Clinical Study of Plasma-Derived Factor VIII/VWF in Subjects with Congenital Hemophilia A and Inhibitors
- Conditions
- Health Condition 1: null- Congenital Hemophilia A with inhibitorsHealth Condition 2: D683- Hemorrhagic disorder due to circulating anticoagulantsHealth Condition 3: D758- Other specified diseases of bloodand blood-forming organs
- Registration Number
- CTRI/2017/04/008336
- Lead Sponsor
- Spectrum Clinical Research Pvt Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Closed to Recruitment of Participants
- Sex
- Not specified
- Target Recruitment
- 0
1. The subject has a documented diagnosis of severe congenital hemophilia A with FVIII:C <1% of normal.
2. The subject is a male �2 and <8 years of age at the Baseline Visit.
3. The subjectââ?¬•s documented historical peak inhibitor titer is ââ?°Â¥10 Bethesda units (BU) and ââ?°Â¤200 BU.
4. The subject has an inhibitor titer >0.6 BU and <10 BU at Screening.
5. The subject has had a delay ââ?°Â¤24 months from the date of diagnosis of the inhibitor to the start of the subjectââ?¬•s ITI treatment.
6. The subject has a caregiver willing to participate and comply with requirements of the protocol, including home infusions, blood sampling, and daily diary for the duration of the trial.
7. The subject has provided signed assent, if applicable (per Institutional Review Board or Ethics Committee requirements), and a parent or legal guardian has provided signed informed consent.
1. The subject has acquired factor VIII (FVIII) deficiency.
2. The subject has previously received ITI treatment.
3. The subject has a recent (within 1 month) history of central line infection at the time of Screening.
4. The subject has a high risk of cardiovascular, cerebrovascular, or thromboembolic event as judged by the investigator.
5. The subject is currently undergoing treatment with immunosuppressive drugs (eg, systemic corticosteroids), azathioprine, cyclophosphamide, high dose immunoglobulin, interferon, or the use of a protein A column or plasmapheresis and is unwilling to discontinue these treatments starting at the screening visit.
6. The subject has a known infection with human immunodeficiency virus (HIV) or has clinical signs and symptoms consistent with current HIV infection.
7. The subject has a known previous infection with hepatitis B virus or hepatitis C virus or has clinical signs and symptoms consistent with current HBV or HCV infection.
8. The subject has significant proteinuria, has a history of acute renal failure or severe renal impairment (blood urea nitrogen or creatinine >2 times the upper limit of normal), or is receiving dialysis at Screening.
9. The subject has a value of aspartate transaminase or alanine aminotransferase >2 times the upper limit of normal at Screening.
10. The subject has clinical evidence of any significant acute or chronic disease that, in the opinion of the investigator, may interfere with successful completion of the trial or place the subject at undue medical risk.
11. The subject has a history of anaphylaxis or severe systemic reaction to any plasma-derived or other blood products.
12. The subject has participated in another clinical trial of an Investigational Product within 30 days prior to Screeningââ?¬â?imaging studies without investigative treatments are permittedââ?¬â?or has received any investigational blood product within the previous 3 months.
13. In the opinion of the investigator, the subject or caregiver may have compliance problems with the protocol or the procedures of the protocol
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Proportion of subjects achieving complete immune tolerance.Timepoint: within 33 months of initiation of ITI treatment
- Secondary Outcome Measures
Name Time Method To assess the annualized frequency of bleeding eventsTimepoint: 45 months;To assess the maintenance of complete or partial immune tolerance without relapseTimepoint: 12 months;To assess the proportion of subjects who achieve either complete or partial immune <br/ ><br>tolerance.Timepoint: Within 33 months of initiating Alphanate for ITI.