This is a multicenter, multinational, prospective, single-arm, nonrandomized, open-label study of approximately 25 male subjects with congenital hemophilia A with FVIII inhibitors who will receive their first (primary)treatment with Alphanate. The study will be conducted at approximately 20 study centers.
- Conditions
- Inhibitors in patients with severe Congenital Haemophilia AMedDRA version: 20.0Level: HLTClassification code 10018847Term: Haematological disordersSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2015-005524-26-IT
- Lead Sponsor
- GRIFOLS BIOLOGICALS INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 25
A subject must meet all of the following inclusion criteria at the time of the Screening/Baseline Visit (as specified below) to be eligible for participation in the study:
1. The subject has a documented diagnosis of severe congenital hemophilia A with FVIII:C <1% of normal.
2. The subject is a male <8 years of age at the Baseline Visit.
3. The subject's documented historical peak inhibitor titer is =10 Bethesda units (BU) and =200 BU.
4. The subject has an inhibitor titer >0.6 BU and <10 BU at Screening.
5. The subject has had a delay =24 months from the date of diagnosis of the inhibitor to the start of the subject'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.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
A subject meeting any of the following exclusion criteria is not eligible for participation in the study:
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 Screening—imaging studies without investigative treatments are permitted—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 clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the proportion of subjects who achieve complete immune tolerance within 33 months of initiating Alphanate for immune tolerance induction (ITI).<br>To assess the safety of Alphanate treatment for ITI;Secondary Objective: To assess the proportion of subjects who achieve either complete or partial immune tolerance within 33 months of initiating Alphanate for ITI.<br>To assess the maintenance of complete or partial immune tolerance without relapse for 12 months.<br>To assess the annualized frequency of bleeding events;Primary end point(s): The primary efficacy endpoint is the proportion of subjects achieving complete immune tolerance within 33 months of initiation of ITI treatment.;Timepoint(s) of evaluation of this end point: within 33 months of initiation of ITI treatment
- Secondary Outcome Measures
Name Time Method