Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02954575
• Date of registration: 27/10/2016
• Prospective Registration: Yes
• Primary sponsor: Octapharma
• Public title: Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
• Scientific title: Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Patients With Severe Hemophilia A
• Date of first enrolment: December 2016
• Target sample size: 57
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02954575
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Bulgaria, Hungary, Poland, Romania, Russian Federation, Serbia, Ukraine

Contacts

• Name: Cristina Solomon, MD
• Affiliation: Octapharma

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Severe hemophilia A (<1% FVIII:C) according to medical history

2. Male patients aged =12 years

3. Previous treatment with a FVIII concentrate for at least 150 exposure days (EDs)

4. Immunocompetence (CD4+ count >200/µL)

5. Good documentation of the historical bleeding rate (at least for the 6 months
preceding study start)

6. 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:

1. Any coagulation disorders other than hemophilia A

2. 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

3. Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate
transaminase [ASAT] levels >5 times of upper limit of normal, creatinine>120 µmol/L)

4. 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

5. Treatment with any investigational medicinal product in another interventional
clinical study currently or within 4 weeks before enrollment



Age minimum: 12 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Severe Hemophilia A

Intervention(s)

Drug: Wilate

Primary Outcome(s)

Total Annualized Bleeding Rate (TABR) [Time Frame: 6 months]

Secondary Outcome(s)

Incremental in Vivo Recovery (IVR) of Wilate Over Time [Time Frame: Baseline, 3 and 6 months]

Pharmacokinetic (PK) Assessment (in Vivo Half-Life (t1/2)) of FVIII:C [Time Frame: Initial PK assessment (Day -1) and PK study completion visit (6 months); data collected 1 h prior to infusion and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection]

Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration [Cmax]) of FVIII:C [Time Frame: Initial PK assessment (Day -1) and 6 months]

Virus Safety Measured by the Number of Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study [Time Frame: 6 months]

Wilate Consumption Data (Average Total Normdose of FVIII IU/kg Per Month of Study) for Prophylaxis [Time Frame: 6 months]

Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate [Time Frame: 6 months]

Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Norm) of FVIII:C [Time Frame: Initial PK visit (Day -1) and PK study completion visit (6 months); data collected 1 h prior to injection and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection]

Efficacy of Wilate in the Treatment of Breakthrough BEs [Time Frame: 6 months]

Spontaneous Annualized Bleeding Rate (SABR) [Time Frame: 6 months]

Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay) [Time Frame: 6 months]

Immunogenicity of Wilate by Testing for FVIII Inhibitors [Time Frame: 6 months]

Safety and Tolerability of Wilate by Monitoring Adverse Events (AEs) Throughout the Study [Time Frame: 6 months]

Secondary ID(s)

WIL-27

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 03/12/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02954575