emicizumab for Hemophilia A

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Washington Center for Bleeding Disorders, Seattle, WA
Hemophilia A+1 More
emicizumab - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This is a phase II multicenter open-label, single-arm prospective study to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).

Eligible Conditions

  • Hemophilia A
  • Hemophilia A, Acquired

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 5 Secondary · Reporting Duration: duration of entire study

1 to 24 weeks
Remission Rate
12 to 24 weeks
Comparison of historic GTH-AH 01/2020 cohort/ Number of clinically significant bleeds
Comparison to a parallel German study cohort/ Number of clinically significant bleeds
12 weeks
Days of treatment with additional hemostatic agent
Hospitalizations
Meassure
Total dose of additional hemostatic agent
duration of entire study
Incidence and severity of adverse events

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

1 Treatment Group

Experimental-treatment
1 of 1
Experimental Treatment

51 Total Participants · 1 Treatment Group

Primary Treatment: emicizumab · No Placebo Group · Phase 2

Experimental-treatment
Drug
Experimental Group · 1 Intervention: emicizumab · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: duration of entire study
Closest Location: Washington Center for Bleeding Disorders · Seattle, WA
Photo of Seattle 1Photo of Seattle 2Photo of Seattle 3
N/AFirst Recorded Clinical Trial
1 TrialsResearching Hemophilia A
0 CompletedClinical Trials

Who is running the clinical trial?

Genentech, Inc.Industry Sponsor
1,482 Previous Clinical Trials
563,294 Total Patients Enrolled
14 Trials studying Hemophilia A
839 Patients Enrolled for Hemophilia A
University of WashingtonLead Sponsor
1,571 Previous Clinical Trials
1,567,945 Total Patients Enrolled
1 Trials studying Hemophilia A
15 Patients Enrolled for Hemophilia A
Rebecca Kruse-Jarres, MD, MPHPrincipal InvestigatorUniversity of Washington
4 Previous Clinical Trials
78 Total Patients Enrolled
4 Trials studying Hemophilia A
78 Patients Enrolled for Hemophilia A

Eligibility Criteria

Age 18+ · All Participants · 7 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You have a reduced FVIII activity (<50 %) and positive FVIII inhibitor (>0.
You are 18 or older at the time of signing the Informed Consent Form.
You are able to comply with the study protocol.
You plan to be adherent to emicizumab prophylaxis during the study.
Use of hormonal contraceptives or sterilization procedures that result in a failure rate of <1% per year during the study period.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

References