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Trial record 1 of 1 for:    NCT00950170
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Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting

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ClinicalTrials.gov Identifier: NCT00950170
Recruitment Status : Completed
First Posted : July 31, 2009
Results First Posted : July 19, 2019
Last Update Posted : July 19, 2019
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date  ICMJE July 29, 2009
First Posted Date  ICMJE July 31, 2009
Results First Submitted Date  ICMJE May 23, 2017
Results First Posted Date  ICMJE July 19, 2019
Last Update Posted Date July 19, 2019
Actual Study Start Date  ICMJE February 10, 2010
Actual Primary Completion Date November 24, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 10, 2019)
Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study [ Time Frame: 2 years ]
Percentage of participants who developed clinically significant FVIII inhibitors: those persistent over a defined period with clinically impactful effects like breakthrough bleed, low recovery, etc., during the course of the study.
Original Primary Outcome Measures  ICMJE
 (submitted: July 30, 2009)
The primary safety outcome measure will be the proportion of subjects who develop clinically significant FVIII inhibitors during the course of the study. [ Time Frame: 5 years (study duration) ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 10, 2019)
  • Annualized Bleeding Rate (ABR) [ Time Frame: 2 years ]
    Annualized bleeding rate was calculated as the number of bleeds divided by the treatment interval duration (enrollment visit to final visit) and then multiplied by 365.25. If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
  • Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF [ Time Frame: 2 years ]
    Number of infusions of Refacto AF required to treat a new bleed were classified on basis of the response to at 4-point response scale of assessment (excellent, good, moderate and no response). Assessment was completed each time a participant experienced a new bleed requiring an 'on-demand' IV infusion. Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen. Bleeds for which response not recorded, reported as: Data Not Recorded.
  • Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion [ Time Frame: Within 48 hours after infusion, up to 2 years treatment duration ]
    Number of infusions of Refacto AF required for resolution of a bleeding episodes were classified on basis of the response at 4-point response scale of assessment (excellent, good, moderate and no response). Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen. Bleeds for which response not recorded, reported as: Data Not Recorded.
  • Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF [ Time Frame: 2 years ]
    The number of breakthrough bleeds (spontaneous or traumatic) within 48 hours following a prophylaxis dose of ReFacto AF are summarized. If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
  • Consumption of Total International Units of Factor VIII [ Time Frame: 2 years ]
  • Consumption of Total International Units of Factor VIII Per Year [ Time Frame: 2 years ]
    Consumption of total international units of Factor VIII per year was calculated for a participant: dividing the total consumption of factor VIII by participant's treatment interval duration (in days), then multiplying by 365.25.
  • Mean Dose (IU) of Study Drug Consumed Per Infusion [ Time Frame: 2 years ]
    Mean dose for each participant was calculated as participant's total factor VIII consumption (in IU) divided by the number of infusions administered.
  • Consumption of Total International Units of Factor VIII by Weight [ Time Frame: 2 years ]
    Consumption of total international units of Factor VIII by weight was calculated for a participant: dividing the total consumption of factor VIII by participant's weight (the most recently recorded).
  • Consumption of Total International Units of Factor VIII Per Year by Weight [ Time Frame: 2 years ]
    Consumption of total international units of Factor VIII per year by weight was calculated for a participant: the total consumption of factor VIII divided by participant's treatment interval duration (in days), then multiplying by 365.25 and then dividing by participant's weight (the most recently recorded).
  • Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight [ Time Frame: 2 years ]
    Mean dose for each participant was calculated as participant's total factor consumption (in IU) divided by the number of infusions administered and then dividing by participant's weight (the most recently recorded).
  • Mean of Total Number of Infusions of Study Drug Received [ Time Frame: 2 years ]
  • Mean of Total Number of Days Participants Exposed to Study Drug [ Time Frame: 2 years ]
  • Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study [ Time Frame: 2 years ]
    The number of participants who met the dose escalation criteria were prescribed a higher dose and/or were prescribed more frequent doses. When dose escalation was required, the specific dose and dosing schedule was at the investigator's discretion.
  • Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting [ Time Frame: 2 years ]
    LETE occurs in OD setting if participant recorded 2 successive "No Response" (no improvement at all between infusions, or condition worsens) ratings after 2 successive infusions of study drug. Infusions must have been given within 24 hours (hr) of each other for treatment of same bleeding event in absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate dose for type and/or severity of bleed in opinion of investigator, delay of greater than (>) 4 hr between onset of bleed to infusion, delay of >24 hr before administration of a follow-up infusion, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for bleed in opinion of investigator, ongoing trauma responsible for continued bleeding.
  • Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting [ Time Frame: 2 years ]
    LETE in prophylaxis setting if there was a spontaneous bleed within 48 hours after a regularly scheduled prophylactic dose of study drug (which was not used to treat a bleed) in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate prophylactic dose [a dose less than that prescribed in participant's regimen], known lack of adherence to the prescribed prophylaxis regimen, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for the bleed in the opinion of the investigator, traumatic injury responsible for bleeding.
  • Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting [ Time Frame: 2 years ]
    LETE was lower than expected recovery of FVIII in the opinion of the investigator following infusion of study drug in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known compromised study drug, faulty administration of study drug including inadequate dosing).
Original Secondary Outcome Measures  ICMJE
 (submitted: July 30, 2009)
Efficacy endpoints include annualized bleeding rates, responses to the first on-demand treatment with Refacto AF for all new bleeds, the number of ReFacto AF infusions to treat each new bleed, and additional efficacy measurements. [ Time Frame: 5 years (study duration) ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
Official Title  ICMJE AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS
Brief Summary Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia. Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study. The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.
Detailed Description Regulatory Commitment
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE Hemophilia A
Intervention  ICMJE Procedure: Laboratory Tests
Blood draws to determine the level of factor VIII activity before and after dosing at appropriate visits, and blood draws to determine levels of factor VIII inhibitor (antibody to factor VIII).
Other Name: ReFacto AF
Study Arms  ICMJE Experimental: 1
The investigator treats subjects with ReFacto AF in the usual care setting.
Intervention: Procedure: Laboratory Tests
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 28, 2016)
23
Original Estimated Enrollment  ICMJE
 (submitted: July 30, 2009)
50
Actual Study Completion Date  ICMJE November 24, 2016
Actual Primary Completion Date November 24, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male subjects <6 years of age with severe hemophilia A (FVIII:C <1%) based on clinical records, including newborns.
  • No prior exposure to factor products or any blood products.

Exclusion Criteria:

  • Presence of any bleeding disorder in addition to hemophilia A.
  • Treatment with any investigational agent or device within the past 30 days.
  • Any condition(s) that compromises the ability to collect study-related observations, or that poses a contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; and expectation of poor adherence to study requirements).
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE up to 6 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Germany,   Italy,   Netherlands,   Spain,   Turkey,   Ukraine
Removed Location Countries Austria,   Georgia,   Greece,   Poland,   Romania,   Sweden
 
Administrative Information
NCT Number  ICMJE NCT00950170
Other Study ID Numbers  ICMJE 3082B2-4434
B1831006 ( Other Identifier: Alias Study Number )
2008-008436-93 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Pfizer CT.gov Call Center Pfizer
PRS Account Pfizer
Verification Date May 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP