What side effects are associated with this type of intervention?

Common treatments for Hemophilia A, such as Emicizumab, factor VIII replacement, and Desmopressin (DDAVP), have distinct side effect profiles. Emicizumab is generally well-tolerated but carries a risk of unusual thrombotic events when used with aPCC. Factor VIII replacement can lead to the development of inhibitors, allergic reactions, and infusion-related reactions. Desmopressin may cause hyponatremia and fluid retention. Patients should discuss these potential side effects with their healthcare provider to determine the best treatment plan.

What prior FDA approvals exist?

The FDA has approved several treatments for Hemophilia A, including Emicizumab, a bispecific monoclonal antibody that bridges activated factor IX and factor X to promote blood clotting. Other notable FDA-approved treatments include recombinant factor VIII products such as rFVIII-Fc (Eloctate), which extends the half-life of factor VIII by fusing it with the Fc domain of human immunoglobulin, and PEGylated factor VIII products that also aim to prolong the therapeutic effect. These treatments enhance the management of Hemophilia A by reducing the frequency of dosing and improving patient outcomes.

What other types of research exist?

Promising novel alternatives to Emicizumab for Hemophilia A patients include: 1) Mim8, a factor VIIIa-mimetic bispecific antibody that has shown efficacy in preclinical studies. 2) Concizumab, an anti-TFPI monoclonal antibody, which has demonstrated safety and efficacy in phase 2 trials. 3) Gene therapy approaches, such as those targeting the delivery of functional factor VIII genes, which are in advanced stages of clinical development.

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Monoclonal Antibodies

Emicizumab for Hemophilia A (HAVEN 6 Trial)

Q: What is the mechanism of action of this treatment?

The most common treatments for Hemophilia A include factor replacement therapy, gene therapy, and newer agents like Emicizumab. Factor replacement therapy involves infusing the missing factor VIII directly into the bloodstream to aid in blood clotting. Gene therapy introduces functional genes to produce the missing clotting factor. Emicizumab, a bispecific monoclonal antibody, bridges activated factor IX and factor X to promote blood clotting, offering a different approach by mimicking the function of factor VIII. These treatments are essential for Hemophilia A patients as they help manage bleeding episodes, reduce the frequency of bleeding, and improve overall quality of life.

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

Diagnosis of mild (FVIII level between >5% and <40%) or moderate (FVIII level between ≥1% and ≤5%) congenital Hemophilia A without FVIII inhibitors

Must not have

Inherited or acquired bleeding disorder other than mild or moderate congenital hemophilia A

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline (week 1), weeks 13, 25, 37 and 49, and every 12 weeks thereafter until study completion/discontinuation visit (up to approximately 48 months)

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial tests emicizumab, a medication that helps blood clot, in people with mild or moderate hemophilia A. It works by mimicking a missing protein to improve blood clotting. Emicizumab has been approved for treatment of hemophilia A patients.

Who is the study for?

This trial is for individuals with mild or moderate Hemophilia A without inhibitors to factor VIII. Participants must weigh at least 3 kg, need prophylaxis, have documented treatment and bleeding episodes history, and proper organ function. Women of childbearing age should agree to use contraception.

What is being tested?

The study tests Emicizumab's safety, effectiveness, how the body processes it (pharmacokinetics), and its impact on the body (pharmacodynamics) in those with mild/moderate Hemophilia A who don't have FVIII inhibitors.

What are the potential side effects?

Potential side effects of Emicizumab may include reactions at the injection site, headaches, fatigue, muscle pain. Since it's designed for people with hemophilia, there might be a risk of developing antibodies against the drug that could reduce its effectiveness.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have mild or moderate Hemophilia A without inhibitors.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have a bleeding disorder that is not mild or moderate hemophilia A.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline (week 1), weeks 13, 25, 37 and 49, and every 12 weeks thereafter until study completion/discontinuation visit (up to approximately 48 months)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline (week 1), weeks 13, 25, 37 and 49, and every 12 weeks thereafter until study completion/discontinuation visit (up to approximately 48 months)

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (week 1), weeks 13, 25, 37 and 49, and every 12 weeks thereafter until study completion/discontinuation visit (up to approximately 48 months) 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 Bleed Rate for Treated Bleeds

Median Calculated Annualized Bleed Rate for Treated Bleeds

Model-Based Annualized Bleed Rate for Treated Bleeds

Secondary study objectives

CATCH Questionnaire for Adult Participants: Change From Baseline in the Daily Activity Risk Perception and Impact Domain Scores Over Time

CATCH Questionnaire for Adult Participants: Change From Baseline in the Preoccupation Domain Score Over Time

CATCH Questionnaire for Adult Participants: Change From Baseline in the Recreational Activity Risk Perception and Impact Domain Scores Over Time

+35 more

Side effects data

From 2022 Phase 3 trial • 48 Patients • NCT03020160

57%

ARTHRALGIA

57%

HEADACHE

43%

OSTEOARTHRITIS

43%

SYNOVITIS

29%

BACK PAIN

29%

ODYNOPHAGIA

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%

MUSCULOSKELETAL CHEST PAIN

14%

EXOSTOSIS

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%

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

Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors will be enrolled to receive the emicizumab loading dose regimen followed by the participant's preference of one of 3 maintenance dose regimens.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Emicizumab

2016

Completed Phase 3

~620

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Hemophilia A include factor replacement therapy, gene therapy, and newer agents like Emicizumab. Factor replacement therapy involves infusing the missing factor VIII directly into the bloodstream to aid in blood clotting. Gene therapy introduces functional genes to produce the missing clotting factor. Emicizumab, a bispecific monoclonal antibody, bridges activated factor IX and factor X to promote blood clotting, offering a different approach by mimicking the function of factor VIII. These treatments are essential for Hemophilia A patients as they help manage bleeding episodes, reduce the frequency of bleeding, and improve overall quality of life.