Clinical trial NCT04584892: Turoctocog Alfa in Haemophilic Italian Patients: Protection and Engagement in Recreational Activities
Official title: Turoctocog Alfa in Haemophilic Italian Patients: Protection and Engagement in Recreational Activities (THIRd). An Observational, Prospective, Multicentre, Cohort Study on Italian Patients With Haemophilia A in Prophylaxis Treatment With Turoctocog Alfa Under Current Clinical Conditions
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Haemophilia A
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2020-10-14
Enrollment: 18
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients enrolled will have Haemophilia A (any severity), needing turoctocog alpha prophylactic therapy.

Brief summary: This is a study on Italian patients with haemophilia A in prophylaxis treatment with Turoctocog alfa under routine clinical conditions. The purpose of the study is to investigate the therapeutic scheme in a population of patients treated with Turoctocog alfa and to investigate the participation in recreational activities, the level of physical activity, and quality of life. Participants will get NovoEight® (Turoctocog alfa) as prescribed to them by the study doctor. The study will last for about 12 months. Participants will be requested to fill in the Questionnaires investigating the participation in recreational activities, the level of physical activity, and quality of life.


Eligibility criteria: Inclusion Criteria:

* Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
* Male or female, age above or equal to 12 years at the time of signing informed consent.
* Haemophilia A (any severity), needing prophylactic therapy (with the usual recommended doses).
* Decision to treat with turoctocog alfa, made by the treating Physician and the subject/Legally Acceptable Representative (LAR) based on local label before and independently from the decision to include the subject in this study.

Exclusion Criteria:

* Previous participation in this study, defined as previously signed informed consent;
* Presence of other coagulation disorders;
* Presence of any inhibitor;
* Mental problems, poor compliance, linguistic barriers or other conditions which can impede the understanding of the study aims and the participation to it.

Primary outcomes:
measure: Individual prophylaxis (PPX) regimen with turoctocog alfa: Every second day, or three times a week (3TW), or twice a week (2TW)
timeFrame: Baseline (week 0)
Secondary outcomes:
measure: Individual PPX regimen with turoctocog alfa: every second day, or 3TW, or 2TW
timeFrame: Final visit (12 months)
measure: Change of PPX regimen: YES/NO
timeFrame: 12 months after treatment initiation
measure: Reported reasons for change of PPX regimens
timeFrame: Final visit (12 months)
measure: Difference in score of 'Bridging Hemophilia Experiences, Results, and Opportunities' BHERO questionnaire
timeFrame: Final visit (12 months) vs Baseline (week 0)
measure: Difference in circulating turoctocog alfa levels
timeFrame: Final visit (12 months) vs Baseline (week 0)
measure: Individual Annual Bleeding Rate (ABR)
timeFrame: From baseline to final visit (12 months)
Other outcomes:
Locations:
city: Milano
country: Italy
facility: Novo Nordisk Investigational Site
state: MI
city: Bologna
country: Italy
facility: Novo Nordisk Investigational Site
city: Castelfranco Veneto
country: Italy
facility: Novo Nordisk Investigational Site
city: Catania
country: Italy
facility: Novo Nordisk Investigational Site
city: Cesena
country: Italy
facility: Novo Nordisk Investigational Site
city: Firenze
country: Italy
facility: Novo Nordisk Investigational Site
city: Milano
country: Italy
facility: Novo Nordisk Investigational Site
city: Napoli
country: Italy
facility: Novo Nordisk Investigational Site
city: Roma
country: Italy
facility: Novo Nordisk Investigational Site
city: Vicenza
country: Italy
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Reporting Anchor and Disclosure 1452
role: STUDY_DIRECTOR
References: