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Cohort A (Prophylactic treatment) for Hemophilia A

N/A
Recruiting
Research Sponsored by Sanofi
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 1, 2, 3, 4 and 5 years
Awards & highlights

Study Summary

This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan. Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 1, 2, 3, 4, 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and at 1, 2, 3, 4, 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
Number of Target joint development, resolution and/or recurrence
Secondary outcome measures
ABR by location for all (treated and untreated) bleeds
ABR by location for treated bleeds
ABR by type all (treated and untreated) bleeds
+30 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort B (On-Demand treatment)Experimental Treatment1 Intervention
Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria
Group II: Cohort A (Prophylactic treatment)Experimental Treatment1 Intervention
All participants on efanesoctocog alfa prophylactic treatment fulfilling the overall study inclusion/exclusion criteria. The prophylactic cohort will include the following sub-cohorts: Sub-cohort A1 (Joint imaging): Participants with severe hemophilia A and joint imaging by Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) or Joint Tissue Activity and Damage Exam (JADE) protocol performed within 6 months of initiating treatment with efanesoctocog alfa or within 3 months after initiating treatment with efanesoctocog alfa available. Sub-cohort A2 (Children with no prior joint damage):Participants with severe hemophilia A who have no prior joint damage

Find a Location

Who is running the clinical trial?

SanofiLead Sponsor
2,163 Previous Clinical Trials
3,512,280 Total Patients Enrolled
7 Trials studying Hemophilia A
9,194 Patients Enrolled for Hemophilia A
Clinical Sciences & OperationsStudy DirectorSanofi
857 Previous Clinical Trials
2,019,099 Total Patients Enrolled
9 Trials studying Hemophilia A
998 Patients Enrolled for Hemophilia A

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
~133 spots leftby Jun 2028