Hemophilia APhase 2/3NCT00189982
Efficacy and Safety Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients in Canada With Hemophilia A - A Continuation of Baxter Study 060101
A Phase 2/3 study testing Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM) in people with Hemophilia A, run by Baxalta now part of Shire at 1 site in Canada.
Trial summary
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of children with hemophilia A. The study is open to pediatric patients in Canada who completed Baxter Study 060101. Read more about this study
- Who is it for?
- Ages Up to 6 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- How many people
- 4 participants
- Where
- 1 siteCanada
- Study dates
- 17 Dec 2004to 10 Nov 2006
- 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
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Subject must have participated and completed participation in Baxter's clinical study 060101
- Subject or parent/legally authorized representative has provided written informed consent
Who cannot join
- Subjects who have withdrawn from Baxter's Clinical Study 060101 prior to the termination of the study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Recombinant Antihemophilic Factor Manufactured and Formulated Without Added Human or Animal Proteins (ADVATE rAHF-PFM): Safety Monitoring in Pediatric Patients Diagnosed With Severe to Moderately Severe Hemophilia A - A Continuation of Baxter Clinical Study 060101
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of children with hemophilia A. The study is open to pediatric patients in Canada who completed Baxter Study 060101.
- Conditions
- Hemophilia A
- Being tested
- Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- 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 Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)Drug
Treatment regimens were determined by the investigator, and may have been any combination of standard prophylaxis (25 to 50 IU/kg body weight, 3 to 4 times per week), investigator-determined prophylaxis, and/or on-demand treatment (dose selected by investigator). Once the treatment regimen was established, the regimen was to be maintained for the duration of the study, unless clinical reasons necessitated a change.
The treatment of bleeding episodes and perioperative management was at the discretion of the investigator and consistent with the institution's standard of care.
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of safety, as measured by the incidence, causality, and severity of adverse experiences
Measured: Throughout the study period of approximately 23 months.
Secondary outcomes (2)
Assessment of the hemostatic efficacy in the treatment of bleeding episodes;
Measured: At least 50 exposure days or a total treatment time of 6 months, whichever came first.
assessment of the hemostatic efficacy in surgical or invasive procedures
Measured: From day of surgery until time of discharge from hospital or clinic (up to approximately 2 weeks post surgery).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2005 (estimated)
- Study start17 Dec 2004
- Primary completion10 Nov 2006
- Study completion10 Nov 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 4 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
- NCT00189982
- Sponsor's protocol ID
- 060401
- Status last verified
- May 2021
- First submitted
- 8 Sep 2005
- Last updated
- 5 May 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Recombinant Antihemophilic Factor Manufactured and Formulated Without Added Human or Animal Proteins (ADVATE rAHF-PFM): Safety Monitoring in Pediatric Patients Diagnosed With Severe to Moderately Severe Hemophilia A - A Continuation of Baxter Clinical Study 060101
- ClinicalTrials.gov ID
- NCT00189982
- Acronym / sponsor ID
- 060401
- Conditions
- Hemophilia A
- Keywords
- Factor VIII Deficiency
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 17 Dec 2004 (actual)
- Primary completion
- 10 Nov 2006 (actual)
- Study completion
- 10 Nov 2006 (actual)
- First posted
- 19 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 children with hemophilia A. The study is open to pediatric patients in Canada who completed Baxter Study 060101.
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
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Subject must have participated and completed participation in Baxter's clinical study 060101
- Subject or parent/legally authorized representative has provided written informed consent
Key exclusion criteria (1 listed)
- Subjects who have withdrawn from Baxter's Clinical Study 060101 prior to the termination of the study
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 Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)Treatment regimens were determined by the investigator, and may have been any combination of standard prophylaxis (25 to 50 IU/kg body weight, 3 to 4 times per week), investigator-determined prophylaxis, and/or on-demand treatment (dose selected by investigator). Once the treatment regimen was established, the regimen was to be maintained for the duration of the study, unless clinical reasons necessitated a change. The treatment of bleeding episodes and perioperative management was at the discretion of the investigator and consistent with the institution's standard of care. | Drug | – |
Primary outcome
Assessment of safety, as measured by the incidence, causality, and severity of adverse experiences
Time frame: Throughout the study period of approximately 23 months.
2 secondary outcomes
Assessment of the hemostatic efficacy in the treatment of bleeding episodes;
Time frame: At least 50 exposure days or a total treatment time of 6 months, whichever came first.
assessment of the hemostatic efficacy in surgical or invasive procedures
Time frame: From day of surgery until time of discharge from hospital or clinic (up to approximately 2 weeks post surgery).
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 5 May 2021.
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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
1 site 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 Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM) in people with Hemophilia A, run by Baxalta now part of Shire at 1 site in Canada.
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 1 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 5 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. 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?
Assessment of safety, as measured by the incidence, causality, and severity of adverse experiences (time frame: Throughout the study period of approximately 23 months.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 May 2021.