Clinical trial NCT04985682: A Study of ADVATE in People With Hemophilia A in India
Official title: Phase 4, Multicenter, Prospective, Interventional, Post-Marketing Study in Hemophilia A Patients in India Receiving ADVATE as On-Demand or Prophylaxis Under Standard Clinical Practice
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hemophilia A
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Takeda Development Center Americas, Inc.
First posted: 2021-08-02
Enrollment: 50
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
Secondary outcomes:
measure: Number of Participants With Non-serious Adverse Events (AEs) at Least Possibly Related to ADVATE
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Total Annualized Bleeding Rate (ABR) With Prophylactic Treatment of ADVATE
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: ABR With Prophylactic Treatment of ADVATE Categorized Based on Location of Bleed
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: ABR With Prophylactic Treatment of ADVATE Categorized Based on Type of Bleed
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Total Number of ADVATE Infusions Required During Prophylactic Treatment
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Average Number of ADVATE Infusions Required Per Week During Prophylactic Treatment
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Average Number of ADVATE Infusions Required Per Month During Prophylactic Treatment of Bleeding Episode
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Total Body Mass Adjusted Consumption of ADVATE During Prophylactic Treatment
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Average Body Mass Adjusted Consumption of ADVATE Per Week During Prophylactic Treatment
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Average Body Mass Adjusted Consumption of ADVATE Per Month During Prophylactic Treatment
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Overall Hemostatic Efficacy Rating of ADVATE for Treatment of Bleeding Episodes
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Number of ADVATE Infusions Required to Achieve Resolution of Bleeding Episodes
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
measure: Total Body Mass Adjusted Consumption of ADVATE Per Bleeding Episode
timeFrame: Baseline (Day 0) up to end of study (up to 12.9 months)
Other outcomes:
Locations:
city: Ernakulam
country: India
facility: Amrita Institute of Medical Science & Research Centre
state: Kerala
city: Bengaluru
country: India
facility: St. John's Medical College
city: Mumbai
country: India
facility: K J Somaiya Hospital & Research Centre
city: New Delhi
country: India
facility: All India Institute of Medical Sciences (AIIMS)
city: Pune
country: India
facility: Unique Children's Hospital Pvt. Ltd.
Study officials:
affiliation: Takeda
name: Medical Director
role: STUDY_DIRECTOR
References: