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Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease VWD) Patients <6 Years of Age

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ClinicalTrials.gov Identifier: NCT04953884
Recruitment Status : Recruiting
First Posted : July 8, 2021
Last Update Posted : February 21, 2022
Sponsor:
Information provided by (Responsible Party):
Octapharma

Study Description
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Brief Summary:
The WIL-33 study aims to determine the efficacy, pharmacokinetics, immunogenicity and safety of wilate as routine prophylaxis in up to 12 paediatric patients (eight evaluable) with severe von Willebrand Disease VWD (defined as screening von Willebrand factor ristocetin cofactor activity [VWF:RCo] <20%) under the age of 6 years, over a period of 12 months.

Condition or disease Intervention/treatment Phase
Von Willebrand Disease Drug: wilate Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Clinical Study to Investigate the Efficacy, Pharmacokinetics, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease Patients Under the Age of 6 Years
Actual Study Start Date : July 28, 2021
Estimated Primary Completion Date : May 2023
Estimated Study Completion Date : May 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: wilate treatment
PK: Single dose of 80 IU/kg body weight (BW). Prophylactic treatment: 30-50 IU/kg BW administered 2-3 times per week at the recommended dose of over 12 months. Minor haemorrhage: loading dose 30-50 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours to achieve von Willebrand factor activity (VWF:Ac) and FVIII:C trough levels of >30%. Major haemorrhage: loading dose 50-80 IU/kg BW followed by a maintenance dose of 30-50 IU/kg BW every 12-24 hours to achieve VWF:Ac and FVIII:C trough levels of >50%. Minor surgery: loading dose of 40-60 IU/kg BW followed by a maintenance dose of 20-30 IU/kg BW every 12- 24 hours for up to 3 days, to achieve VWF:Ac peak levels of 50% after loading dose and trough levels >30% during maintenance. Major surgery: loading dose of 60-80 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours for up to 6 days or longer, to achieve VWF:Ac peak levels of 100% after loading dose and trough levels >50% during maintenance
 Drug: wilate
wilate is a plasma-derived, stable, highly purified, double virus inactivated concentrate of freeze-dried active VWF and factor VIII (FVIII) prepared from cryoprecipitate and intended for the treatment of patients with von Willebrand disease (VWD) and/or haemophilia A


Outcome Measures
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Primary Outcome Measures :
  1. Total annualised bleeding rate (tABR) during prophylactic treatment with wilate. [ Time Frame: Up to 12 months of treatment ]

Secondary Outcome Measures :
  1. Area under the curve (AUC) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time [ Time Frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate ]
  2. AUC normalised for the administered dose (AUCnorm) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time [ Time Frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate ]
  3. In vivo half-life (T1/2) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time [ Time Frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate ]
  4. Maximum plasma concentration (Cmax) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time [ Time Frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate ]
  5. Time to reach maximum plasma concentration (Tmax) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time [ Time Frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate ]
  6. Mean residence time (MRT) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time [ Time Frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate ]
  7. Volume of distribution (Vd) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time [ Time Frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate ]
  8. Clearance (CL) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time [ Time Frame: At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate ]
  9. Incremental in-vivo recovery (IVR) of wilate for VWF:Ac (VWF:RCo) and FVIII:C (OS) over time [ Time Frame: At baseline and at 1, 2, 3, 6, 9, and 12 months of treatment ]
  10. Efficacy of wilate in the treatment of spontaneous and traumatic breakthrough BEs based on the proportion of spontaneous and traumatic BEs successfully treated with wilate [ Time Frame: Up to 12 months of treatment ]
    Assessed by the use of a 4-point ordinal haemostatic efficacy scale (excellent - good - moderate - none)

  11. The overall efficacy of wilate in perioperative prophylaxis against excessive bleeding as assessed at the end of the postoperative period by the responsible treating investigator [ Time Frame: Up to 12 months of treatment ]
    Assessed by the use of a 4-point ordinal haemostatic efficacy scale (excellent - good - moderate - none)

  12. wilate consumption data for prophylactic treatment, for on-demand treatment and during surgical prophylaxis [ Time Frame: Up to 12 months of treatment ]
  13. Incidence of VWF and FVIII inhibitors [ Time Frame: Up to 12 months of treatment ]
  14. Incidence of thromboembolic events [ Time Frame: Up to 12 months of treatment ]
  15. Joint Health Status determination by the use of the Hemophilia Joint Health Score, given the patient's age and constitutional development allow this assessment [ Time Frame: At baseline and at 12 months of treatment ]
  16. Safety and tolerability of wilate assessed by monitoring adverse events (AEs) throughout the study [ Time Frame: Up to 12 months of treatment ]

Eligibility Criteria
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Ages Eligible for Study:   up to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients aged <6 years at the time of screening
  2. Type 3 (at least four patients), severe type 2 (except 2N) or severe type 1 VWD (VWF:RCo <20%) according to medical history, requiring substitution therapy with a VWF-containing product
  3. Minimum body weight 12.5 kg at the time of screening
  4. Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted (obtained from the patient's parent(s)/ legal guardian(s))

Exclusion Criteria:

  1. History or current suspicion of VWF or FVIII inhibitors
  2. Injection of DDAVP or VWF-containing product within 72 hours prior to inclusion
  3. Medical history of a thromboembolic event
  4. Platelet count <100,000/µL at screening (except for VWD type 2B)
  5. Patients receiving, or scheduled to receive, immunosuppressant drugs (other than antiretroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or similar drugs
  6. Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within four weeks before enrolment
  7. Other coagulation disorders or bleeding disorders
  8. Known hypersensitivity to any of the components of the study drug
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04953884


Contacts
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Contact: Sigurd Knaub, PhD +41 554512141 Sigurd.Knaub@octapharma.com

Locations
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United States, California
Loma Linda University Health Active, not recruiting
San Bernardino, California, United States, 92408
Belarus
Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology Active, not recruiting
Minsk, Belarus
Moldova, Republic of
IMSP Mother and Child Institute Recruiting
Chişinău, Moldova, Republic of
Contact: Valentin Ţurea         
North Macedonia
PHI University Clinic for Child Diseases Recruiting
Skopje, North Macedonia
Contact: Zorica Antevska         
Russian Federation
FSBI National Research Medical Center of Pediatric Hematology, Oncology and Immunology Recruiting
Moscow, Russian Federation
Contact: Pavel Zharkov         
Morozovskaya Children's Hospital Recruiting
Moscow, Russian Federation
Contact: Vladamir Vdovin         
Ukraine
State Institution National Children's Specialized Clinic "OHMATDET" of Ministry of Health of Ukraine, Centre of pathology of hemostasis Active, not recruiting
Kiev, Ukraine
Danylo Halytsky Lviv National Medical University, Communal Institution of Lviv Regional Council "Western Ukrainian Specialized Children's Medical Centre" Recruiting
Lviv, Ukraine
Contact: Leonid Dubey         
Sponsors and Collaborators
Octapharma
More Information
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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT04953884    
Other Study ID Numbers: WIL-33
First Posted: July 8, 2021    Key Record Dates
Last Update Posted: February 21, 2022
Last Verified: February 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Von Willebrand Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn