Phase 3
Completed N=57
Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
Source: ClinicalTrials.gov NCT02954575 ↗Enrolled (actual)
57
Serious AEs
0.0%
Results posted
Dec 2019
Primary outcomePrimary: Total Annualized Bleeding Rate (TABR) — 2.10 No. of BEs / year (ABR) — p=<0.0001
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Total Annualized Bleeding Rate (TABR) |
2.10 | <0.0001 sig |
| SECONDARY Spontaneous Annualized Bleeding Rate (SABR) |
1.51 | <0.0001 sig |
| SECONDARY Efficacy of Wilate in the Treatment of Breakthrough BEs |
16; 32; 9 | 0.0096 sig |
| SECONDARY Wilate Consumption Data (Average Total Normdose of FVIII IU/kg Per Month of Study) for Prophylaxis |
405.06 | — |
| SECONDARY Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Norm) of FVIII:C |
28.61; 30.75 | — |
| SECONDARY Pharmacokinetic (PK) Assessment (in Vivo Half-Life (t1/2)) of FVIII:C |
10.82; 11.50 | — |
| SECONDARY Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration [Cmax]) of FVIII:C |
106.70; 103.49 | — |
| SECONDARY Incremental in Vivo Recovery (IVR) of Wilate Over Time |
2.14; 2.14; 1.97 | — |
| SECONDARY Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay) |
15.867 | 0.0346 sig |
| SECONDARY Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate |
5.104 | 0.4244 |
| SECONDARY Safety and Tolerability of Wilate by Monitoring Adverse Events (AEs) Throughout the Study |
17; 16 | — |
| SECONDARY Immunogenicity of Wilate by Testing for FVIII Inhibitors |
— | — |
| SECONDARY Virus Safety Measured by the Number of Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study |
— | — |
Eligibility Criteria
Inclusion Criteria
- Severe hemophilia A ( 200/µL)
- Good documentation of the historical bleeding rate (at least for the 6 months preceding study start)
- Voluntarily given, fully informed written and signed consent obtained by the patient (or parent/legal guardian in case of adolescents) before any study-related procedures are conducted
Whenever possible, the interval between the Screening Visit and the PK or Non-PK Visit should not exceed 30 days. If the 30-day interval is exceeded, determination of the CD4+ count is to be repeated and must be >200/µL for patients to be enrolled (i.e., exclusion criterion no. 4).
Exclusion Criteria
- Any coagulation disorders other than hemophilia A
- History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory anti-bodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory
- Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine>120 µmol/L)
- Patients receiving or scheduled to receive immunomodulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs
- Treatment with any investigational medicinal product in another interventional clinical study currently or within 4 weeks before enrollment
Data sourced from ClinicalTrials.gov (NCT02954575). 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.