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

Brief Title

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

Official Title

Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Patients With Severe Hemophilia A

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.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Total Annualized Bleeding Rate (TABR)

Secondary Outcome

 Spontaneous Annualized Bleeding Rate (SABR)

Condition

Severe Hemophilia A

Intervention

Wilate

Study Arms / Comparison Groups

 All patients

Description:  All patients will receive Wilate for prophylactic treatment

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

Start Date

December 2016

Completion Date

March 29, 2018

Primary Completion Date

March 29, 2018

Eligibility 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

Gender

Male

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Cristina Solomon, MD, ,

Location Countries

Bulgaria

Location Countries

Bulgaria

Administrative Informations

NCT ID

NCT02954575

Organization ID

WIL-27

Responsible Party

Sponsor

Study Sponsor

Octapharma

Study Sponsor

Cristina Solomon, MD, Study Director, Octapharma

Verification Date

December 2020