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

NCT02954575 | Completed Phase 3 Results FDA Drug

• 1 other identifier: WIL-27
• Study Type: interventional
• Target: 57
• Locations: 5 countries
• Sites: 7

Brief 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.

Conditions

Severe Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Total Annualized Bleeding Rate (TABR)

  The total number of bleeding events (BEs) was documented by patients in a patient diary (together with the investigator in case of on-site treatments), which was reviewed at each follow-up visit by site personnel.

  6 months

Secondary Outcomes (12)

• Spontaneous Annualized Bleeding Rate (SABR)

  6 months

• Efficacy of Wilate in the Treatment of Breakthrough BEs

  6 months

• Wilate Consumption Data (Average Total Normdose of FVIII IU/kg Per Month of Study) for Prophylaxis

  6 months

• Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Norm) of FVIII:C

  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

• Pharmacokinetic (PK) Assessment (in Vivo Half-Life (t1/2)) of FVIII:C

  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

Other Outcomes (1)

• Efficacy of Wilate in Surgical Prophylaxis

  6 months

Study Arms (1)

All patients

EXPERIMENTAL

All patients will receive Wilate for prophylactic treatment

Drug: Wilate

Interventions

Wilate DRUG

Also known as: von Willebrand factor / Factor VIII (plasma derived)

All patients

Eligibility Criteria

• Age: 12 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Severe hemophilia A (\<1% FVIII:C) according to medical history
• Male patients aged ≥12 years
• Previous treatment with a FVIII concentrate for at least 150 exposure days (EDs)
• Immunocompetence (CD4+ count \>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

You may not qualify if:

• 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

Sponsors & Collaborators

• Octapharma: lead

Study Sites (7)

Specialized Hospital for Active Treatment "Joan Pavel"

Sofia, Bulgaria

Location

National Haemophilia Centre

Budapest, Hungary

Location

Krakowskie Centrum Medyczne

Krakow, Poland

Location

Korczowski Bartosz Gabinet Lekarski

Rzeszów, Poland

Location

Centrul Medical Unirea -Policlinica Enescu

Bucharest, Romania

Location

Barnaul Branch of RAMS hematology center

Barnaul, Russia

Location

Federal Scientific Hematology Center

Moscow, Russia

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

von Willebrand Factor; Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Biological Factors; Protein Precursors

Results Point of Contact

• Title: Cristina Solomon
• Organization: Octapharma AG

Cristina.Solomon@octapharma.com

+41 (0)55 451 21 89

Study Officials

• Cristina Solomon, MD

  Octapharma

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 27, 2016
• First Posted: November 3, 2016
• Study Start: December 1, 2016
• Primary Completion: March 29, 2018
• Study Completion: March 29, 2018
• Last Updated: January 19, 2021
• Results First Posted: December 3, 2019

Record last verified: 2020-12

Data Sharing

• IPD Sharing: Will not share

Locations

RO (1); BU (1); RU (2); HU (1); PL (2)