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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

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.

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.Check my eligibility

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.See study design

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

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%

BACK PAIN

29%

PHARYNGITIS

29%

ODYNOPHAGIA

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%

CHEST PAIN

14%

TOOTHACHE

14%

CHOLELITHIASIS OBSTRUCTIVE

14%

COVID-19

14%

MYALGIA

14%

COMPLICATION ASSOCIATED WITH DEVICE

14%

MUSCULOSKELETAL CHEST PAIN

14%

JOINT CONTRACTURE

14%

SUBCUTANEOUS ABSCESS

14%

HEAD INJURY

14%

DEVICE BREAKAGE

14%

FALL

14%

JOINT LOCK

14%

TONGUE INJURY

14%

EXOSTOSIS

14%

GREATER TROCHANTERIC PAIN SYNDROME

14%

ARTHRITIS

14%

TINEA CAPITIS

14%

GINGIVAL INJURY

14%

PARAESTHESIA

14%

Injection site reaction

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

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

Do I qualify?Apply below to find out

Find a Location

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

1,541 Previous Clinical Trials

568,123 Total Patients Enrolled

16 Trials studying Hemophilia A

3,840 Patients Enrolled for Hemophilia A

Amy D Shapiro, MDPrincipal InvestigatorIndiana Hemophilia &Thrombosis Center, Inc.

4 Previous Clinical Trials

666 Total Patients Enrolled

Media Library

Emicizumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT04567511 — Phase 4

Hemophilia A Research Study Groups: Single Arm

Hemophilia A Clinical Trial 2023: Emicizumab Highlights & Side Effects. Trial Name: NCT04567511 — Phase 4

Emicizumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04567511 — Phase 4

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many individuals have been enrolled in the research thus far?

"Affirmative, the data on clinicaltrials.gov reveals that this investigation is currently accepting participants. It was first posted on September 1st 2021 and last modified October 6th of the same year. The study requires 40 enrollees from a single research site."

Answered by AI

Has Emicizumab achieved regulatory authorization from the FDA?

"The safety rating assigned to Emicizumab is 3, as it has already been approved following Phase 4 clinical trials."

Answered by AI

What other empirical research has been conducted on the impact of Emicizumab?

"Presently, 9 clinical studies are ongoing regarding Emicizumab. Of those trials, 5 have entered the late stages of Phase 3. As a majority of these experiments occur in Dallas, Texas; there is also presence across 148 sites worldwide."

Answered by AI

Is this investigation a fresh attempt?

"First studied in 2018, Emicizumab has since developed a body of evidence via 85 participants sponsored by Hoffmann-La Roche. Subsequently the drug was approved for Phase 3 clinical trials and currently there are 9 ongoing assessments across 56 cities and 22 nations."

Answered by AI

Are there any current opportunities for individuals to join this trial?

"Affirmative. According to clinicaltrials.gov, this health investigation is still in search of participants since it was posted on September 1st 2021 and last updated October 6th 2021. 40 persons are need to be recruited from the single medical centre involved in the trial."

Answered by AI

Does this research endeavor include elderly individuals in its sample?

"This clinical trial demands that participants must be aged between 5 and 45, in accordance with the given eligibility requirements."

Answered by AI

To what demographic does the eligibility criteria for this experiment apply?

"This medical trial is recruiting 40 patients aged 5 to 45 with hemophilia A, provided they satisfy the following criteria: Medical records of bleeding episodes and hemostatic product use over the previous year, Compliance with all relevant visits, tests, treatments and questionnaires mentioned in this study's protocol., Male sex, Mild form of congenital haemophilia (FVIII level between >5-30%), BMI under 30 kg/m2 , Willingness for a Stimate or DDAVP challenge if no adverse events have been reported before it; The patient should also present an adequate hepatic function (bilirubin ≤"

Answered by AI

Recent research and studies

New England Journal of MedicineJournal

Emicizumab Prophylaxis in Hemophilia A with Inhibitors

Oldenburg, Johannes, Johnny N. Mahlangu, Benjamin Kim, Christophe Schmitt, Michael U. Callaghan, Guy Young, Elena Santagostino, et al.. 2017. “Emicizumab Prophylaxis in Hemophilia A with Inhibitors”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa1703068.

Journal of Thrombosis and HaemostasisJournal

Mild Hemophilia A

FRANCHINI, M., E. J. FAVALORO, and G. LIPPI. 2009. “Mild Hemophilia A”. Journal of Thrombosis and Haemostasis. Wiley. doi:10.1111/j.1538-7836.2009.03717.x.

British Journal of HaematologyJournal

Inhibitor Eradication with Rituximab in Haemophilia: Where Do We Stand?

Franchini, Massimo, and Pier Mannuccio Mannucci. 2014. “Inhibitor Eradication with Rituximab in Haemophilia: Where Do We Stand?”. British Journal of Haematology. Wiley. doi:10.1111/bjh.12829.

New England Journal of MedicineJournal