Hemophilia A Clinical Trial
— INITIATEOfficial title:
INdividualized ITI Based on Fviii(ATE) Protection by VWF (INITIATE)
NCT number | NCT03204539 |
Other study ID # | 988465 |
Secondary ID | |
Status | Terminated |
Phase | Phase 4 |
First received | |
Last updated | |
Start date | June 1, 2017 |
Est. completion date | July 19, 2019 |
Verified date | May 2020 |
Source | University of California, Davis |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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)].
Status | Terminated |
Enrollment | 1 |
Est. completion date | July 19, 2019 |
Est. primary completion date | July 19, 2019 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | N/A and older |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
United States | Tulane University | New Orleans | Louisiana |
United States | University of California, Davis | Sacramento | California |
United States | Rady Children's Hospital San Diego | San Diego | California |
Lead Sponsor | Collaborator |
---|---|
University of California, Davis |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Time to Negative Inhibitor | This endpoint was chosen because a shorter time to negative inhibitor should decrease monthly break-through bleeding frequency in the early phase of ITI | completion of immune tolerance induction, up to 18 months | |
Secondary | Time to Achieve Partial and Complete Success | Secondary endpoints include time to achieve partial and complete success as defined according to the following criteria: Inhibitor titer <0.6 BU. Incremental in vivo FVIII recovery in the normal range [=66% of normal (1.5% per IU/kg), equal to 0.99%per IU/kg] with samples taken prior to and 15 or 30 minutes after concentrate treatment. The recovery assessment should be done without any wash-out period. Half-life of FVIII >6 hours. The half-life assessment should be done in a non-bleeding status without any wash-out period. Complete Success (CS) of ITI: All three criteria above met. Partial Success (PS) of ITI: The first two of the three criteria above met. Partial Response (PR) of ITI: One of the three criteria above met. Partial Failure (PF) of ITI: Inhibitor still present, but titer is decreased to <5 BU in contrast to =5 BU before start. Complete Failure (CF) of ITI: None of the above mentioned criteria met, and the inhibitor titer is still =5 BU. |
completion of immune tolerance induction, up to 18 months | |
Secondary | Absence of Relapse, up to 12 Months After Achievement of Complete or Partial ITI Success | one year after completion of immune tolerance induction, up to 30 months | ||
Secondary | The Number of Break-through Bleeding Events During the Course of ITI-treatment· | completion of immune tolerance induction, up to 18 months | ||
Secondary | Cost of ITI - Including Bleeding Control Using Bypassing Agents Prior to Start and During ITI | completion of immune tolerance induction, up to 18 months | ||
Secondary | Subject Quality of Life | measured with the Haemo-QOL questionnaire | completion of immune tolerance induction, up to 18 months | |
Secondary | Subject Compliance With ITI Treatment Regimen | We will be looking at drug accountability reports/ logs which will reflect each subject's usage of Wilate | completion of immune tolerance induction, up to 18 months | |
Secondary | The Impact of Inhibitor Titer at Start of ITI and During the Course of ITI, Including the Peak Titer of the Inhibitor | completion of immune tolerance induction, up to 18 months | ||
Secondary | Understand Other Factors Related to ITI Success Using Additional Biologic Assays | If subject consents, the following assays will be performed: epitope mapping immunogenotyping/HLA genotyping FVIII genetic testing |
screening/baseline |
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