Safety of ADVATE in previously treated Indian patients for treatment of mild , moderate and severe bleeding episodes in Haemophilia A

Phase 4

Active, not recruiting

• Conditions: Health Condition 1: - Health Condition 2: D66- Hereditary factor VIII deficiency

• Registration Number: CTRI/2021/10/037406
• Lead Sponsor: Baxalta US Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 29 May 2023

Recruitment & Eligibility

• Status: Closed to Recruitment of Participants
• Sex: Not specified
• Target Recruitment: 50

Inclusion Criteria

1. The subject or legally authorized representative (in case of study participants <18 years of age) gave written informed consent to participate in the study.

2. Subject of any age with hemophilia A.

3. Subject is defined as previously treated patient (PTP):

• Subject aged >= 6 years that has been previously treated with plasma-derived and/or recombinant FVIII concentrate(s) for a minimum of 150 exposure days (EDs).

• Subject aged < 6 years that has been previously treated with plasma-derived and/or recombinant FVIII concentrate(s) for a minimum of 50 EDs.

4. Subject has negative history of FVIII inhibitors and negative inhibitor at screening defined as less than 0.6 Bethesda units (BU)/mL (Nijmegen-modified Bethesda assay).

5. Subject is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count >= 200 cells/mm3, as confirmed by central laboratory at screening.

6. Subject is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.

7. The subject is willing and able to comply with the requirements of the protocol

Exclusion Criteria

1. Subject has known hypersensitivity to mouse or hamster proteins or to any of the excipients of FVIII concentrates.

2. Subject has been diagnosed with bleeding disorder(s) other than congenital hemophilia A, such as acquired hemophilia A, von Willebrand´s disease or thrombocytopenia (platelet count <100,000/mL).

3. Subject has received treatment for hemophilia A with non-FVIII products/concentrates (eg, emicizumab [Hemlibra®]) in the 6 months prior to screening.

4. Subject has severe chronic hepatic dysfunction [eg, >= 5 times upper limit of normal alanine aminotransferase (ALT), aspartate aminotransferase (AST) or INR > 1.5 as confirmed by central laboratory at screening].

5. Subject has planned, or is likely to have, surgery during the study period.

6. Subject has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subjectâ??s safety or compliance.

7. Subject is currently receiving or is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone greater than mg/day, or α-interferon) other than antiretroviral chemotherapy.

8. Subject has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.

9. Subject is a family member or employee of the investigator.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Incidence of SAEs (including FVIII inhibitor formation) that are at least possibly related to ADVATETimepoint: 6 months ± 1 week

Secondary Outcome Measures

Name	Time	Method
Efficacy: <br/ ><br>1. Annualized bleeding rate (ABR) with prophylactic use of ADVATE. <br/ ><br>2. Total number of infusions and the average number of infusions per week per month during prophylactic treatment. <br/ ><br>3. Total and average body mass adjusted consumption of ADVATE per week per month during prophylactic treatment. <br/ ><br>4. Overall hemostatic efficacy rating for treatment of bleeding episodes. <br/ ><br>5. Number of ADVATE infusions required to achieve bleed resolution. <br/ ><br>6. Body mass adjusted consumption of ADVATE per bleeding episode. <br/ ><br>Timepoint: 6 months ± 1 week;Safety: <br/ ><br>1. Incidence of non-serious AEs that are at least possibly related to ADVATE. <br/ ><br>2. Clinically significant changes in clinical laboratory parameters (hematology and clinical chemistry) <br/ ><br>Timepoint: 6 months ± 1 week