Hemophilia APhase 2/3NCT00157053
Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901
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 24 sites in 9 countries.
Trial summary
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A subjects. The study consists of 2 parts: Part 1 is a pharmacokinetic and safety study, and Part 2 is an evaluation of efficacy and safety. The study is open to patients who completed Baxter Study 069901. Read more about this study
- Who is it for?
- Ages 10 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
- 82 participants
- Where
- 24 sitesin 9 countries
- Study dates
- 22 Nov 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 10 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject has completed Baxter protocol 069901
- Subject is HIV-1 seronegative or, if HIV-1 seropositive, has a CD4+ lymphocyte count >= 400/mm3 documented within three months of the screening visit
- Subject (and his legally acceptable representative, in the case of study participants >= 10 and < 18 years of age) 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
Who cannot join
- The subject received factor VIII products other than rAHF-PFM upon completion of Baxter protocol 069901
- The subject developed an inhibitor to factor VIII, measured in the central laboratory, during Baxter protocol 069901. An inhibitor is defined as a Bethesda titer > 1.0 or, if Bethesda titer < 1.0, confirmation using the Nijmegen modification of the Bethesda assay with a titer > 0.6
- The subject is scheduled to receive an immunomodulating drug other than anti-retroviral chemotherapy (e.g., a-interferon, steroids) during the course of the study
- The subject is identified by the investigator as being unable or unwilling to cooperate with study procedures
Trial locations and contacts
24 sites in 9 countries.
24 sites in 9 countries
United States11 sites
Childrens Healthcare of Atlanta Blood Bank
Atlanta, Georgia, 30322
Mountain States Regional Hemophilia and Thrombosis Center
Aurora, Colorado, 80045
Children's Hospital Medical Center Pharmacy
Cincinnati, Ohio, 45229
Michigan State University
East Lansing, Michigan, 48824
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, 46260
University of Medicine & Dentistry of New Jersey
New Brunswick, New Jersey, 08903-0019
Cornell Medical Center
New York, New York, 10021
Mt. Sinai Medical School
New York, New York, 10029
Comprehensive Bleeding Disorders Center
Peoria, Illinois, 61614
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213-4306
Puget Sound Blood Center
Seattle, Washington, 98104-1256
United Kingdom4 sites
University Hospital of Wales
Cardiff, Wales, CF4 4XW
The Royal Free Hospital
London, England, NW3 2QG
Central Manchester Healthcare NHS Trust
Manchester, England, M13 9WL
The Churchill Hospital
Oxford, England, OX3 7LJ
Germany3 sites
Klinikum der J. W. Goethe - Universität
Frankfurt am Main, 60590
Medizinische Hochschule Hannover
Hanover, 30623
Klinikum Innenstadt der Universität München
Munich, D-80336
About this study
Official title: Evaluation of Pharmacokinetics, Safety, Efficacy and Immunogenicity of rAHF-PFM in Previously Treated Hemophilia A Patients - A Continuation Study
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A subjects. The study consists of 2 parts: Part 1 is a pharmacokinetic and safety study, and Part 2 is an evaluation of efficacy and safety. The study is open to patients who completed Baxter Study 069901.
- Conditions
- Hemophilia A
- Being tested
- Antihemophilic factor, recombinant, manufactured protein-free
- Keywords
- Factor VIII Deficiency
- 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.
Terminal phase half-life for Recombinant Antihemophilic Factor (rAHF-PFM)
Measured: Within 30 minutes prior to the pharmacokinetic infusion and at 1 hour ± 5 minutes, 9 ± 1 hour, 24 ± 2 hours, and 48 ± 2 hours after the infusion.
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 start22 Nov 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
- 82 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
- NCT00157053
- Sponsor's protocol ID
- 060102
- 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
- Evaluation of Pharmacokinetics, Safety, Efficacy and Immunogenicity of rAHF-PFM in Previously Treated Hemophilia A Patients - A Continuation Study
- ClinicalTrials.gov ID
- NCT00157053
- Acronym / sponsor ID
- 060102
- Conditions
- Hemophilia A
- Keywords
- Factor VIII Deficiency
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 82 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 22 Nov 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 subjects. The study consists of 2 parts: Part 1 is a pharmacokinetic and safety study, and Part 2 is an evaluation of efficacy and safety. The study is open to patients who completed Baxter Study 069901.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 10 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Subject has completed Baxter protocol 069901
- Subject is HIV-1 seronegative or, if HIV-1 seropositive, has a CD4+ lymphocyte count >= 400/mm3 documented within three months of the screening visit
- Subject (and his legally acceptable representative, in the case of study participants >= 10 and < 18 years of age) 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
Key exclusion criteria (4 listed)
- The subject received factor VIII products other than rAHF-PFM upon completion of Baxter protocol 069901
- The subject developed an inhibitor to factor VIII, measured in the central laboratory, during Baxter protocol 069901. An inhibitor is defined as a Bethesda titer > 1.0 or, if Bethesda titer < 1.0, confirmation using the Nijmegen modification of the Bethesda assay with a titer > 0.6
- The subject is scheduled to receive an immunomodulating drug other than anti-retroviral chemotherapy (e.g., a-interferon, steroids) during the course of the study
- The subject is identified by the investigator as being unable or unwilling to cooperate with study procedures
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
Terminal phase half-life for Recombinant Antihemophilic Factor (rAHF-PFM)
Time frame: Within 30 minutes prior to the pharmacokinetic infusion and at 1 hour ± 5 minutes, 9 ± 1 hour, 24 ± 2 hours, and 48 ± 2 hours after the infusion.
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
24 sites in 9 countries: 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 24 sites in 9 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 3 inclusion and 4 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 24 sites in United States, United Kingdom, Germany, Austria, Belgium and 4 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 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?
Terminal phase half-life for Recombinant Antihemophilic Factor (rAHF-PFM) (time frame: Within 30 minutes prior to the pharmacokinetic infusion and at 1 hour ± 5 minutes, 9 ± 1 hour, 24 ± 2 hours, and 48 ± 2 hours after the infusion.). 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 NCT00157053, 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.