Emicizumab
Treatment for Hemophilia
Typical Dosage: 3 mg/kg weekly for 4 weeks, then 1.5 mg/kg weekly (or 3 mg/kg every 2 weeks, or 6 mg/kg every 4 weeks) subcutaneous
Effectiveness
95%
Safety Score
58%
Clinical Trials
48
Participants
3K
Comparative Safety Scale(Higher is safer)
Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
58
DangerousModerateSafe
Treatment Details
Dosage Range
3 mg/kg weekly for 4 weeks, then 1.5 mg/kg weekly (or 3 mg/kg every 2 weeks, or 6 mg/kg every 4 weeks) subcutaneous
Time to Effect
2-4 weeks
Treatment Duration
lifetime
Evidence Quality
HIGHNumber Needed to Treat (NNT)
1.5(Treat 1.5 patients to see 1 additional successful outcome)
Number Needed to Harm (NNH)
500(Treat 500 patients to see 1 additional serious adverse event)
Confidence Score
98%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$500,000
Monitoring:$3,000
Side Effect Mgmt:$2,000
Total Annual:$505,000
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
MODERATEICER
$150,000/QALY
QALYs Gained
0.7
Outcome-Based Costs
Cost per Responder
$515,306.12
Comparison vs Bypass agents (e.g., aPCC or rFVIIa)
Cost Difference
+$50,000/year
More expensive
QALY Difference
+0.70 QALYs
Better outcomes
Dominance
No dominance
Emicizumab Outcomes
for Hemophilia
Efficacy Outcomes
Overall Effectiveness
+95%
Response Rate
+98%
Common Side Effects
Injection site reactions
+15%
Headache
+12%
Arthralgia
+12%
Thrombotic microangiopathy/Thromboembolism (with aPCC)
+0.5%
Sources:
Active Clinical Trials
6 active trials recruiting for Emicizumab in Hemophilia
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
NCT05181618ACTIVE NOT RECRUITINGPHASE4
136 participants
INTERVENTIONAL
Los Angeles, United States +26 more
Started: Jun 20, 2022
Emicizumab in Patients With Acquired Hemophilia A
NCT05345197ACTIVE NOT RECRUITINGPHASE2
51 participants
INTERVENTIONAL
San Diego, United States +15 more
Started: Aug 31, 2022
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
NCT04431726ACTIVE NOT RECRUITINGPHASE3
55 participants
INTERVENTIONAL
Phoenix, United States +30 more
Started: Feb 4, 2021
Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis
NCT05022459RECRUITING
120 participants
OBSERVATIONAL
Little Rock, United States +6 more
Started: Aug 16, 2023
Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
NCT05500807RECRUITINGPHASE1
40 participants
INTERVENTIONAL
Orange, United States +8 more
Started: Nov 1, 2022
SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
NCT04563520RECRUITINGPHASE3
5 participants
INTERVENTIONAL
Atlanta, United States +1 more
Started: Jan 1, 2026
Completed Clinical Trials
10 completed trials for Emicizumab in Hemophilia
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A
NCT03020160COMPLETEDPHASE3
48 participants
INTERVENTIONAL
Sacramento, United States +16 more
Started: Jan 30, 2017
Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants
NCT03315455COMPLETEDPHASE3
85 participants
INTERVENTIONAL
Beijing, China +11 more
Started: Apr 26, 2018
A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors
NCT02795767COMPLETEDPHASE3
88 participants
INTERVENTIONAL
Los Angeles, United States +27 more
Started: Jul 22, 2016
A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
NCT03191799COMPLETEDPHASE3
195 participants
INTERVENTIONAL
Camperdown, Australia +71 more
Started: Sep 5, 2017
Emicizumab in Acquired Hemophilia A
NCT04188639COMPLETEDPHASE2
47 participants
INTERVENTIONAL
Wien, Austria +15 more
Started: Mar 23, 2021
A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese Volunteers
NCT03380780COMPLETEDPHASE1
16 participants
INTERVENTIONAL
Beijing, China
Started: Mar 12, 2018
Outcomes of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast
NCT05279924COMPLETEDNA
33 participants
INTERVENTIONAL
Abidjan, Côte d’Ivoire
Started: Jul 1, 2021
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors
NCT02622321COMPLETEDPHASE3
113 participants
INTERVENTIONAL
Los Angeles, United States +43 more
Started: Nov 18, 2015
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
NCT02847637COMPLETEDPHASE3
152 participants
INTERVENTIONAL
Santa Monica, United States +38 more
Started: Sep 27, 2016
Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A
NCT05248594COMPLETED
71 participants
OBSERVATIONAL
Hartford, United States +9 more
Started: Aug 1, 2024
Showing 20 of 49 total trials