Clinical trial NCT00759655: Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Hemophilia A
Keywords: hemophilia A
Xyntha
ReFacto
moroctocog alfa
bleeding disorder
Lead sponsor: Wyeth is now a wholly owned subsidiary of Pfizer
Sponsor class: INDUSTRY
First posted: 2008-09-25
Enrollment: 1
Sex: MALE
Minimum age (years): 0
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
timeFrame: Baseline to 24 months or early withdrawal.
measure: Percentage of Participants With Less Than Expected Therapeutic Effects (LETE) in the On-Demand Setting
timeFrame: Baseline to 24 months or early withdrawal.
measure: Percentage of Participants With LETE in the Prophylaxis Setting
timeFrame: Baseline to 24 months or early withdrawal.
measure: Percentage of Participants With Low Recovery LETE
timeFrame: Baseline to 24 months or early withdrawal.
Secondary outcomes:
measure: Mean Annualized Bleed Rate (ABR)
timeFrame: Baseline to 24 months or early withdrawal.
measure: Number of Xyntha Infusions Needed to Treat Each New Bleed
timeFrame: Baseline to 24 months or early withdrawal.
measure: Response to First On-demand Xyntha Treatment for All New Bleeds as Assessed by the Caregiver
timeFrame: Baseline to 24 months or early withdrawal
measure: Mean Number of Breakthrough (Spontaneous/Non-traumatic) Bleeds
timeFrame: Baseline to 24 months or early withdrawal.
Other outcomes:
Locations:
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: