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

Brief Title

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

Official Title

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

Brief 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

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC)) of FVIII:C

Secondary Outcome

 Total Annualized Bleeding Rate (TABR)

Condition

Severe Hemophilia A

Intervention

Wilate

Study Arms / Comparison Groups

 All patients

Description:  All patients will receive Wilate for prophylactic treatment. Patients will also receive Wilate for treatment of breakthrough bleeding events as required

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

November 22, 2017

Completion Date

November 3, 2018

Primary Completion Date

November 3, 2018

Eligibility Criteria

        Inclusion Criteria:

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

          2. Male patients aged 1 to <12 years

          3. Previous treatment with a FVIII concentrate for at least 50 exposure days (EDs)

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

          5. 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:

          1. Any coagulation disorders other than haemophilia A

          2. 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

          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
             antiretroviral chemotherapy), such as alpha-interferon, prednisone (equivalent to >10
             mg/day), or similar drugs

Gender

Male

Ages

1 Year - 11 Years

Accepts Healthy Volunteers

No

Contacts

Cristina Solomon, MD, ,

Location Countries

Russian Federation

Location Countries

Russian Federation

Administrative Informations

NCT ID

NCT03376516

Organization ID

WIL-30

Responsible Party

Sponsor

Study Sponsor

Octapharma

Study Sponsor

Cristina Solomon, MD, Study Director, Octapharma

Verification Date

December 2019