Hemophilia APhase 3NCT00759655
Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age
A Phase 3 study testing Moroctocog alfa in people with Hemophilia A, run by Wyeth is now a wholly owned subsidiary of Pfizer.
Trial summary
This study will be investigating the safety and efficacy of Xyntha (moroctocog alfa (AF-CC)) in male patients less than 6 years old. Annualized bleeding rates and physician / caregiver assessments of responses to treatment will be characterized. FVIII inhibitor levels will be assessed throughout the study. Read more about this study
- Who is it for?
- Ages 0 to 5 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Moroctocog alfa
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- No sites listed yet
- Study dates
- Jun 2009to Dec 2009
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 0 to 5 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male patients less than 6 years of age with moderately severe to severe hemophilia A (FVIII less than or equal to 2%).
- Treatment history of less than 50 exposure days to prior recombinant or plasma-derived FVIII replacement products.
- Not receiving treatment for HIV or hepatitis infection, or the patient is on a stable antiviral regimen at the time of enrollment in the study.
Who cannot join
- Presence of any bleeding disorder in addition to hemophilia A.
- Inhibitor titer of greater than or equal to 5 Bethesda Units (BU) at screening.
- Treated with immunomodulatory therapy during the screening period
- Treatment history of more than 5 exposure days (ED) to Xyntha.
- Known hypersensitivity to hamster protein.
About this study
Official title: An Open-Label Study To Evaluate The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age In Usual Care Settings
This study will be investigating the safety and efficacy of Xyntha (moroctocog alfa (AF-CC)) in male patients less than 6 years old. Annualized bleeding rates and physician / caregiver assessments of responses to treatment will be characterized. FVIII inhibitor levels will be assessed throughout the study.
Read the detailed description
The study was terminated on 22 Sept 2009 due to competition with another Wyeth study for a similar patient population. The decision to terminate the trial was not based on any safety issues.
- Conditions
- Hemophilia A
- Being tested
- Moroctocog alfa
- Keywords
- hemophilia A, Xyntha, ReFacto, moroctocog alfa, bleeding disorder
- Medical subject headings
- Hemophilia A, Hemostatic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Moroctocog alfaBiological
Patients will receive Moroctocog alfa according to their investigator's prescription.
| Study group | Role | What they receive |
|---|---|---|
| open label | Other | Not described Receives: Biological: Moroctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
Incidence of inhibitor development was defined as any result determined positive at a central laboratory (Bethesda inhibitor titer of >=0.6 BU/mL) using Nijmegen modification of the Bethesda assay.
Measured: Baseline to 24 months or early withdrawal.
Percentage of Participants With Less Than Expected Therapeutic Effects (LETE) in the On-Demand Setting
LETE in the on-demand setting was based on the response to the treatment of a bleeding episode. LETE in the on-demand setting occurred if the participant recorded 2 successive "No Response" ratings (indicated there was no improvement at all between infusions or during the 24 hour interval following an infusion, or condition worsened) after 2 successive Xyntha infusions, respectively. The infusions was to be administered within 24 hours (=<24 hours) of each other for the treatment of the same bleeding event in the absence of confounding factor.
Measured: Baseline to 24 months or early withdrawal.
Percentage of Participants With LETE in the Prophylaxis Setting
The LETE in the prophylaxis setting was the occurrence of a bleed. LETE in the prophylaxis setting occurred if there was a spontaneous bleed within 48 hours (=<48 hours) after a regularly scheduled prophylactic dose of Xyntha (which was not used to treat a bleed) in the absence of confounding factors.
Measured: Baseline to 24 months or early withdrawal.
Percentage of Participants With Low Recovery LETE
The LETE could be considered lower than expected recovery of FVIII in the opinion of the investigator following infusion of Xyntha in the absence of confounding factors.
Measured: Baseline to 24 months or early withdrawal.
Secondary outcomes (4)
Mean Annualized Bleed Rate (ABR)
An annualized bleeding rate (ABR) for each participant was calculated as the number of bleeds requiring administration of FVIII replacement product (taken from the electronic Infusion Log Diary), divided by his total therapy duration (in days), and then multiplied by 365.25.
Measured: Baseline to 24 months or early withdrawal.
Number of Xyntha Infusions Needed to Treat Each New Bleed
The data from the electronic Infusion Log Diary plus the Test Article case report form (CRF) was used to determine the number of infusions administered to treat a bleed. This was calculated by adding the initial 'for a new bleed' (on demand) infusion to any subsequent (on demand) infusions for the (same) 'previously treated bleed'. An on-demand infusion for a 'previously treated bleed' was counted toward the bleed with the most recent start time prior to that infusion.
Measured: Baseline to 24 months or early withdrawal.
Response to First On-demand Xyntha Treatment for All New Bleeds as Assessed by the Caregiver
A 4-point response scale to be completed is as defined as follows: (Excellent: definite pain relief/improvement in signs of bleeding starting within 8 hrs after an infusion, with no additional infusion; Good: definite pain relief/improvement in signs of bleeding starting within 8 hrs or following the infusion; Moderate: probable/slight improvement starting after 8 hours following the infusion; No Response: no improvement at all between infusions).
Measured: Baseline to 24 months or early withdrawal
Mean Number of Breakthrough (Spontaneous/Non-traumatic) Bleeds
The number of breakthrough (spontaneous/non-traumatic) bleeds within 48 hours following a prophylaxis dose of Xyntha was summarized. The data from the electronic Infusion Log Diary plus the Test Article CRF was used to determine the number of infusions administered to treat a new bleed, counting only those infusions administered =<48 hours after an infusion marked as 'prophylaxis' (which had no associated bleed).
Measured: Baseline to 24 months or early withdrawal.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2008 (estimated)
- Study startJun 2009
- Primary completionDec 2009
- Study completionDec 2009
Why it stopped: See termination reason in detailed description.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00759655
- Sponsor's protocol ID
- 3082B2-3315
- Other IDs
- B1831002, 3082B2-3315-WW
- Status last verified
- May 2022
- First submitted
- 23 Sep 2008
- Last updated
- 15 Jun 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study To Evaluate The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age In Usual Care Settings
- ClinicalTrials.gov ID
- NCT00759655
- Acronym / sponsor ID
- 3082B2-3315
- Conditions
- Hemophilia A
- Keywords
- hemophilia A, Xyntha, ReFacto, moroctocog alfa, bleeding disorder
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: See termination reason in detailed description.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Wyeth is now a wholly owned subsidiary of PfizerIndustry (pharma, biotech, devices)
- Study start
- Jun 2009
- Primary completion
- Dec 2009 (actual)
- Study completion
- Dec 2009 (actual)
- First posted
- 25 Sep 2008 (estimated)
- Results
- Posted 4 Jan 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will be investigating the safety and efficacy of Xyntha (moroctocog alfa (AF-CC)) in male patients less than 6 years old. Annualized bleeding rates and physician / caregiver assessments of responses to treatment will be characterized. FVIII inhibitor levels will be assessed throughout the study.
Detailed description
The study was terminated on 22 Sept 2009 due to competition with another Wyeth study for a similar patient population. The decision to terminate the trial was not based on any safety issues.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| open label | Other | Biological: Moroctocog alfa |
Who may qualify
- Ages
- 0 to 5 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Male patients less than 6 years of age with moderately severe to severe hemophilia A (FVIII less than or equal to 2%).
- Treatment history of less than 50 exposure days to prior recombinant or plasma-derived FVIII replacement products.
- Not receiving treatment for HIV or hepatitis infection, or the patient is on a stable antiviral regimen at the time of enrollment in the study.
Key exclusion criteria (5 listed)
- Presence of any bleeding disorder in addition to hemophilia A.
- Inhibitor titer of greater than or equal to 5 Bethesda Units (BU) at screening.
- Treated with immunomodulatory therapy during the screening period
- Treatment history of more than 5 exposure days (ED) to Xyntha.
- Known hypersensitivity to hamster protein.
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 |
|---|---|---|
| Moroctocog alfaPatients will receive Moroctocog alfa according to their investigator's prescription. | Biological | open label |
Primary outcome
Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
Time frame: Baseline to 24 months or early withdrawal.
Incidence of inhibitor development was defined as any result determined positive at a central laboratory (Bethesda inhibitor titer of >=0.6 BU/mL) using Nijmegen modification of the Bethesda assay.
Percentage of Participants With Less Than Expected Therapeutic Effects (LETE) in the On-Demand Setting
Time frame: Baseline to 24 months or early withdrawal.
LETE in the on-demand setting was based on the response to the treatment of a bleeding episode. LETE in the on-demand setting occurred if the participant recorded 2 successive "No Response" ratings (indicated there was no improvement at all between infusions or during the 24 hour interval following an infusion, or condition worsened) after 2 successive Xyntha infusions, respectively. The infusions was to be administered within 24 hours (=<24 hours) of each other for the treatment of the same bleeding event in the absence of confounding factor.
Percentage of Participants With LETE in the Prophylaxis Setting
Time frame: Baseline to 24 months or early withdrawal.
The LETE in the prophylaxis setting was the occurrence of a bleed. LETE in the prophylaxis setting occurred if there was a spontaneous bleed within 48 hours (=<48 hours) after a regularly scheduled prophylactic dose of Xyntha (which was not used to treat a bleed) in the absence of confounding factors.
Percentage of Participants With Low Recovery LETE
Time frame: Baseline to 24 months or early withdrawal.
The LETE could be considered lower than expected recovery of FVIII in the opinion of the investigator following infusion of Xyntha in the absence of confounding factors.
4 secondary outcomes
Mean Annualized Bleed Rate (ABR)
Time frame: Baseline to 24 months or early withdrawal.
Number of Xyntha Infusions Needed to Treat Each New Bleed
Time frame: Baseline to 24 months or early withdrawal.
Response to First On-demand Xyntha Treatment for All New Bleeds as Assessed by the Caregiver
Time frame: Baseline to 24 months or early withdrawal
Mean Number of Breakthrough (Spontaneous/Non-traumatic) Bleeds
Time frame: Baseline to 24 months or early withdrawal.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
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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.
Pfizer CT.gov Call Center study director
Pfizer
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Moroctocog alfa in people with Hemophilia A, run by Wyeth is now a wholly owned subsidiary of Pfizer.
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 5 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: stopped early, as of the sponsor's last update on 15 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
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 phase mean?
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?
Yes. Results are posted in the registry (first posted 4 Jan 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants With Factor VIII (FVIII) Inhibitor Development (time frame: Baseline to 24 months or early withdrawal.). The study lists 4 primary outcomes in all. 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 15 Jun 2022.