • Ludhiana, Punjab, India, 141008
        • Research Site
    • Tamilnadu
      • Vellore, Tamilnadu, India, 632004
        • Research Site
  • Ireland
      • Dublin, Ireland, D12 N512
        • Research Site
  • Israel
      • Ramat Gan, Israel, 52621
        • Research Site
  • Italy
      • Firenze, Italy, 50134
        • Research Site
      • Milano, Italy, 20122
        • Research Site
      • Vicenza, Italy, 36100
        • Research Site
  • Japan
    • Aichi-Ken
      • Nagoya-shi, Aichi-Ken, Japan, 466-8560
        • Research Site
    • Fukuoka-Ken
      • Kitakyushu, Fukuoka-Ken, Japan, 807-8556
        • Research Site
    • Kanagawa-Ken
      • Kawasaki, Kanagawa-Ken, Japan, 216-8511
        • Research Site
    • Nara-Ken
      • Kashihara-shi, Nara-Ken, Japan, 634-8522
        • Research Site
    • Tokyo-To
      • Shinjuku-ku, Tokyo-To, Japan, 160-0023
        • Research Site
      • Tokyo, Tokyo-To, Japan, 167-8515
        • Research Site
  • Netherlands
      • Groningen, Netherlands, 9713 GZ
        • Research Site
  • New Zealand
      • Auckland, New Zealand, 1023
        • Research Site
      • Christchurch, New Zealand, 8011
        • Research Site
      • Hamilton, New Zealand, 3200
        • Research Site
      • Palmerston North, New Zealand, 4410
        • Research Site
      • Wellington, New Zealand, 6021
        • Research Site
  • Poland
      • Lublin, Poland, 20-093
        • Research Site
  • South Africa
    • Gauteng
      • Johannesburg, Gauteng, South Africa, 2193
        • Research Site
    • Western Cape
      • Cape Town, Western Cape, South Africa, 7925
        • Research Site
  • Spain
      • Barcelona, Spain, 8035
        • Research Site
      • Madrid, Spain, 28046
        • Research Site
  • Sweden
      • Göteborg, Sweden, 41345
        • Research Site
  • Switzerland
      • Zuerich, Switzerland, 8091
        • Research Site
  • United Kingdom
      • London, United Kingdom, SE1 7EH
        • Research Site
    • Cambridgeshire
      • Cambridge, Cambridgeshire, United Kingdom, CB2 0QQ
        • Research Site
    • Greater London
      • London, Greater London, United Kingdom, E1 1BB
        • Research Site
      • London, Greater London, United Kingdom, SE1 7EH
        • Research Site
      • London, Greater London, United Kingdom, WC1N 3JH
        • Research Site
    • Hampshire
      • Basingstoke, Hampshire, United Kingdom, RG24 9NA
        • Research Site
    • London
      • Hamstead, London, United Kingdom, NW3 2QG
        • Research Site
    • Strathclyde
      • Glasgow, Strathclyde, United Kingdom, G3 8SJ
        • Research Site
      • Glasgow, Strathclyde, United Kingdom, G4 0SF
        • Research Site
  • United States
    • California
      • Los Angeles, California, United States, 90007
        • Research Site
      • Los Angeles, California, United States, 90027
        • Research Site
      • Orange, California, United States, 92868
        • Research Site
      • Sacramento, California, United States, 95817
        • Research Site
      • San Diego, California, United States, 92123
        • Research Site
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Research Site
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Research Site
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Research Site
    • Louisiana
      • New Orleans, Louisiana, United States, 70112
        • Research Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Research Site
    • Michigan
      • East Lansing, Michigan, United States, 48823
        • Research Site
    • Missouri
      • Saint Louis, Missouri, United States, 63104
        • Research Site
    • Nevada
      • Las Vegas, Nevada, United States, 89109
        • Research Site
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • Research Site
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Research Site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Research Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Research Site
      • Philadelphia, Pennsylvania, United States, 19104
        • Research Site
      • Pittsburgh, Pennsylvania, United States, 15213
        • Research Site
    • Texas
      • Houston, Texas, United States, 77030
        • Research Site
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • Research Site
    • Washington
      • Seattle, Washington, United States, 98104
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 second and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Key Inclusion Criteria:

  • 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:

  • Confirmed positive high-titer inhibitor (≥5.00 BU/mL).

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.
Drug: rFVIIIFc
Administered as specified in the treatment arm.
Other Names:
  • Eloctate
  • recombinant coagulation factor VIII Fc fusion protein
  • BIIB031
  • antihemophilic factor (recombinant) Fc fusion protein
  • efmoroctocog alfa
Experimental: Prophylaxis
Tailored prophylaxis, Weekly prophylaxis or Personalized prophylaxis available.
Drug: rFVIIIFc
Administered as specified in the treatment arm.
Other Names:
  • Eloctate
  • recombinant coagulation factor VIII Fc fusion protein
  • BIIB031
  • antihemophilic factor (recombinant) Fc fusion protein
  • efmoroctocog alfa

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
Approximately 5 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Annualized Bleeding Rate (ABR)
Time Frame: Approximately 5 years
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.
Approximately 5 years
Annualized Spontaneous Joint Bleeding Episodes
Time Frame: Approximately 5 years
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.
Approximately 5 years
Total Number of Exposure Days (EDs)
Time Frame: Approximately 5 years
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.
Approximately 5 years
Annualized rFVIIIFc Consumption (International Units Per Kilogram [IU/kg])
Time Frame: Approximately 5 years
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.
Approximately 5 years
Physicians' Global Assessment of Participant's Response to rFVIIIFc Regimen Using a 4-Point Scale
Time Frame: Approximately 5 years
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.
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
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.
Approximately 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Bioverativ Therapeutics Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, Bioverativ Therapeutics Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 1, 2011

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 1, 2017

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 1, 2017

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 29, 2011

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 17, 2011

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 19, 2011

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 19, 2020

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 16, 2020

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 8HA01EXT
  • 2011-003072-37

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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