A Study of ADVATE in People With Hemophilia A in India

Baxalta now part of Shire50 enrolled

Overview

The main aim of this study is to learn more about side effects of Advate when given as standard treatment to people with hemophilia A who have already been treated. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. Participants will need to visit the study doctor 5 times in total during the study. During these visits, study data will be collected by the study doctor.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hemophilia A

Interventions

ADVATEBIOLOGICAL

Antihemophilic factor (AHF) activity expressed in international units (IU) per vial.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: * The participant or legally authorized representative (in case of study participants less than (\<) 18 years of age) gave written informed consent to participate in the study. * Participant of any age with hemophilia A. * Participant defined as a previously treated patient (PTP): * Participant aged greater than or equal to (\>=) 6 years that has been previously treated with plasma-derived and/or recombinant FVIII concentrate(s) for a minimum of 150 exposure doses (EDs). * Participant aged less than \<6 years that has been previously treated with plasma-derived or recombinant FVIII concentrate(s) for a minimum of 50 EDs. * Participant as negative history of FVIII inhibitors and negative inhibitor at screening defined as less than 0.6 Bethesda units (BU) per milliliter (Nijmegen-modified Bethesda assay). * Participant is human immunodeficiency virus negative (HIV-); or human immunodeficiency virus positive (HIV+) with stable disease and cluster of differentiation 4 (CD4+) count \>=200 cells per cubic millimeter (mm\^3), as confirmed by central laboratory at screening. * Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, anti-body titer will be confirmed by PCR), as confirmed by central laboratory at screening; or hepatitis C virus positive (HCV+) with chronic stable hepatitis. * Participant is willing and able to comply with the requirements of the protocol. Exclusion Criteria: * Participant has known hypersensitivity to mouse or hamster proteins or to any of the excipients of FVIII (factor VIII) concentrates. * Participant has been diagnosed with bleeding disorder(s) other than congenital hemophilia A, such as acquired hemophilia A, von Willebrand´s disease (VWD) or thrombocytopenia (platelet count \<100,000 per milliliter). * Participant has received treatment for hemophilia A with non-FVIII products or concentrates (example, emicizumab \[Hemlibra®\]) in the 6 months prior to screening. * Participant has severe chronic hepatic dysfunction (example, \>=5 times upper limit of normal alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\] or international normalized ratio \[INR\] \>1.5 as confirmed by central laboratory at screening). * Participant has planned or is likely to have, surgery during the study period. * Participant has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug or alcohol use that, in the opinion of the investigator, would affect participant's safety or compliance. * Participant currently receiving or is scheduled to receive during the course of the study, an immunomodulating drug (example, corticosteroid agents at a dose equivalent to hydrocortisone \>10 milligram per day, or α-interferon) other than antiretroviral chemotherapy. * Participant 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. * Participant is a family member or employee of the investigator.

Locations (5)

Amrita Institute of Medical Science & Research Centre

Ernākulam, Kerala, India

St. John's Medical College

Bengaluru, India

K J Somaiya Hospital & Research Centre

Mumbai, India

All India Institute of Medical Sciences (AIIMS)

New Delhi, India

Outcomes

Primary Outcomes

Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE

An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. An SAE was defined as any untoward medical occurrence that resulted in death; was life-threatening; required inpatient hospitalization or prolongation of present hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect or was a medically important event. Number of participants with SAEs (including FVIII inhibitor formation) that were at least possibly related to ADVATE were reported.

Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)

Secondary Outcomes

Number of Participants With Non-serious Adverse Events (AEs) at Least Possibly Related to ADVATE

An AE was any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. Number of participants with non-serious AEs that were at least possibly related to ADVATE were reported.

Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)

Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters

Clinical laboratory parameters included hematology, clinical chemistry, viral serology, factor VIII (FVIII) antigen, FVIII activity, incremental recovery, and FVIII inhibitor. Clinical significance was judged as per Investigator's assessment.

Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)

Total Annualized Bleeding Rate (ABR) With Prophylactic Treatment of ADVATE

ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error was estimated using a generalized linear model (GLM). The total ABR is reported in this outcome measure.

Data from ClinicalTrials.gov

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