Clinical trial NCT04876365: A Study in Children, Teenagers and Adults With Severe Hemophilia A Who Switched From Other Factor VIII Treatments to Adynovate
Official title: Real-World Effectiveness of PEGylated, Recombinant Antihemophilic Factor (Adynovate) Prophylaxis in Patients With Hemophilia A in Canada: A Retrospective, Intra-patient Comparison With a Before-After Design
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2021-05-06
Enrollment: 153
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
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).

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.


Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Percentage of Time Spent With Factor Levels Above 0.01 IU/ml
timeFrame: approximately 72 months
measure: Number of Participants With Adverse Events (AEs)
timeFrame: approximately 72 months
Secondary outcomes:
measure: Percentage of Time Spent With Factor Levels Above 0.03 IU/ml
timeFrame: approximately 72 months
measure: Percentage of Time Spent With Factor Levels Above 0.05 IU/ml
timeFrame: approximately 72 months
measure: Terminal Half-life of Adynovate
timeFrame: approximately 72 months
measure: Clearance (Cl) of Adynovate
timeFrame: approximately 72 months
measure: Maximum Observed Drug Concentration (Cmax) of Adynovate
timeFrame: approximately 72 months
measure: Area Under the Curve (AUC) of Adynovate
timeFrame: approximately 72 months
measure: Annualized Total Factor Consumption
timeFrame: approximately 72 months
measure: Annualized Total Factor Consumption for Bleeds
timeFrame: approximately 72 months
measure: Estimated Factor Consumption
timeFrame: approximately 72 months
measure: Theoretical Factor Consumption
timeFrame: approximately 72 months
measure: Annualized Bleeding Rate (ABR)
timeFrame: approximately 72 months
measure: Annualized Spontaneous Bleeding Rate (AsBR),
timeFrame: approximately 72 months
measure: Annualized Joint Bleeding Rate (AjBR)
timeFrame: approximately 72 months
measure: Hemophilia Joint Health Score (HJHS)- Total Score
timeFrame: approximately 72 months
measure: Health-Related Quality of Life (HRQoL) Assessed by Patient-reported Outcome Burdens and Experiences (PROBE) Questionnaire
timeFrame: approximately 72 months
measure: HRQoL Assessed by EuroQoL Group 5-Dimension 5-Level Self-Report (EQ-5D-5L)
timeFrame: approximately 72 months
Other outcomes:
Locations:
city: Hamilton
country: Canada
facility: Hamilton-Niagara Regional Hemophilia Treatment Centre
state: Ontario
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: