Clinical trial: A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors (NCT04158648)

Eligibility criteria:
Inclusion Criteria:

          -  Diagnosis of mild (FVIII level between >5% and <40%) or moderate (FVIII level between
             ≥1% and ≤5%) congenital Hemophilia A without FVIII inhibitors

          -  Weight ≥3 kilograms (kg)

          -  Need for prophylaxis based on investigator assessment

          -  A negative test for inhibitor (i.e., <0.6 Bethesda Units per milliliter [BU/mL])
             within 8 weeks prior to enrollment

          -  No documented inhibitor (i.e., <0.6 BU/mL), FVIII half-life <6 hours, or FVIII
             recovery <66% in the last 5 years

          -  Documentation of the details of prophylactic or episodic FVIII treatment and of number
             of bleeding episodes for at least the last 24 weeks prior to enrollment

          -  Adequate hematologic hepatic and renal function

          -  For women of childbearing potential: agreement to remain abstinent or use
             contraception (as defined in the protocol) during the treatment period and for at
             least 24 weeks after the final dose of study drug

        Exclusion Criteria:

          -  Inherited or acquired bleeding disorder other than mild or moderate congenital
             hemophilia A

          -  History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the
             investigator's judgment

          -  Previous (within the last 12 months) or current treatment for thromboembolic disease
             or signs of thromboembolic disease

          -  Other conditions that may currently increase the risk of bleeding or thrombosis

          -  History of clinically significant hypersensitivity associated with monoclonal antibody
             therapies or components of the emicizumab injection

          -  Planned surgery during the emicizumab loading dose phase (surgeries in participants on
             emicizumab from Week 5 onwards are allowed)

          -  Known HIV infection with CD4 counts <200 cells per microlitre (/μL)

          -  Concomitant disease, condition, significant abnormality on screening evaluation or
             laboratory tests, or treatment that could interfere with the conduct of the study, or
             that would in the opinion of the investigator, pose an additional unacceptable risk in
             administering study drug to the participant

          -  Receipt of any of the following: An investigational drug to treat or reduce the risk
             of hemophilic bleeds within 5 half-lives of last drug administration with the
             exception of prior emicizumab prophylaxis; A non-hemophilia-related investigational
             drug within last 30 days or 5 half-lives, whichever is shorter; or Any other
             investigational drug currently being administered or planned to be administered

          -  Inability to comply with the study protocol in the opinion of the investigator

          -  Pregnant or breastfeeding, or intending to become pregnant during the study (women of
             childbearing potential must have a negative serum pregnancy test result within 7 days
             prior to initiation of study drug)
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.



Patient description (medical language): A 20-year-old man comes to the emergency due to bleeding after a tooth extraction.  The bleeding has persisted for approximately 30 minutes despite constant direct pressure.  He is a known case of Hemophilia type A treated with FVIII. Blood pressure is 95/60 mm Hg and pulse is 105/min. His weight is 70 Kg. Family history is positive for Hemophilia type A in his maternal uncle. He also has a lipoma on his left arm which he plans to remove surgically. His FVIII activity is 40%.

Patient description (patient's own words): I got my tooth extracted and I could not stop bleeding for 30 min. I'm a 20 yo dude but I have Hemophilia type A treated with FVIII. My blood pressure was 95/60 and my pulse 105/min. I'm 70 kg. My uncle on my mom's side also has type A hemophilia. I also have a fatty swelling on my left arm and I was supposed to remove it. My FVIII activity was 40%.

Does the patient match the trial criteria? 1