Phase 4
N=23
Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT00950170 ↗Enrolled (actual)
23
Serious AEs
47.8%
Results posted
Jul 2019
Primary outcome: Primary: Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study — 21.74 Percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Laboratory Tests (Procedure)
- Age
- Pediatric
- Sex
- Male
- Sponsor
- Pfizer
- Primary completion
- Nov 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study |
21.74 | — |
| SECONDARY Annualized Bleeding Rate (ABR) |
5.88 | — |
| SECONDARY Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF |
51; 7; 58; 48; 19; 67 | — |
| SECONDARY Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion |
48; 3; 0; 0; 51; 43 | — |
| SECONDARY Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF |
12 | — |
| SECONDARY Consumption of Total International Units of Factor VIII |
4186; 1857; 55543; 1634; 57799 | — |
| SECONDARY Consumption of Total International Units of Factor VIII Per Year |
72336 | — |
| SECONDARY Mean Dose (IU) of Study Drug Consumed Per Infusion |
552; 544; 628; 481; 619 | — |
| SECONDARY Consumption of Total International Units of Factor VIII by Weight |
388; 93; 4766; 187; 4966 | — |
| SECONDARY Consumption of Total International Units of Factor VIII Per Year by Weight |
6398 | — |
| SECONDARY Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight |
49; 42; 55; 50; 53 | — |
| SECONDARY Mean of Total Number of Infusions of Study Drug Received |
9; 5; 80; 2; 86 | — |
| SECONDARY Mean of Total Number of Days Participants Exposed to Study Drug |
8; 3; 76; 2; 81 | — |
| SECONDARY Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study |
15 | — |
| SECONDARY Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting |
— | — |
| SECONDARY Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting |
0.11 | — |
| SECONDARY Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting |
10 | — |
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.
Eligibility Criteria
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).
Data sourced from ClinicalTrials.gov (NCT00950170). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.