A Study in Children, Teenagers and Adults With Severe Hemophilia A Who Switched From Other Factor VIII Treatments to Adynovate

NCT04876365 | Completed FDA Drug

• 2 other identifiers: TAK-660-4004MACS-2020-061601
• Study Type: observational
• Target: 153
• Locations: 1 country
• Sites: 1

Brief Summary

The main aims of the study are to assess the safety profile of Adynovate as well as how well people respond to the preventive treatment with Adynovate. This study is about reviewing and collecting data of the participants before and after the switch to Adynovate that are already available. No new information will be collected during this study. The total time for data collection in the study will be approximately 72 months (36 months before and 36 months after switching to Adynovate). Participants will not receive Adynovate as part of this study. As participants are not treated in this study, they do not need to visit their doctor in addition to their normal visits.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (2)

• Percentage of Time Spent With Factor Levels Above 0.01 IU/ml

  Percentage of time spent with factor levels above 0.01 (International units per milliliter \[IU/ml\], is calculated as: time spent with factor levels above 0.01 IU/ml/ total time in the study. The calculation will be performed by simulating, based on the infusions recorded in the treatment diaries and the individual PK profiles, all the times intervals between each infusion and the time at which the concentration of 0.01 IU/mL is reached, and the time between reaching the 0.01 IU/mL and the subsequent infusion.

  approximately 72 months

• Number of Participants With Adverse Events (AEs)

  An AEs is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. Number of participants with AEs related to inhibitor development, thrombosis, death, infection, cancer and other will be reported.

  approximately 72 months

Secondary Outcomes (16)

• Percentage of Time Spent With Factor Levels Above 0.03 IU/ml

  approximately 72 months

• Percentage of Time Spent With Factor Levels Above 0.05 IU/ml

  approximately 72 months

• Terminal Half-life of Adynovate

  approximately 72 months

• Clearance (Cl) of Adynovate

  approximately 72 months

• Maximum Observed Drug Concentration (Cmax) of Adynovate

  approximately 72 months

Study Arms (1)

All Participants

All participants diagnosed with severe hemophilia A previous received prophylaxis regimen for Standard Half-life/Extended Half-life Factor VIII (SHL/EHL-FVIII) products will be compared to after the participants switched to regular prophylaxis with Adynovate with at least 6 months follow up.

Other: Non-Interventional

Interventions

Non-Interventional OTHER

This is a Non-Interventional Study.

All Participants

Eligibility Criteria

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

Study Population

Study population will comprise of participants of all ages receiving regular prophylactic treatment with Adynovate at a Canadian Hemophilia Treatment Center and registered in CBDR (Canadian Bleeding Registry).

You may qualify if:

• Participants with severe hemophilia A.
• All age groups (less than \[\<\] 12 and greater than \[\>\] 12 years of age).
• Participant with greater than equal to (\>=) 150 documented exposure days (EDs).
• Treated with Adynovate:
• Having a PK analysis done/data available for post-hoc modeling
• Having recorded clinical outcomes analysis: for \> 6 months
• Treated with a SHL/EHL-FVIII product for at least six months before switching to Adynovate
• To qualify for the secondary objective participants will need to have a WAPPS study performed on Adynovate and on the SHL/EHL-FVIII they were treated before switching.

You may not qualify if:

• Any participant who meets any of the following criteria will not qualify for entry into the study:
• Participants with only on-demand Factor VIII (FVIII) use.
• Current presence of FVIII inhibitory antibodies. (Participants with a history of inhibitors, if any, will be considered for a sensitivity analysis).
• Diagnosis of other inherited or acquired hemostatic defect other than hemophilia A.

Sponsors & Collaborators

• Takeda: lead

Study Sites (1)

Hamilton-Niagara Regional Hemophilia Treatment Centre

Hamilton, Ontario, L8N 3Z5, Canada

Location

Related Links

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

MeSH Terms

Conditions

Hemophilia A

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

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 5, 2021
• First Posted: May 6, 2021
• Study Start: November 30, 2021
• Primary Completion: February 28, 2022
• Study Completion: February 28, 2022
• Last Updated: July 24, 2023

Record last verified: 2023-07

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

CA (1)