A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A

NCT05707351 | Completed Phase 3 Results

• 2 other identifiers: TAK-660-30012023-000502-26
• Study Type: interventional
• Target: 37
• Locations: 1 country
• Sites: 12

Brief Summary

The main aim of the study is to determine how well Adynovate works to decrease bleeding in previously treated Chinese men and boys with severe hemophilia A when given prophylactically. Participants will be treated with Adynovate twice a week for 26 weeks or until participants have received 50 days of treatment with Adynovate (whichever takes longer). Participants will need to visit their study clinic several times during their participation.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Total Annualized Bleeding Rates (ABR)

  Total ABR was defined as the number of treated and non-treated bleeding episodes (BEs) that occurred during the treatment period, calculated as, ABR= number of unique bleeds during treatment period/(length of treatment period \[days\]/365.25). Total ABR for all BEs, spontaneous or traumatic, recorded in the participant's electronic diary and/or recorded in the physician/nurse/study site notes were reported.

  Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)

Secondary Outcomes (28)

• ABR Based on Bleeding Site

  Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)

• ABR Based on Bleeding Cause

  Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)

• Number of Adynovate Infusions Per Week During the Prophylactic Treatment Period

  Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)

• Number of Adynovate Infusions Per Month During the Prophylactic Treatment Period

  Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)

• Weight-adjusted Consumption of Adynovate Per Week During the Prophylactic Treatment Period

  Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)

Study Arms (1)

Adynovate 45±5 IU/kg

EXPERIMENTAL

Participants received prophylactic treatment with Adynovate (45 \[±5\] IU/kg), infusion, IV, twice weekly, for at least 50 EDs, or approximately 28 weeks.

Biological: Adynovate

Interventions

Adynovate BIOLOGICAL

Adynovate was injected intravenously using an appropriately sized syringe as a bolus infusion over a period of less than or equal to (\<=) 5 minutes (maximum infusion rate, 10 milliliters per minute \[mL/min\]).

Also known as: Antihemophilic Factor (recombinant) PEGylated, Rurioctocog Alfa Pegol

Adynovate 45±5 IU/kg

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant and/or legally authorized representative must voluntarily sign a written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the Participant. For the participants less than (\<) 18 years old, participants will give assent AND their parents/legally authorized representative should sign the ICF accordingly.
• Participant and/or legally authorized representative understands and is willing and able to comply with all requirements of the study protocol.
• Participant should be ethnic Chinese.
• Participant is 12 to 65 years of age at screening and male.
• Participant has severe hemophilia A (FVIII clotting activity \<1 percent \[%\]) as confirmed by the central laboratory at screening after a washout period of at least 72 to 96 hours.
• The last on-demand or prophylactic treatment received is within 3 months before screening.
• Participant has documented previous treatment with plasma-derived FVIII concentrates or recombinant FVIII for greater than (\>) 150 EDs.
• Participant is human immunodeficiency virus (HIV)-negative, or HIV-positive with stable disease and CD4+ count greater than or equal to (\>=) 200 cells per cubic millimeter (/mm\^3).
• Participant is hepatitis C virus (HCV) negative by antibody testing (if positive, additional polymerase chain reaction testing will be performed to confirm), as confirmed at screening; or HCV-positive with chronic stable hepatitis, as assessed by the investigator.

You may not qualify if:

• Participant has detectable FVIII inhibitory antibodies (\>=0.6 Bethesda units \[BU\] per milliliter \[/mL\] using the Nijmegen modification of the Bethesda assay) as confirmed by the central laboratory at screening.
• Participant has a confirmed history of FVIII inhibitory antibodies (\>=0.6 BU using the Nijmegen modification of the Bethesda assay or \>=0.6 BU using the Bethesda assay) at any time prior to screening.
• Participant has a known hypersensitivity to Adynovate or ADVATE or any of the components of the study drugs, such as mouse or hamster proteins, or other FVIII products.
• Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (example, qualitative platelet defect or von Willebrand's disease).
• Participant has severe hepatic dysfunction (example, \>=5 times the upper limit of normal \[ULN\] for alanine aminotransferase \[ALT\] or aspartate aminotransferase \[AST\], a recent or persistent international normalized ratio \[INR\] \>1.5, as confirmed by the local laboratory at screening).
• Participant has severe renal impairment (serum creatinine \>1.5 times the ULN) as confirmed by the local laboratory at screening.
• Participant is planned or likely to undergo major surgery during the study period.
• Participant has current or recent (\<30 days) use of other PEGylated drugs before study participation or scheduled use of such drugs during study participation.
• Participant has received emicizumab therapy within 6 months of screening.
• Participant is currently receiving, or scheduled to receive during the study, an immunomodulating drug (example, systemic corticosteroid agent at a dose equivalent to hydrocortisone \>10 milligram per day \[mg/day\], or alpha-interferon) other than antiretroviral chemotherapy.
• Participant has participated in another clinical study involving the use of an investigational product (IP) other than Adynovate or an investigational device within 30 days before the screening visit or is scheduled to participate in another clinical study involving an IP or investigational device during this study.
• Participant has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
• Participant, in the opinion of the investigator, is unable or unwilling to comply with the study protocol.

Sponsors & Collaborators

• Takeda: lead

Study Sites (12)

Beijing Children's Hospital, Capital Medical University

Beijing, China

Location

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

Beijing, China

Location

Xiangya Hospital of Central South University

Changsha, China

Location

Fujian Medical University Union Hospital

Fuzhou, China

Location

Nanfang Hospital Southern Medical University

Guangzhou, China

Location

Anhui Province Hospital

Hefei, China

Location

Jinan Central Hospital

Jinan, China

Location

Shenzhen Second People's Hospital

Shenzhen, China

Location

The First Affiliated Hospital of Soochow University

Suzhou, China

Location

Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences

Tianjin, China

Location

Tongji Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, China

Location

Union Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, China

Location

Related Links

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

MeSH Terms

Conditions

Hemophilia A

Interventions

BAX 855; 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
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2023
• First Posted: January 31, 2023
• Study Start: March 27, 2023
• Primary Completion: September 5, 2024
• Study Completion: September 5, 2024
• Last Updated: May 1, 2025
• Results First Posted: May 1, 2025

Record last verified: 2025-04

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

CN (12)