Phase 3 N=11 Treatment

Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A

Severe Hemophilia A

Bottom Line

View on ClinicalTrials.gov: NCT03376516 ↗

Enrolled (actual)

Serious AEs

10.0%

Results posted

Dec 2019

Primary outcome: Primary: Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC)) of FVIII:C — 768.8; 671.9; 720.3 h*IU/dL

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Wilate (Drug)
Age: Pediatric · 1+ yrs
Sex: Male
Sponsor: Octapharma
Primary completion: Nov 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC)) of FVIII:C	768.8; 671.9; 720.3	—
PRIMARY Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Normalised (AUCNorm)) of FVIII:C for Wilate	15.38; 13.44; 14.41	—
PRIMARY Pharmacokinetic (PK) Assessment (Half-life (h)) of FVIII:C	8.28; 9.35; 8.82	—
PRIMARY Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration) for FVIII:C	83.0; 78.94; 80.99	—
PRIMARY Pharmacokinetic (PK) Assessment (Time to Reach Maximum Plasma Concentration (Tmax)) of FVIII:C	0.25; 0.25; 0.25	—
PRIMARY Pharmacokinetic (PK) Assessment (Mean Residence Time (MRT)) of FVIII:C	11.47; 12.56; 12.01	—
PRIMARY Pharmacokinetic (PK) Assessment (Volume of Distribution (Vd)) of FVIII:C	0.784; 1.081; 0.933	—
PRIMARY Pharmacokinetic (PK) Assessment (Clearance) of FVIII:C	0.071; 0.098; 0.084	—
PRIMARY Incremental In Vivo Recovery (IVR) of FVIII:C	1.65; 1.57; 1.61	—
SECONDARY Total Annualized Bleeding Rate (TABR)	6.47; 10.62; 8.54	—
SECONDARY Spontaneous Annualized Bleeding Rate (SABR)	2.70; 1.20; 1.95	—
SECONDARY Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs)	7; 9; 16; 9; 8; 17	—
SECONDARY Wilate Consumption Data: Average Dose of Wilate Per Week of Study	58.52; 68.08; 63.30	—
SECONDARY Incremental in Vivo Recovery (IVR) of Wilate Over Time	1.65; 1.57; 1.61; 1.55; 1.56; 1.56	—
SECONDARY Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay)	0.185; 6.100; 0.922	0.9593
SECONDARY Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate	0.121; 0.006; 0.003	0.8536
SECONDARY Safety and Tolerability of Wilate by Monitoring The Number of Adverse Events (AEs) Throughout the Study	6	—
SECONDARY Immunogenicity of Wilate: Number of Participants With FVIII Inhibitor Activity at 6 Months	—	—
SECONDARY Virus Safety Measured by the Number With Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study	1	—

Summary

A prospective, non-controlled, international, multi-centre phase 3 study to investigate the pharmacokinetics, efficacy, safety, and immunogenicity of Wilate in previously treated children with severe haemophilia A

Eligibility Criteria

Inclusion Criteria

Severe haemophilia A ( 200/μL)
Voluntarily given, fully informed written and signed consent obtained by the patient's parent(s) or legal guardian and, depending on the children's developmental stage and intellectual capacity, informed assent by the patients before any study-related procedures are performed

The interval between the Screening Visit and the 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., inclusion criterion no. 4).

Exclusion Criteria

Any coagulation disorders other than haemophilia A
History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory antibodies (≥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 antiretroviral chemotherapy), such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03376516). 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.