Efanesoctocog Alfa for Hemophilia A

Not currently recruiting at 81 trial locations

Overseen By Trial Transparency email recommended (Toll free number for US & Canada)

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Sanofi

Must be taking: Efanesoctocog alfa

Disqualifiers: Other bleeding disorders, FVIII inhibitor, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on evaluating the effectiveness of efanesoctocog alfa for individuals with hemophilia A, a condition where blood doesn't clot properly. Researchers aim to assess how this treatment affects joint health over time. Participants will either receive regular doses to prevent bleeding or take the treatment as needed. Ideal candidates for this trial are those with hemophilia A who have recently started using efanesoctocog alfa as part of their regular care routine. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants an opportunity to contribute to the potential availability of a new treatment.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with your doctor.

What is the safety track record for efanesoctocog alfa?

Research has shown that efanesoctocog alfa is generally safe for people with hemophilia A. In one study, patients who received this treatment did not report any major safety issues, and no bleeding episodes occurred during the treatment period. Another study found that weekly treatment maintained high factor levels for most of the week, potentially preventing bleeding.

Evidence also suggests that even children with severe hemophilia A were well-protected against bleeding with efanesoctocog alfa. Overall, various studies have demonstrated that this treatment is safe, making it a promising option for those with hemophilia A. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Efanesoctocog alfa is unique because it targets hemophilia A with a focus on long-term joint health, which is a significant concern for patients. Unlike standard treatments that often involve frequent infusions to manage bleeding episodes, efanesoctocog alfa is designed for both prophylactic and on-demand use, offering flexibility based on individual needs. Researchers are excited because it's engineered to provide extended protection with less frequent dosing, which could improve the quality of life by potentially reducing joint damage over time. This innovative approach targets the underlying issue of joint health, offering hope for better long-term outcomes.

What is the effectiveness track record for efanesoctocog alfa in treating hemophilia A?

Research has shown that efanesoctocog alfa effectively manages hemophilia A. Administering this treatment once a week has resulted in a 77% reduction in bleeding compared to standard treatments. Studies confirm it offers strong protection against bleeding for both children and adults. In this trial, participants will be divided into two cohorts: Cohort A will receive efanesoctocog alfa as a prophylactic treatment, while Cohort B will receive it on-demand for treating bleeding episodes. Overall, efanesoctocog alfa appears to be a reliable option for individuals with severe hemophilia A. ^{1 2 5 6 7}

Who Is on the Research Team?

Clinical Sciences & Operations

Principal Investigator

Sanofi

Are You a Good Fit for This Trial?

Inclusion Criteria

Have a diagnosis of hemophilia A

Patients starting efanesoctocog alfa treatment as per standard of care no more than one month prior to the enrollment date, for either on demand or prophylaxis. Patients starting efanesoctocog alfa treatment for a surgery event may also be enrolled only if the treatment is prescribed at enrollment.

Physician's decision to treat the patient with efanesoctocog alfa is made prior to and independently of participation in the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Enrollment

Participants are enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape

Up to 5 years

Annual/semi-annual visits

Follow-up

Participants are monitored for safety and effectiveness of efanesoctocog alfa

Up to 5 years

Annual/semi-annual visits

What Are the Treatments Tested in This Trial?

Interventions

• Efanesoctocog Alfa

How Is the Trial Designed?

Group I: Cohort B (On-Demand treatment) Experimental Treatment 1 Intervention

Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria

Group II: Cohort A (Prophylactic treatment) Experimental Treatment 1 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

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Citations

1.

nejm.org

nejm.org/doi/full/10.1056/NEJMoa2209226

Efanesoctocog Alfa Prophylaxis for Patients with Severe ...

In patients with severe hemophilia A, once-weekly efanesoctocog alfa provided superior bleeding prevention to prestudy prophylaxis, normal to near-normal ...

2.

ncbi.nlm.nih.gov

ncbi.nlm.nih.gov/books/NBK615900

Efanesoctocog alfa for treating and preventing bleeding ...

Efanesoctocog alfa is recommended as an option for treating and preventing bleeding episodes in people 2 years and over with haemophilia A (congenital factor ...

3.

sobi.com

sobi.com/en/press-releases/late-breaking-efanesoctocog-alfa-data-presented-isth-demonstrates-highly-effective-bleed-protection-children-severe-haemophilia-once

Late-breaking efanesoctocog alfa data presented at ISTH ...

These data confirm that a once-weekly 50 IU/kg dose of efanesoctocog alfa provides highly effective bleed protection in both children and adults ...

4.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04644575

Study Details | NCT04644575 | Long-term Safety and ...

Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment. To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes.

5.

hematologyadvisor.com

hematologyadvisor.com/news/hemophilia-efanesoctocog-alfa-highly-effective-treatment-risk/

Perioperative Management of Efanesoctocog Alfa Highly ...

Researchers determined perioperative management of severe hemophilia A using efanesoctocog alfa was considered highly effective.

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC8864644/

Efanesoctocog alfa for hemophilia A: results from a phase 1 ...

4 once-weekly doses of efanesoctocog alfa were well tolerated, no safety concerns were identified, and no bleeds were reported during the treatment period.

7.

sanofi.com

sanofi.com/en/media-room/press-releases/2023/2023-01-25-22-30-00-2595681

NEJM publishes once-weekly efanesoctocog alfa Phase 3 ...

These data demonstrate that efanesoctocog alfa delivered normal to near-normal factor activity levels (>40%) for the majority of the week with once-weekly ...