Emicizumab for Hemophilia A

Phase-Based Progress Estimates
3
Effectiveness
3
Safety
Hemophilia AEmicizumab - Drug
Eligibility
5 - 45
Male
What conditions do you have?
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Study Summary

This trial will study the safety and effectiveness of Hemlibra in mild hemophilia A patients with altered FVIII. Secondary outcomes will assess changes in joint health and quality of life.

Eligible Conditions
  • Hemophilia A

Treatment Effectiveness

Effectiveness Progress

3 of 3
This is further along than 93% of similar trials

Study Objectives

1 Primary · 8 Secondary · Reporting Duration: Before treatment, month 4, month 7, and month 13

Month 13
ADA development
At time of patient's surgery if applicable
Alternate hemostatic therapies with surgery
Month 25
Change in activity: questionnaire
Change in quality of life: questionnaire
Month 13
Factor VIII alteration and coagulation
Interaction of Hemlibra (emicizumab) binding with endogenous altered FVIII protein in an individual with mild hemophilia A and the combined effect on thrombin generation and hemostatic characteristics
Month 35
AE, SAE, and ADA
Change from baseline joint disease annually
Month 35
Breakthrough bleeds

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Side Effects for

Emicizumab: Expansion Cohort
41%ARTHRALGIA
34%NASOPHARYNGITIS
29%HEADACHE
27%Nasopharyngitis
22%Injection site reaction
22%INJECTION SITE REACTION
20%Arthralgia
15%JOINT INJURY
15%BACK PAIN
15%COVID-19
15%PAIN IN EXTREMITY
12%TOOTHACHE
12%Headache
10%PYREXIA
10%INFLUENZA
10%HYPERTENSION
10%Pain in extremity
7%UPPER RESPIRATORY TRACT INFECTION
7%PAIN
7%FALL
7%LIMB INJURY
7%NECK PAIN
7%LIGAMENT SPRAIN
7%Upper respiratory tract infection
5%ANXIETY
5%ABDOMINAL PAIN
5%HYPERCHOLESTEROLAEMIA
5%SYNOVITIS
5%RASH
5%HEAD INJURY
5%DIARRHOEA
5%MYALGIA
5%Fatigue
5%Pyrexia
5%Neck pain
5%Dizziness
5%Synovitis
5%Nephrolithiasis
2%CELLULITIS
2%RHABDOMYOLYSIS
2%TUMOUR COMPRESSION
2%VITAMIN D DEFICIENCY
2%ARTHRITIS
2%Otitis media
2%BLADDER NEOPLASM
2%CHEST PAIN
2%OSTEOARTHRITIS
2%ATRIAL FIBRILLATION
2%MUSCULOSKELETAL CHEST PAIN
2%Insomnia
2%DIFFUSE LARGE B-CELL LYMPHOMA
2%DEVICE LOOSENING
2%SEASONAL ALLERGY
2%URINARY TRACT INFECTION
2%NEPHROLITHIASIS
2%HAEMORRHAGE
2%PARAESTHESIA
2%Thrombocytopenia
2%Chronic sinusitis
2%Urinary tract infection
2%Pain in jaw
2%Ligament sprain
2%Diarrhoea
2%Chills
2%Road traffic accident
2%Aneurysm
2%Erythema nodosum
2%Gastroenteritis viral
2%Muscle contracture
2%Liver disorder
2%Goitre
2%Faeces discoloured
2%Rhabdomyolysis
2%Injection site pain
2%Splenomegaly
2%Joint injury
2%Oral herpes
2%Fall
2%Iron deficiency
2%Hypoaesthesia
2%Bronchospasm
2%Cough
2%Pain
2%Wound
2%Venomous sting
2%Sleep disorder
2%Hyperglycaemia
2%Atrioventricular block first degree
2%Benign prostatic hyperplasia
2%Cataract
2%Rash
2%Vitamin B12 deficiency
2%Asthenia
2%Presyncope
2%Gastroenteritis
2%Genital tract inflammation
2%Erythema
2%Oropharyngeal pain
2%Laceration
2%Muscle strain
2%Rhinorrhea
2%Dysuria
2%Psoriasis
2%Glycosylated haemoglobin increased
2%Myalgia
2%Dermatitis
2%Malaise
2%Dental caries
2%Hydronephrosis
2%Back pain
2%Aphthous ulcer
This histogram enumerates side effects from a completed 2022 Phase 3 trial (NCT03020160) in the Emicizumab: Expansion Cohort ARM group. Side effects include: ARTHRALGIA with 41%, NASOPHARYNGITIS with 34%, HEADACHE with 29%, Nasopharyngitis with 27%, Injection site reaction with 22%.

Trial Design

1 Treatment Group

Single Arm
1 of 1

Experimental Treatment

40 Total Participants · 1 Treatment Group

Primary Treatment: Emicizumab · No Placebo Group · Phase 4

Single Arm
Drug
Experimental Group · 1 Intervention: Emicizumab · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Emicizumab
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: before treatment, month 4, month 7, and month 13

Who is running the clinical trial?

Indiana Hemophilia &Thrombosis Center, Inc.Lead Sponsor
5 Previous Clinical Trials
666 Total Patients Enrolled
Genentech, Inc.Industry Sponsor
1,497 Previous Clinical Trials
564,619 Total Patients Enrolled
14 Trials studying Hemophilia A
850 Patients Enrolled for Hemophilia A
Amy D Shapiro, MDPrincipal InvestigatorIndiana Hemophilia &Thrombosis Center, Inc.
4 Previous Clinical Trials
666 Total Patients Enrolled

Eligibility Criteria

Age 5 - 45 · Male Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have medical documentation of bleeding events, outcomes and hemostatic product usage for 12 months prior to study enrollment.
You are willing and able to comply with the study procedures, including the health-related questionnaires, activity tracking, and bleed diaries, using systems provided during the study.
References