Hemophilia APhase 2/3NCT00157040
Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A
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 12 sites in 3 countries.
Trial summary
The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts. Part 1 of the study is a pharmacokinetic evaluation, and Part 2 is an evaluation of efficacy and safety. Read more about this study
- Who is it for?
- Ages Up to 6 years
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Antihemophilic factor, recombinant, manufactured protein-free
- How many people
- 50 participants
- Where
- 12 sitesin 3 countries
- Study dates
- 7 Jun 2002to 4 Jan 2005
- 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 Up to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Subject is less than 6 years of age
- Severe or moderately-severe hemophilia A as defined by a baseline factor VIII level <= 2%, documented at screening or on the basis of historical data (e.g., at hemophilia diagnosis)
- Documented medical history of at least 50 exposure days for treatment with all other factor VIII products
- Subject's parent or legally authorized representative has provided informed consent
Who cannot join
- Detectable inhibitor to factor VIII measured in the screening sample by the local or central hemostasis laboratory
- History of inhibitor to factor VIII at any time prior to screening
- Subject has any one of the following laboratory abnormalities at the time of screening:
- platelet count < 100,000/mm3
- hemoglobin concentration < 10 g/dL (100 g/L)
- serum creatinine > 1.5 times the ULN for age
Show all 11Show fewer
- total bilirubin > 2 times the ULN for age
- Subject has an inherited or acquired hemostatic defect other than hemophilia A (e.g., platelet dysfunction secondary to uremia, liver failure, von Willebrand's Disease)
- Subject has known hypersensitivity to RECOMBINATE rAHF
- Subject is currently participating in another investigational drug study or has participated in any clinical study involving an investigational drug within 30 days of study entry
- Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures
Trial locations and contacts
12 sites in 3 countries.
12 sites in 3 countries
United States10 sites
University of Michigan Hemophilia Treatment Center
Ann Arbor, Michigan, 48109
Emory University, Department of Pediatrics
Atlanta, Georgia, 30322
Children´s Memorial Hospital
Chicago, Illinois, 60614
Children´s Hospital of Michigan
Detroit, Michigan, 48201
University of Texas Health Science Center
Houston, Texas, 77030
Indiana Hemophilia and 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
Los Angeles, California, 90027
Comprehensive Bleeding Disorders Center
Peoria, Illinois, 61614
Children´s Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Canada1 site
Hospital for Sick Children, Division of Hematology/Oncology
Toronto, Ontario, M5G 1X8
About this study
Official title: Recombinant Antihemophilic Factor (rAHF-PFM) Manufactured and Formulated Without Added Human or Animal Proteins: Evaluation of Pharmacokinetics, Immunogenicity, Efficacy and Safety in Previously Treated Pediatric Patients With Hemophilia A
The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts. Part 1 of the study is a pharmacokinetic evaluation, and Part 2 is an evaluation of efficacy and safety.
- 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.
The terminal phase half-life of Recombinant Antihemophilic Factor (rAHF-PFM)
Terminal phase half-life of rAHF-PFM was to be determined using the biphasic linear regression model.
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 start7 Jun 2002
- Primary completion4 Jan 2005
- Study completion4 Jan 2005
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
- 50 participants
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
- NCT00157040
- Sponsor's protocol ID
- 060101
- Status last verified
- Apr 2021
- First submitted
- 8 Sep 2005
- Last updated
- 27 Apr 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 27 Apr 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 Pharmacokinetics, Immunogenicity, Efficacy and Safety in Previously Treated Pediatric Patients With Hemophilia A
- ClinicalTrials.gov ID
- NCT00157040
- Acronym / sponsor ID
- 060101
- Conditions
- Hemophilia A
- Keywords
- Factor VIII Deficiency
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 7 Jun 2002 (actual)
- Primary completion
- 4 Jan 2005 (actual)
- Study completion
- 4 Jan 2005 (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 effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts. Part 1 of the study is a pharmacokinetic evaluation, and Part 2 is an evaluation of efficacy and safety.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Subject is less than 6 years of age
- Severe or moderately-severe hemophilia A as defined by a baseline factor VIII level <= 2%, documented at screening or on the basis of historical data (e.g., at hemophilia diagnosis)
- Documented medical history of at least 50 exposure days for treatment with all other factor VIII products
- Subject's parent or legally authorized representative has provided informed consent
Key exclusion criteria (11 listed)
- Detectable inhibitor to factor VIII measured in the screening sample by the local or central hemostasis laboratory
- History of inhibitor to factor VIII at any time prior to screening
- Subject has any one of the following laboratory abnormalities at the time of screening:
- Subject has an inherited or acquired hemostatic defect other than hemophilia A (e.g., platelet dysfunction secondary to uremia, liver failure, von Willebrand's Disease)
- Subject has known hypersensitivity to RECOMBINATE rAHF
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
The terminal phase half-life of 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
Terminal phase half-life of rAHF-PFM was to be determined using the biphasic linear regression model.
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 27 Apr 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
12 sites in 3 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 12 sites in 3 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 4 inclusion and 11 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 27 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Canada, Puerto Rico. 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?
The terminal phase half-life of 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 NCT00157040, 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 27 Apr 2021.