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Factor VIII Replacement

Emicizumab for Hemophilia A

Phase 3
Waitlist Available
Led By Margaret V Ragni, MD, MPH
Research Sponsored by Margaret Ragni
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 48 weeks
Awards & highlights

Study Summary

This trial will compare two treatments to see which is better at preventing inhibitors (a protein that stops blood from clotting) in people with severe hemophilia A.

Eligible Conditions
  • Hemophilia A

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~48 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 48 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Inhibitor Formation
Secondary outcome measures
Bleeding Events
FVIII Mutation
FVIII Trough Level
+2 more

Side effects data

From 2014 Phase 2 trial • 21 Patients • NCT00560794
100%
Pyrexia
67%
Blood immunoglobulin A decreased
62%
Blood immunoglobulin G decreased
48%
Blood immunoglobulin M decreased
48%
Hypokalaemia
48%
Headache
43%
Chills
38%
Fatigue
38%
Oedema peripheral
33%
Nasopharyngitis
33%
Leukopenia
33%
Weight increased
29%
Back pain
29%
Thrombocytopenia
29%
C-reactive protein increased
29%
Lymphopenia
24%
Growing pains
24%
Diarrhoea
24%
Dizziness
24%
Haematuria
24%
Nausea
24%
Gamma-glutamyltransferase increased
24%
Tremor
24%
Insomnia
24%
Dyspnoea
24%
Rash
24%
Hypotension
19%
Alanine aminotransferase increased
19%
Hyperglycaemia
19%
Constipation
19%
Fibrin D dimer increased
19%
Tachycardia
19%
Weight decreased
19%
Periorbital oedema
14%
Cough
14%
Vomiting
14%
Blood potassium decreased
14%
Hyperhidrosis
10%
Monocyte count increased
10%
Neck pain
10%
Oral herpes
10%
Cystitis
10%
Arthralgia
10%
Sinusitis
10%
Myalgia
10%
Pharyngitis
10%
Blood alkaline phosphatase increased
10%
Pain in extremity
10%
Bradycardia
10%
Lacrimation increased
10%
Dry mouth
10%
Catheter site erythema
10%
Catheter site pain
10%
Bronchitis
10%
Coagulation factor XIII level increased
10%
Immunoglobulins decreased
10%
Anorexia
10%
Immunodeficiency
10%
Blood lactate dehydrogenase increased
10%
Night sweats
10%
Hypertension
5%
Convulsion
5%
Somnolence
5%
Epilepsy
5%
Syncope
5%
Bacterial sepsis
5%
Bronchopneumonia
5%
Catheter related infection
5%
Escherichia sepsis
5%
Medical device complication
5%
Thrombosis in device
100%
80%
60%
40%
20%
0%
Study treatment Arm
Blinatumomab

Trial Design

2Treatment groups
Experimental Treatment
Active Control
Group I: EmicizumabExperimental Treatment1 Intervention
Arm B: Emicizumab 1.5 mg/kg will be administered weekly by subcutaneous injection (following 3 mg/kg/wk x4 induction) in previously untreated children with severe hemophilia A beginning before the first bleed and continue up to 48 weeks.
Group II: EloctateActive Control1 Intervention
Arm A: Eloctate 65 IU/kg will be administered weekly by intravenous infusion in previously untreated children with severe hemophilia A beginning before the first bleed and continued up to 48 weeks.

Find a Location

Who is running the clinical trial?

Health Resources and Services Administration (HRSA)FED
84 Previous Clinical Trials
151,486 Total Patients Enrolled
1 Trials studying Hemophilia A
1 Patients Enrolled for Hemophilia A
Margaret RagniLead Sponsor
6 Previous Clinical Trials
67 Total Patients Enrolled
2 Trials studying Hemophilia A
1 Patients Enrolled for Hemophilia A
Margaret V Ragni, MD, MPHPrincipal InvestigatorUniversity of Pittsburgh
5 Previous Clinical Trials
60 Total Patients Enrolled
2 Trials studying Hemophilia A
1 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.
Recent research and studies
clinicaltrials.govStudy
The Hemophilia Inhibitor Prevention Trial
Margaret Ragni 2021. "The Hemophilia Inhibitor Prevention Trial". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04303559.
~0 spots leftby Apr 2025
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