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A Global, Open-Label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
This is an open-label drug study of SerpinPC (a subcutaneously administered drug) for patients with severe hemophilia A or moderately severe to severe Hemophilia B. The study consists of 3 parts after an observation phase of 12-24 weeks: 24 week justification phase, 24 week confirmatory phase & 24 week extension phase.
This is a subcutaneous drug treatment clinical trial with an observation period of up to 24 weeks, pre-dosing/dosing period of 8 visits on site or phone calls & an extension period of 6 visits.
$75.00 travel reimbursement per visit
Subjects include prophylactic dosing or on-demand dosing for factor replacement
Adequate laboratory functions
Previous deep vein thrombosis, pulmonary embolism, MI or stroke.
Uncontrolled hypertension, active cancer (except basal cell)
Weight > 150kg (330 lbs.) or BMI > 40kg/m2
A Phase 3, single-arm, open-label, multicenter study of thesafety and efficacy of dirloctocogene samoparvovec(SPK-8011, adeno-associated viral vector with B-domaindeleted human factor VIII gene) in adults with severe ormoderately severe hemophilia A (KEYSTONESM 1)
This is a Phase 3 study of single-dose SPK-8011 AAV gene therapy in combination with immunomodulation. Patients will receive a one-time dose of SPK 8011, three weeks of methylprednisolone & then will be followed for safety & efficacy per study schedule.
1) Screening period of < 8wks.-1 visit2) Lead-in for >24 wks.-1 visit3) Pre-dose day -24-1 visit4) Dosing-Day 1 & Day 2-2 visits5) Primary study (15 mos.)-37 visits6) Long term follow-up approx. 9 yrs.-20 visitsSome visits may be done via home health when a physical isn't required.
$75.00 travel reimbursement per visit
>150 documented exposure days to a FVIII protein product
No evidence of cirrhosis or advanced liver disease
Negative test for inhibitor against FVIII during screening
No documented FVIII inhibitor in 5 yrs. prior to screening
Currently undergoing antiviral therapy
Have an inherited bleeding or acquired disorder other than hemophilia A
Prior treatment with a vector or gene transfer agent
Major surgical procedure planned in a 15 month period following SPK 8011 infusion