Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients
Condition: Hemophilia A
MeSH terms: Hemophilia A
Intervention: Moroctocog-alfa (AF-CC)
Intervention description: Moroctocog-alfa (AF-CC) is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).
Intervention MeSH terms: Factor VIII
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 50.0
Sex: Male
Minimum age: 12 Years
Maximum age: 65 Years
Healthy volunteers: No

Summary:

      Moroctocog-alfa (AF-CC) is indicated for the control and prevention of hemorrhagic episodes
      and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor
      VIII deficiency or classic hemophilia). The current single country, multi-centric, open
      label, non-randomized pragmatic clinical trial is a post-approval study to fulfill the
      Central Drugs Standard Control Organization (CDSCO) request for supplementary information
      relating to the use of moroctocog-alfa (AF-CC) in Indian subjects with hemophilia A.

      The primary objective of study is to study the safety of moroctocog alfa (AF-CC) when
      administered for prophylaxis with respect to incidence of FVIII inhibitor development. The
      secondary objectives are to evaluate the incidence of adverse events (AEs) and serious
      adverse events (SAEs) in subjects receiving moroctocog alfa (AF-CC) prophylaxis, to evaluate
      the efficacy of moroctocog alfa (AF-CC) during a prophylaxis regimen, to evaluate the total
      annualized consumption of moroctocog alfa (AF-CC) by subjects following a prophylaxis
      regimen, to evaluate the efficacy of moroctocog alfa (AF-CC) for the treatment of
      breakthrough bleeding episodes (on-demand treatment) while following a prophylaxis regimen.

      Fifty male subjects aged >/= 12 years to ≤65 years with moderate or severe hemophilia A will
      be enrolled in the study. The subjects will be selected based on protocol specified
      eligibility criteria. The overall treatment duration for each subject will be up to 8 weeks,
      with up to a 4-week screening period and a subsequent post-treatment 28-day safety
      observation period. Subjects are requested to continue in the study until 24 exposure days
      (EDs) or a period of up to 8 weeks on moroctocog alfa (AF-CC) treatment had occurred
      (whichever occurs first). Efficacy and safety assessments will be performed as specified in
      the protocol.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Male subjects ≥12 years to ≤65 years with a diagnosis of congenital moderate or severe
             hemophilia A (FVIII:C ≤5%).

          2. Documented history of at least 50 exposure days (EDs) to FVIII-containing products.

          3. Evidence of a personally signed and dated informed consent document indicating that
             the subject (or a legally acceptable representative, parent(s)/legal guardian) has
             been informed of all pertinent aspects of the study. For minors under the age of legal
             consent in India, assent of the participating child needs to be documented for the age
             range 12 to 18 in addition to the parental informed consent.

        Exclusion Criteria:

          1. Prior history of inhibitor to FVIII or positive inhibitor testing (≥0.6 BU/mL) during
             Screening. Clinical signs or symptoms of decreased response to FVIII.

          2. Known hypersensitivity to the active substance or any of the excipients.

          3. Known allergic reaction to hamster proteins.

          4. Presence of any bleeding disorder in addition to hemophilia A.

          5. Participation in other studies involving investigational drug(s) (Phases 1-4) within
             30 days before the current study begins and/or during study participation.

          6. Planned surgery within 6 months from the start of the study.

          7. Unsuitable to participate in study for any other reason as assessed by the
             investigator; including any disorder, except for conditions associated with hemophilia
             A, which in the investigator's opinion might jeopardize subject's safety or compliance
             with the protocol.

          8. Subjects (or a legally acceptable representative) is not able to understand study
             documents and study procedure.

          9. Immunocompromised subjects due to human immunodeficiency virus (HIV) infection
             (defined as viral load above or equal to 100,000 copies/mL; and for HIV+ subjects:
             cluster of differentiation 4 positive (CD4+) lymphocyte count below or equal to
             200/μL). HIV status and CD4+ lymphocyte count results may be obtained at screening or
             from available medical records; results must be not older than 6 months prior to
             screening.

         10. Subjects who are investigational site staff members directly involved in the conduct
             of the study and their family members, site staff members otherwise supervised by the
             Investigator, subjects who have been previously enrolled into the study, or subjects
             who are Pfizer employees directly involved in the conduct of the study.

         11. Planned use of any non-study medication for treatment of hemophilia (eg, other factor
             replacement agents, bypassing agents, or non-factor treatments [such as anti-tissue
             factor pathway inhibitors]).

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