INdividualized ITI Based on Fviii(ATE) Protection by VWF

• Status: Terminated
• Trial end date: 2019-07-19
• Target enrollment: 0
• Participant gender: Male

Summary

The primary goal of the INITIATE trial is to compare the clinical outcome of individualized lot selection to random lot selection utilizing one plasma-derived von Willebrand factor (VWF)/coagulation factor (FVIII) complex concentrate for immune tolerance induction (ITI) in subjects with congenital Hemophilia A, FVIII activity ≤2%, and a historical high-titer inhibitor [≥5 Bethesda Unit (BU)].

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of California, Davis

Criteria

Inclusion Criteria:

1. Diagnosis of congenital Hemophilia A and baseline FVIII ≤2%.

2. Weight ≥ 5 kg

3. History of FVIII inhibitor titer ≥5 BU

4. Current FVIII inhibitor titer ≥5 BU or ≥0.6 BU and failed ITI defined by FVIII recovery <66% normal and half-life <6 hours

5. Adequate venous access for daily concentrate infusions

6. For participants <18 years, a parent or guardian willing and able to provide informed consent with verbal or written assent from the child if require by the local institution. For participants ≥18 years, a willingness and ability to provide informed consent from the subject.

7. Ability to comply with study related treatments, evaluations, and follow-up.

Exclusion Criteria:

1. Acquired hemophilia

2. Congenital or acquired bleeding disorder in addition to Hemophilia A

3. ITI factor replacement regimen within the past one month unless there is clear evidence of ITI failure with no reduction in inhibitor titer over the past two months

4. HIV positive with viral load ≥200 particles/μL or ≥400,000 copies/mL

5. Rituximab within the past 3 months

6. IVIG within the past 1 month

7. Treatment with other immunosuppressive drugs within the past 1 month (excluding intermittent steroid use for asthma)

8. Concomitant experimental treatment

9. History of hypersensitivity to plasma-derived VWF- or FVIII-containing concentrates

10. Elective surgery planned in the next 6 months (excluding vascular access procedure)

11. Any condition or chronic illness, which in the opinion of the investigator makes participation ill-advised

12. Inability or unwillingness to complete required screening, follow-up, and exit studies