Phase 4
Completed N=251
Survey of Inhibitors in Plasma-Product Exposed Toddlers
Source: ClinicalTrials.gov NCT01064284 ↗Enrolled (actual)
251
Serious AEs
12.5%
Results posted
Apr 2017
Primary outcomePrimary: To Assess the Immunogenicity of Plasma Derived VWF/FVIII and rFVIII Concentrates by Determining the Frequency of Inhibitor Development in the First 50 EDs or in the First 3 Years From Enrolment, Whichever Comes First in PUPs and MBCTs — 29; 47 participants
◆ Published Evidence
Highly cited
495citations · ~50 / year
A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A.
Summary
The primary objective of the study is to assess the immunogenicity of VWF/FVIII and of rFVIII concentrates by determining the frequency of inhibitor development in previously untreated patients (PUPs) or minimally blood component-treated (MBCTPs) in the first 50 EDs or in the first 3 years from enrollment, whichever occurs first.
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Linked Publications (2)
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A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A.
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Genetic risk stratification to reduce inhibitor development in the early treatment of hemophilia A: a SIPPET analysis.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY To Assess the Immunogenicity of Plasma Derived VWF/FVIII and rFVIII Concentrates by Determining the Frequency of Inhibitor Development in the First 50 EDs or in the First 3 Years From Enrolment, Whichever Comes First in PUPs and MBCTs |
29; 47 | — |
| SECONDARY To Evaluate the Anamnestic Response of Inhibitor Patients |
— | — |
| SECONDARY To Evaluate the Frequency of Transient Inhibitors |
7; 12 | — |
| SECONDARY To Evaluate the Modality of Occurrence of Inhibitors (Number of EDs) |
8; 8 | — |
| SECONDARY To Evaluate the Modality of Occurrence of Inhibitors (Titre at Onset) |
12; 16.3 | — |
| SECONDARY To Evaluate Clinical Factors Potentially Associated to Inhibitor Development |
— | — |
| SECONDARY To Evaluate Laboratory Factors Potentially Associated to Inhibitor Development |
— | — |
| SECONDARY To Evaluate the Incidence of All Other Adverse Events Related and Not Related to the Products Used |
— | — |
Eligibility Criteria
Inclusion Criteria
- Male subjects
- Any ethnicity
- Age = 1% on testing at the central laboratory will be separately recorded in the screening list.
- Previously untreated (0 EDs to any FVIII concentrates or blood products) or minimally treated ( = 1%, as assayed at the central laboratory
o Those patients originally diagnosed locally as severe but subsequently found to have FVIII levels ranging from 1% to 2% on testing at the central laboratory will be separately recorded in the screening list.
- Concomitant congenital or acquired immunodeficiency
- Concomitant treatment with systemic immunosuppressive drugs
- Concomitant treatment with any investigational drug
Data sourced from ClinicalTrials.gov (NCT01064284) and the linked publication. 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. Informational only — not medical advice.