Inhibitors in patients with severe Congenital Haemophilia A MedDRA version: 19.0 Level: HLT Classification code 10018847 Term: Haematological disorders System Organ Class: 100000004851
Conditions
Interventions
Trade Name: Alphanate
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: HUMAN COAGULATION FACTOR VIII
Other descriptive name: Antihemophilic Factor/von Willebrand Factor Comp
Sponsors
Grifols Biologicals Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Inclusion Criteria: 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 0.6 BU and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Exclusion Criteria: 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 is positive for human immunodeficiency virus by nucleic acid amplification technology at Screening. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary Objectives: • To assess the proportion of subjects who achieve complete immune tolerance within 33 months of initiating Alphanate for immune tolerance induction (ITI). • To assess the safety of Alphanate treatment for ITI;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 Objective: Secondary Objective(s) (if applicable) • To assess the proportion of subjects who achieve either complete or partial immune tolerance within 33 months of initiating Alphanate for ITI. • To assess the maintenance of complete or partial immune tolerance without relapse for 12 months. • To assess the annualized frequency of bleeding events | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Efficacy (if applicable): 1.Time to achieve inhibitor titer <0.6 BU, time to complete immune tolerance, and time to partial immune tolerance. (See protocol section 5.1.3.2, paragraph 3) 2.The proportion of subjects who achieve either complete or partial immune tolerance within 33 months of receiving Alphanate for ITI. 3. The proportion of subjects who maintain complete immune tolerance or partial immune tolerance without relapse for 12 months 4. The annualized frequencies of bleeding events during the ITI Treatment Phase and the Prophylactic Phase;Timepoint(s) of evaluation of this end point: A. 33 months of receiving Alphanate for ITI for the proportion of subjects who achieve either complete or partial immune tolerance. B. 12 months for the proportion of subjects who maintain complete immune tolerance or partial immune tolerance without relapse. C. 33 months + 12 months for treatment and prophylactic phases respectively for the proportion of subjects who achieve either complete or partial immune tolerance within annualized frequencies of bleeding events during the ITI Treatment Phase and the Prophylactic Phase | — |
Countries
India, Italy, Russian Federation, Spain, United States
Contacts
Public ContactSenior Mgr, Clinical Development
Grifols Therapeutics Inc.
Outcome results
None listed