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Monoclonal Antibodies

Hemlibra for Mild Hemophilia A

Phase 4

Recruiting

Led By Amy D Shapiro, MD

Research Sponsored by Indiana Hemophilia &Thrombosis Center, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up before treatment, month 4, month 7, and month 13

Awards & highlights

Pivotal Trial

No Placebo-Only Group

All Individual Drugs Already Approved

Drug Has Already Been Approved

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.

Who is the study for?

This trial is for males aged 5-45 with mild congenital hemophilia A (FVIII level >5% to 30%), without FVIII inhibitors or history of them. Participants must have documented bleeding events and be willing to undergo specific challenges if they've had no prior adverse reactions. Exclusions include previous emicizumab use, other investigational drugs recently, certain cardiovascular risks, additional bleeding disorders, drug/alcohol abuse, and significant hypersensitivity to monoclonal antibodies.

What is being tested?

The study tests Hemlibra (emicizumab) in a single-arm phase 4 trial focusing on its blood clotting effects in mild hemophilia A patients. It aims to determine safety and effectiveness by monitoring coagulation lab parameters, changes in joint health, and quality of life improvements.

What are the potential side effects?

Potential side effects may include allergic reactions related to the injection components or general issues associated with monoclonal antibody therapies such as infusion-related reactions. Specific side effect profiles for emicizumab will be monitored throughout the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ before treatment, month 4, month 7, and month 13

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~before treatment, month 4, month 7, and month 13

This trial's timeline: 3 weeks for screening, Varies for treatment, and before treatment, month 4, month 7, and month 13 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Secondary study objectives

ADA development

AE, SAE, and ADA

Alternate hemostatic therapies with surgery

+5 more

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%

GREATER TROCHANTERIC PAIN SYNDROME

14%

EXOSTOSIS

14%

COMPLICATION ASSOCIATED WITH DEVICE

14%

MUSCULOSKELETAL CHEST PAIN

14%

COVID-19

14%

JOINT LOCK

14%

SUBCUTANEOUS ABSCESS

14%

TONGUE INJURY

14%

JOINT CONTRACTURE

14%

HEAD INJURY

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%

Study treatment Arm

Emicizumab: PK Run-In Cohort

Emicizumab: Expansion Cohort

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Single ArmExperimental Treatment1 Intervention

Patients with mild hemophilia A (without inhibitors) will be treated with prophylactic emicizumab. The clinical hemostatic efficacy and safety will be assessed. Secondary outcomes will assess changes in quality of life and joint health in treated patients.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Emicizumab

FDA approved

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