Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age

NCT00759655 | Terminated Phase 3 Results

• 3 other identifiers: 3082B2-3315B18310023082B2-3315-WW
• Study Type: interventional
• Target: 1
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Hemophilia A

Keywords

hemophilia A; Xyntha; ReFacto; moroctocog alfa; bleeding disorder

Outcome Measures

Primary Outcomes (4)

• 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.

  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.

  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.

  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.

  Baseline to 24 months or early withdrawal.

Secondary Outcomes (4)

• Mean Annualized Bleed Rate (ABR)

  Baseline to 24 months or early withdrawal.

• Number of Xyntha Infusions Needed to Treat Each New Bleed

  Baseline to 24 months or early withdrawal.

• Response to First On-demand Xyntha Treatment for All New Bleeds as Assessed by the Caregiver

  Baseline to 24 months or early withdrawal

• Mean Number of Breakthrough (Spontaneous/Non-traumatic) Bleeds

  Baseline to 24 months or early withdrawal.

Study Arms (1)

open label

OTHER

Biological: Moroctocog alfa

Interventions

Moroctocog alfa BIOLOGICAL

Patients will receive Moroctocog alfa according to their investigator's prescription.

open label

Eligibility Criteria

• Age: 0 Years - 5 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Wyeth is now a wholly owned subsidiary of Pfizer: lead

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hemophilia A; Hemostatic Disorders

Interventions

recombinant factor VIII SQ

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Vascular Diseases; Cardiovascular Diseases

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2008
• First Posted: September 25, 2008
• Study Start: June 1, 2009
• Primary Completion: December 1, 2009
• Study Completion: December 1, 2009
• Last Updated: June 15, 2022
• Results First Posted: January 4, 2011

Record last verified: 2022-05