A Study of ADVATE in People With Hemophilia A in India

NCT04985682 | Completed Phase 4 Results

• 2 other identifiers: TAK-761-40092022-004149-11
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 5

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.

Conditions

Hemophilia A

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (1)

• 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.

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

Secondary Outcomes (14)

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

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

• Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters

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

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

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

• ABR With Prophylactic Treatment of ADVATE Categorized Based on Location of Bleed

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

• ABR With Prophylactic Treatment of ADVATE Categorized Based on Type of Bleed

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

Study Arms (1)

Hemophilia A Group

EXPERIMENTAL

Participants with hemophilia A were treated with ADVATE according to a regimen determined by the treating physician at the study site and in accordance with the national product label under standard clinical practice for 6.7 months.

Biological: ADVATE

Interventions

ADVATE BIOLOGICAL

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

Hemophilia A Group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Baxalta now part of Shire: lead
• Takeda Development Center Americas, Inc.: collaborator

Study Sites (5)

Amrita Institute of Medical Science & Research Centre

Ernākulam, Kerala, 682041, India

Location

St. John's Medical College

Bengaluru, 560034, India

Location

K J Somaiya Hospital & Research Centre

Mumbai, 400022, India

Location

All India Institute of Medical Sciences (AIIMS)

New Delhi, 110029, India

Location

Unique Children's Hospital Pvt. Ltd.

Pune, 411019, India

Location

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Medical Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 29, 2021
• First Posted: August 2, 2021
• Study Start: January 14, 2022
• Primary Completion: February 10, 2023
• Study Completion: February 10, 2023
• Last Updated: March 7, 2024
• Results First Posted: March 7, 2024

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

IN (5)