Turoctocog Alfa in Haemophilic Italian Patients: Protection and Engagement in Recreational Activities

Completed

• Conditions: Haemophilia A
• Interventions: Drug: Turoctocog alfa

• Registration Number: NCT04584892
• Lead Sponsor: Novo Nordisk A/S

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-10-06
• Study First Submitted QC: 2020-10-06
• Study First Posted: 2020-10-14
• Study Start: 2021-04-12
• Primary Completion: 2023-03-27
• Study Completion: 2023-03-27
• Status Verified: 2023-11
• Last Update Submitted: 2023-11-27
• Last Update Posted: 2023-11-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 18

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Haemophilia A	Turoctocog alfa	Patients enrolled will have Haemophilia A (any severity), needing turoctocog alpha prophylactic therapy.

Primary Outcome Measures

Name	Time	Method
Individual prophylaxis (PPX) regimen with turoctocog alfa: Every second day, or three times a week (3TW), or twice a week (2TW)	Baseline (week 0)	Count

Secondary Outcome Measures

Name	Time	Method
Individual Annual Bleeding Rate (ABR)	From baseline to final visit (12 months)	Count
Reported reasons for change of PPX regimens	Final visit (12 months)	Count (reasons according to the predefined list, see below); Individual reported reasons for switch will be selected among the following predefined: 1. to improve articular protection; 2. due to bleedings; 3. due to patients' needs, including: physical inactivity, work, recreational activity, travel, other.
Individual PPX regimen with turoctocog alfa: every second day, or 3TW, or 2TW	Final visit (12 months)	Count
Change of PPX regimen: YES/NO	12 months after treatment initiation	Count (participants who have changed regimen or not)
Difference in score of 'Bridging Hemophilia Experiences, Results, and Opportunities' BHERO questionnaire	Final visit (12 months) vs Baseline (week 0)	Points; The following sections of the BHERO questionnaire will be used: 1. Haemophilia Activities List (HAL: measures the impact of haemophilia on self-perceived functional abilities in patients (42 questions in 7 domains). Scores from 1 (worst) to 6 (best). The final score is the sum of all the scores.); 2. Recreational and sport activities; 3. Brief pain inventory, exploring the presence and intensity of physical pain, and its impact on performance of daily activities.; 4. International Physical Activities questionnaire (IPAQ) score (Score\<700 = Inactive Patient, Score 700 to 2519 = Sufficiently active patient, Score ≥ 2520 = Active or very active patient.
Difference in circulating turoctocog alfa levels	Final visit (12 months) vs Baseline (week 0)	IU/mL; Before 12 months visit, monitoring will be reported only if measured in clinical practice due to bleedings or other clinical reasons.

Trial Locations

Locations (1)

Novo Nordisk Investigational Site; 🇮🇹 Vicenza, Italy