Goal Attainment and Physical Activity in People With Hemophilia A

NCT06530030 | Terminated FDA Drug

• 2 other identifiers: OBS18140U1111-1306-7241
• Study Type: observational
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase 4, multi-center, observational study conducted in patients aged 12 to 50 years with moderate or severe hemophilia A who are newly starting prophylaxis with efa in the US and Japan. This study aims to enroll 35 patients.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (19)

• Proportion of total goals achieved

  Reported as number of individual goals with a raw GAS score of 0, +1, or +2 divided by the total number of goals set

  Month 3

• Proportion of total goals achieved

  Month 6

• Proportion of total goals achieved

  Month 9

• Proportion of total goals achieved

  Month 12

• Proportion of patients who reached a +2 or +1 on at least one goal

  Each goal will be ranked in a 5-point Likert scale ranging from +2 to -2 with +2 indicating a better goal achievement.

  Month 3

• Proportion of patients who reached a +2 or +1 on at least one goal

  Month 6

• Proportion of patients who reached a +2 or +1 on at least one goal

  Month 9

• Proportion of patients who reached a +2 or +1 on at least one goal

  Month 12

• Overall GAS score

  Goal Attainment Scaling (GAS) is a patient-centric clinician-reported outcome measure utilized to select and scale goals, with higher scores indicating greater goal attainment. An overall GAS score utilizing all goals set by a patient at each time point of interest will be calculated.

  Month 3

• Overall GAS score

  Goal Attainment Scaling (GAS) is a patient-centric clinician-reported outcome measure utilized to select and scale goals, with higher scores indicating greater goal attainment. An overall GAS score utilizing all goals set by a patient at each time point of interest will be calculated.

  Month 6

• Overall GAS score

  Goal Attainment Scaling (GAS) is a patient-centric clinician-reported outcome measure utilized to select and scale goals, with higher scores indicating greater goal attainment. An overall GAS score utilizing all goals set by a patient at each time point of interest will be calculated.

  Month 9

• Overall GAS score

  Goal Attainment Scaling (GAS) is a patient-centric clinician-reported outcome measure utilized to select and scale goals, with higher scores indicating greater goal attainment. An overall GAS score utilizing all goals set by a patient at each time point of interest will be calculated.

  Month 12

• Frequency of selection of category of physical activity

  Categories of the goals selected from the goal inventory

  Baseline

• Monthly average of daily step count

  The monthly average of daily step count (recorded by physical tracker) will be calculated using the total steps recorded in a 30-day period, accounting for the number of compliant days in that period.

  From Baseline to Month 12

• Monthly average of daily activity count

  Activity count includes: time spent in x, y, and z axes, vector magnitude counts, estimated calories, wear time, time spent in locomotion and non-locomotion. Counts will be averaged across the month.

  From Baseline to Month 12

• Monthly average of daily Metabolic equivalent of tasks (MET)

  Estimated by the physical activity tracker

  From Baseline to Month 12

• Monthly average of daily Moderate and Vigorous Physical Activity (MVPA)

  Summarized from MET data with time spent in moderate intensity, and vigorous intensity physical activity

  From Baseline to Month 12

• Monthly average of daily intensity gradient

  The intensity gradient is a measure of activity intensity distribution that reflects the distribution of intensity of physical activity by modeling the relationship between intensity and time.

  From Baseline to Month 12

• Monthly average of weekly intensity gradient

  From Baseline to Month 12

Secondary Outcomes (31)

• Change in PROMIS Pain intensity score

  From Baseline to Month 12

• Change in HAL Score

  From Baseline to Month 12

• Change in PROMIS pediatric Pain intensity score

  From Baseline to Month 12

• Change in PedHAL Score

  From Baseline to Month 12

• Change in school/work hours missed as assessed by WPAI+CIQ:HS

  From Baseline to Month 12

Study Arms (1)

Cohort

patients aged 12 to 50 years with moderate or severe hemophilia A who are newly starting prophylaxis with efa

Drug: efanesoctocog alfa

Interventions

efanesoctocog alfa DRUG

All treatment decisions must be made independent of study participation and study-related visits/assessments are not intended to inform treatment decisions. No study medication is provided as part of study participation.

Also known as: Altuviiio

Cohort

Eligibility Criteria

• Age: 12 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Patients with moderate or severe Hemophilia A

You may qualify if:

• At enrollment, newly starting prophylaxis therapy with efanesoctocog alfa according to usual clinical practice, with efanesoctocog alfa initiated within 6 months after the enrollment visit
• Diagnosis of moderate (endogenous FVIII activity between 1% to 5% of normal) or severe (endogenous FVIII activity \<1% of normal) hemophilia A
• Aged 12 to 50 years at time of enrollment, inclusive
• Access to a smartphone device (Android version 12.0 or higher; or iOS 13 or higher) with Bluetooth 4.0 (minimum) or 5.0 (recommended) capabilities for compatibility with physical activity tracker
• Availability of home-based access to internet for electronic patient-reported outcome (ePRO)/diary assessments
• Willingness to utilize the activity tracking device

You may not qualify if:

• Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥ 0.60 BU/mL
• Use of efanesoctocog alfa for prophylaxis in the 6 months prior to enrollment

Sponsors & Collaborators

• Sanofi: lead

Study Sites (1)

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61614, United States

Location

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 Design

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

Study Record Dates

• First Submitted: July 17, 2024
• First Posted: July 31, 2024
• Study Start: November 26, 2024
• Primary Completion: September 18, 2025
• Study Completion: September 18, 2025
• Last Updated: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

US (1)