Hemophilia APhase 2/3NCT00157105
Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery
A Phase 2/3 study testing Antihemophilic factor, recombinant, manufactured protein-free in people with Hemophilia A, run by Baxalta now part of Shire at 10 sites in United States.
Trial summary
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A patients undergoing surgery. Read more about this study
- Who is it for?
- Ages 5 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Antihemophilic factor, recombinant, manufactured protein-free
- How many people
- 59 participants
- Where
- 10 sitesUnited States
- Study dates
- 12 Feb 2001to 3 Aug 2004
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
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 5 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age >= 5 years
- The subject has severe or moderately severe hemophilia A defined by a baseline factor VIII level <= 2% of normal documented at screening or historically (e.g., at hemophilia diagnosis)
- Subjects may enroll regardless of their serologic status for human immunodeficiency virus (HIV-1) and hepatitis C virus (HCV)
- The subject requires a surgical, dental or other invasive procedure--either elective or emergency
- The subject has a history of at least 150 exposure days for all other factor VIII products (as estimated by the study site investigator) prior to study entry
- The subject has a life expectancy of at least 28 days from the day of surgery
- The subject has been informed of the nature of the study, agreed to its provisions, and signed and dated the informed consent form approved by the appropriate IRB/IEC and Baxter BioScience
Who cannot join
- The subject has a detectable inhibitor to factor VIII in the local hemostasis laboratory at the investigative site at the time of enrollment
- The subject has a history of inhibitor to factor VIII > 1.0 BU. Note: If the subject has a history of an inhibitor titer > 1.0 BU at any time prior to enrollment but demonstrated expected clinical responses to conventional doses of factor VIII therapy, the subject may enroll
- The subject has known hypersensitivity to Recombinate
- The subject is currently participating in another investigational drug study, or has participated in any clinical trial involving an investigational drug within 30 days of study entry
- The subject has clinical and/or laboratory evidence of abnormal hemostasis from causes other than hemophilia A (e.g., late-stage chronic liver disease, immune thrombocytopenic purpura, disseminated intravascular coagulation)
- The subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Children´s Healthcare of Atlanta Blood Bank
Atlanta, Georgia, 30322
Brigham and Women´s Hospital, Hematology Division
Boston, Massachusetts, 02115
Children´s Hospital Medical Center Pharmacy, Hemophilia Treatment Center
Cincinnati, Ohio, 45229
Michigan State University
East Lansing, Michigan, 48824
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, 46260
University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics
Iowa City, Iowa, 52242
Children´s Hospital Los Angeles, Hemophilia Comprehensive Care Center, Division of Pediatric Hematology/Oncology
Los Angeles, California, 90027
Mt. Sinai Medical School, Hemophilia Comprehensive Care Center
New York, New York, 10029
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213-4306
Puget Sound Blood Center
Seattle, Washington, 98104-1256
About this study
Official title: Recombinant Antihemophilic Factor (rAHF PFM) Manufactured and Formulated Without Added Human or Animal Proteins: Evaluation of Safety and Efficacy in Hemophilia A Patients Undergoing Surgical or Invasive Procedures
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A patients undergoing surgery.
- Conditions
- Hemophilia A
- Being tested
- Antihemophilic factor, recombinant, manufactured protein-free
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Antihemophilic factor, recombinant, manufactured protein-freeDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent of participants experiencing treatment-related adverse experiences (AEs)
Measured: Throughout the study period of approximately 3.5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2005 (estimated)
- Study start12 Feb 2001
- Primary completion3 Aug 2004
- Study completion3 Aug 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)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 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00157105
- Sponsor's protocol ID
- 069902
- Status last verified
- Aug 2021
- First submitted
- 8 Sep 2005
- Last updated
- 24 Aug 2021
- Results posted
- No
Publications
Shapiro A, Gruppo R, Pabinger I, Collins PW, Hay CR, Schroth P, Casey K, Patrone L, Ehrlich H, Ewenstein BM. Integrated analysis of safety and efficacy of a plasma- and albumin-free recombinant factor VIII (rAHF-PFM) from six clinical studies in patients with hemophilia A. Expert Opin Biol Ther. 2009 Mar;9(3):273-83. doi: 10.1517/14712590902729392.
PubMed 19216617
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Recombinant Antihemophilic Factor (rAHF PFM) Manufactured and Formulated Without Added Human or Animal Proteins: Evaluation of Safety and Efficacy in Hemophilia A Patients Undergoing Surgical or Invasive Procedures
- ClinicalTrials.gov ID
- NCT00157105
- Acronym / sponsor ID
- 069902
- Conditions
- Hemophilia A
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 59 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 12 Feb 2001 (actual)
- Primary completion
- 3 Aug 2004 (actual)
- Study completion
- 3 Aug 2004 (actual)
- First posted
- 12 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A patients undergoing surgery.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 5 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Age >= 5 years
- The subject has severe or moderately severe hemophilia A defined by a baseline factor VIII level <= 2% of normal documented at screening or historically (e.g., at hemophilia diagnosis)
- Subjects may enroll regardless of their serologic status for human immunodeficiency virus (HIV-1) and hepatitis C virus (HCV)
- The subject requires a surgical, dental or other invasive procedure--either elective or emergency
- The subject has a history of at least 150 exposure days for all other factor VIII products (as estimated by the study site investigator) prior to study entry
Key exclusion criteria (6 listed)
- The subject has a detectable inhibitor to factor VIII in the local hemostasis laboratory at the investigative site at the time of enrollment
- The subject has a history of inhibitor to factor VIII > 1.0 BU. Note: If the subject has a history of an inhibitor titer > 1.0 BU at any time prior to enrollment but demonstrated expected clinical responses to conventional doses of factor VIII therapy, the subject may enroll
- The subject has known hypersensitivity to Recombinate
- The subject is currently participating in another investigational drug study, or has participated in any clinical trial involving an investigational drug within 30 days of study entry
- The subject has clinical and/or laboratory evidence of abnormal hemostasis from causes other than hemophilia A (e.g., late-stage chronic liver disease, immune thrombocytopenic purpura, disseminated intravascular coagulation)
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 |
|---|---|---|
| Antihemophilic factor, recombinant, manufactured protein-free | Drug | – |
Primary outcome
Percent of participants experiencing treatment-related adverse experiences (AEs)
Time frame: Throughout the study period of approximately 3.5 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2021.
- Derived from this study: PubMed 19216617Shapiro A, Gruppo R, Pabinger I, Collins PW, Hay CR, Schroth P, Casey K, Patrone L, Ehrlich H, Ewenstein BM. Integrated analysis of safety and efficacy of a plasma- and albumin-free recombinant factor VIII (rAHF-PFM) from six clinical studies in patients with hemophilia A. Expert Opin Biol Ther. 2009 Mar;9(3):273-83. doi: 10.1517/14712590902729392.
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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.
Study Director study director
Takeda
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Antihemophilic factor, recombinant, manufactured protein-free in people with Hemophilia A, run by Baxalta now part of Shire at 10 sites in United States.
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 7 inclusion and 6 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 24 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Percent of participants experiencing treatment-related adverse experiences (AEs) (time frame: Throughout the study period of approximately 3.5 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 NCT00157105, which also lists 1 linked publication.
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 24 Aug 2021.