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Monoclonal Antibodies

Emicizumab for Hemophilia A (HAVEN 7 Trial)

Phase 3
Waitlist Available
Research Sponsored by Hoffmann-La Roche
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Documentation of hemophilia-related treatments received since birth
Mandatory receipt of vitamin K prophylaxis according to local standard practice
Must not have
Current active severe bleed such as intracranial hemorrhage
Hereditary or acquired maternal conditions predisposing to thrombotic events
Timeline
Screening 3 weeks
Treatment Varies
Follow Up as clinically indicated from baseline until study completion (up to 8 years)
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is testing a new medicine for babies with severe hemophilia A. The medicine will be given once every 2 weeks for a year, and then once a week, every 2 weeks, or every 4 weeks for 7 more years.

Who is the study for?
This trial is for babies from birth to 12 months with severe Hemophilia A (FVIII level <1%) without inhibitors. They must be previously untreated or minimally treated, weigh at least 3 kg, have no history of FVIII inhibitor or thrombotic disease, and not be infected with HIV or hepatitis. Parents must commit to the study's requirements.
What is being tested?
The study tests Emicizumab's effectiveness and safety in preventing bleeding episodes in young children with Hemophilia A. It involves a weekly injection for one year followed by a long-term follow-up period where dosage frequency may vary.
What are the potential side effects?
Potential side effects include reactions at the injection site, headaches, joint pain, general discomfort, and possibly an increased risk of developing blood clots or experiencing abnormal bleeding.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have records of all hemophilia treatments I've received since birth.
Select...
I have received vitamin K as per local health guidelines.
Select...
I have severe hemophilia A with very low factor VIII levels.
Select...
I don't have a history of FVIII inhibitor, my FVIII lasts over 6 hours, and recovers well.
Select...
I am a baby aged 12 months or younger.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I do not have any severe bleeding, like a brain bleed.
Select...
I have a genetic or acquired condition that increases my risk of blood clots.
Select...
I have a condition that increases my risk of bleeding or blood clots.
Select...
I am not planning any major surgeries during the study.
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I have a bleeding disorder that is not severe hemophilia A.
Select...
I am at high risk for blood clotting disorders.
Select...
I am not using, nor plan to use, immune system altering drugs during the study.
Select...
I have not had a serious infection needing antibiotics or antivirals in the last 14 days.
Select...
I have been treated for blood clots before.
Select...
I am not willing to receive blood products or standard treatments.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~as clinically indicated from baseline until study completion (up to 8 years)
This trial's timeline: 3 weeks for screening, Varies for treatment, and as clinically indicated from baseline until study completion (up to 8 years) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Mean Calculated Annualized Bleeding Rate for All Bleeds
Mean Calculated Annualized Bleeding Rate for Treated Bleeds
Mean Calculated Annualized Bleeding Rate for Treated Joint Bleeds
+9 more
Secondary study objectives
Incidence of De Novo Development of Factor VIII Inhibitors
Number of Participants According to Hematology and Serum Chemistry Laboratory Test Result Shifts From Baseline WHO Grade to the Highest WHO Grade Post-Baseline

Side effects data

From 2022 Phase 3 trial • 48 Patients • NCT03020160
57%
ARTHRALGIA
57%
HEADACHE
43%
OSTEOARTHRITIS
43%
SYNOVITIS
29%
ODYNOPHAGIA
29%
BACK PAIN
29%
PHARYNGITIS
29%
CONTUSION
29%
EAR INFECTION
29%
UPPER RESPIRATORY TRACT INFECTION
29%
URINARY TRACT INFECTION
29%
HYPERTENSION
29%
Arthralgia
29%
Osteoarthritis
29%
Headache
29%
Upper respiratory tract infection
29%
Back pain
29%
ABDOMINAL PAIN
14%
EXOSTOSIS
14%
MUSCULOSKELETAL CHEST PAIN
14%
COVID-19
14%
SUBCUTANEOUS ABSCESS
14%
JOINT LOCK
14%
TONGUE INJURY
14%
COMPLICATION ASSOCIATED WITH DEVICE
14%
JOINT CONTRACTURE
14%
HEAD INJURY
14%
GREATER TROCHANTERIC PAIN SYNDROME
14%
Injection site reaction
14%
TINEA CAPITIS
14%
FALL
14%
GINGIVAL INJURY
14%
CHEST PAIN
14%
CHOLELITHIASIS OBSTRUCTIVE
14%
TOOTHACHE
14%
ARTHRITIS
14%
DEVICE BREAKAGE
14%
MYALGIA
14%
PARAESTHESIA
14%
MEDICAL DEVICE DISCOMFORT
14%
POST PROCEDURAL INFLAMMATION
14%
ANXIETY
14%
RASH
14%
Temporomandibular joint syndrome
14%
Post procedural inflammation
14%
INFLAMMATION
14%
INJECTION SITE REACTION
14%
Dyspepsia
14%
Device related infection
14%
Contusion
14%
Synovitis
14%
Tendon disorder
14%
Joint lock
14%
Cholelithiasis
14%
Myalgia
14%
HYPERCHOLESTEROLAEMIA
14%
VITAMIN D DEFICIENCY
14%
Abdominal pain
14%
Tongue injury
14%
Eczema eyelids
14%
Ear infection
14%
Osteitis
14%
Pharyngitis
14%
Hypertension
14%
Musculoskeletal chest pain
14%
IRON DEFICIENCY ANAEMIA
14%
ECZEMA EYELIDS
14%
DIARRHOEA
14%
DYSPEPSIA
14%
PYREXIA
14%
SEASONAL ALLERGY
14%
DEVICE RELATED INFECTION
14%
SPINAL OSTEOARTHRITIS
14%
TEMPOROMANDIBULAR JOINT SYNDROME
14%
TENDON DISORDER
14%
MOTOR DYSFUNCTION
100%
80%
60%
40%
20%
0%
Study treatment Arm
Emicizumab: PK Run-In Cohort
Emicizumab: Expansion Cohort

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: EmicizumabExperimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Emicizumab
2016
Completed Phase 3
~620

Find a Location

Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor
2,463 Previous Clinical Trials
1,103,109 Total Patients Enrolled
13 Trials studying Hemophilia A
1,317 Patients Enrolled for Hemophilia A
Clinical TrialsStudy DirectorHoffmann-La Roche
2,233 Previous Clinical Trials
902,238 Total Patients Enrolled
14 Trials studying Hemophilia A
1,284 Patients Enrolled for Hemophilia A

Media Library

Emicizumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT04431726 — Phase 3
Hemophilia A Research Study Groups: Emicizumab
Hemophilia A Clinical Trial 2023: Emicizumab Highlights & Side Effects. Trial Name: NCT04431726 — Phase 3
Emicizumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04431726 — Phase 3
~11 spots leftby Dec 2025
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