Clinical trial: Gene Therapy Study in Severe Haemophilia A Patients (270-201) (NCT02576795)

Eligibility criteria:
Inclusion Criteria:

          1. Males 18 years or older with established severe Haemophilia A (endogenous FVIII level
             ≤1 IU/dL) as evidenced by their medical history.

          2. Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure
             days (EDs)

          3. Greater than or equal to 12 bleeding episodes for patients on on-demand FVIII
             replacement therapy over the previous 12 months. Does not apply to patients on
             prophylaxis

          4. No history of inhibitor, and results from a modified Nijmegen Bethesda assay of less
             than 0.6 Bethesda Units (BU) 2 consecutive occasions at least one week apart within
             the past 12 months

          5. Sexually active patients must be willing to use an acceptable method of contraception.

        Exclusion Criteria:

          1. Detectable pre-existing immunity to the AAV5 capsid as measured by AAV5 transduction
             inhibition or AAV5 total antibodies

          2. Any evidence of immunosuppressive disorder or active chronic infection including
             hepatis B, hepatitis C, HIV

          3. Significant liver dysfunction as defined by abnormal elevation ofliver function tests,
             or for patients who have undergone liver imaging or biopsy and found to have evidence
             of grade 3 or higher fibrosis

          4. Evidence of any bleeding disorder not related to haemophilia A

          5. 12. Treatment with any investigational product within 30 days prior to the end of the
             screening period, or any previous exposure to any gene transfer therapy

          6. Any disease or condition that per the physician's discretion would prevent the patient
             from fully complying with the requirements of the study including possible
             corticosteroid treatment outlined in the protocol. The physician may exclude patients
             unwilling or unable to agree on not using alcohol for the 16-week period following the
             viral infusion.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.


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? Contradiction