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Monoclonal Antibodies
Emicizumab for Hemophilia A (Beyond ABR Trial)
Phase 4
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 severe congenital hemophilia A (intrinsic factor VIII [FVIII] level <1%) or moderate congenital hemophilia A (intrinsic FVIII level ≤5%) if previously prescribed prophylaxis
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)
Awards & highlights
Beyond ABR Trial Summary
This trial will look at the effect of a new drug, emicizumab, on people with severe hemophilia A. The goal is to see if it improves overall health, physical activity, and joint outcomes.
Who is the study for?
This trial is for individuals aged 13-69 with severe or moderate Hemophilia A without Factor VIII inhibitors. They must have no history of FVIII inhibitory antibodies in the last 5 years, adequate organ function, and agree to use contraception if applicable. Excluded are those planning major joint procedures, with uncontrolled diseases like cardiovascular issues or HIV not managed by medication, or who've had certain treatments that might interfere with the study.Check my eligibility
What is being tested?
The trial is testing Emicizumab prophylaxis on overall health, physical activity, and joint outcomes in participants. It's an open-label Phase IV study where all participants receive Emicizumab to see how it affects their condition compared to previous standard treatments they were receiving.See study design
What are the potential side effects?
Emicizumab may cause injection site reactions, headaches, fatigue, muscle pain. Rare but serious side effects include blood clots and allergic reactions. Participants will be monitored closely for any adverse effects throughout the study.
Beyond ABR Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have severe or moderate hemophilia A and was on prophylaxis if moderate.
Beyond ABR Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ from baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from Baseline in the Average Daily Time Spent Doing Physical Activities by Intensity Level Over Time, as Assessed by Participant Responses to the International Physical Activity Questionnaire Short Format (IPAQ-SF)
Change from Baseline in the CATCH Domain Scores Over Time, as Assessed with the CATCH Questionnaire for Pediatric Participants
Change from Baseline in the CATCH Domain Scores Over Time, as Assessed with the Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire for Adult Participants
+25 moreSecondary outcome measures
Number of Participants who Develop Anti-FVIII Inhibitors During the Study
Number of Participants with Anti-Drug Antibodies (ADAs) Against Emicizumab at Baseline and During the Study
Number of Participants with at Least One Adverse Event, with Severity Determined According to the World Health Organization (WHO) Toxicity Scale
+4 moreSide effects data
From 2022 Phase 3 trial • 48 Patients • NCT0302016057%
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%
MYALGIA
14%
COMPLICATION ASSOCIATED WITH DEVICE
14%
MUSCULOSKELETAL CHEST PAIN
14%
EXOSTOSIS
14%
GREATER TROCHANTERIC PAIN SYNDROME
14%
TOOTHACHE
14%
COVID-19
14%
JOINT CONTRACTURE
14%
SUBCUTANEOUS ABSCESS
14%
HEAD INJURY
14%
DEVICE BREAKAGE
14%
JOINT LOCK
14%
FALL
14%
TONGUE INJURY
14%
CHEST PAIN
14%
ARTHRITIS
14%
TINEA CAPITIS
14%
CHOLELITHIASIS OBSTRUCTIVE
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%
0%
Study treatment Arm
Emicizumab: PK Run-In Cohort
Emicizumab: Expansion Cohort
Beyond ABR Trial Design
3Treatment groups
Experimental Treatment
Group I: Cohort 3, Hemophilia A and with Osteochondral Damage: EmicizumabExperimental Treatment1 Intervention
Cohort 3 comprises participants with severe or moderate hemophilia A and with osteochondral damage (HEAD-US bone and cartilage score of ≥1) in at least one index joint and with any synovitis score.
Group II: Cohort 2, Hemophilia A and with Synovitis Only: EmicizumabExperimental Treatment1 Intervention
Cohort 2 comprises participants with severe or moderate hemophilia A and with synovitis (HEAD-US synovitis score of ≥1) in at least one index joint and no osteochondral damage (HEAD-US bone and cartilage score of 0).
Group III: Cohort 1, Hemophilia A and Without Arthropathy: EmicizumabExperimental Treatment1 Intervention
Cohort 1 comprises participants with severe or moderate hemophilia A and with no synovitis and no osteochondral damage (Haemophilia Early Arthropathy Detection with Ultrasound [HEAD-US] score of 0) in all index joints.
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
2,430 Previous Clinical Trials
1,089,286 Total Patients Enrolled
13 Trials studying Hemophilia A
1,240 Patients Enrolled for Hemophilia A
Clinical TrialsStudy DirectorHoffmann-La Roche
2,200 Previous Clinical Trials
888,386 Total Patients Enrolled
14 Trials studying Hemophilia A
1,207 Patients Enrolled for Hemophilia A
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My HIV is not controlled by medication.I have a bleeding disorder but not severe or moderate hemophilia A without previous long-term treatment.I had a joint surgery less than 2 years ago or more than 3 years ago with ongoing pain.I am scheduled for surgery during the initial phase of emicizumab treatment.My blood, liver, and kidney functions are all within normal ranges.I have been treated with emicizumab before.I have low bone density due to a significant vitamin D deficiency.My heart condition is not stable and I'm not on any specific medication for it.I have conditions affecting my joints or mobility not related to hemophilia A.I am planning to undergo a joint replacement or procedure soon.I have a condition that could increase my risk of bleeding or blood clots.I have severe or moderate hemophilia A and was on prophylaxis if moderate.I have been on regular FVIII treatment to prevent bleeding for the last 24 weeks.I haven't taken any experimental drugs for hemophilia or other conditions recently.I have been treated for blood clots in the past year, except for catheter-related ones.I haven't had FVIII inhibitory antibodies in the last 5 years.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 1, Hemophilia A and Without Arthropathy: Emicizumab
- Group 2: Cohort 2, Hemophilia A and with Synovitis Only: Emicizumab
- Group 3: Cohort 3, Hemophilia A and with Osteochondral Damage: Emicizumab
Awards:
This trial has 3 awards, including:- Drug Has Already Been Approved - The FDA has already approved this drug, and is just seeking more data.
- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is this an innovative exploration into treatments?
"Currently, there are 9 ongoing trials of Emicizumab spanning 22 countries and 56 cities. The pioneering research on the drug was completed by Hoffmann-La Roche in 2018 with 85 participants. Since then, a further 7 studies were conducted to support its Phase 3 drug approval status."
Answered by AI
Has the FDA accepted Emicizumab for clinical use?
"Emicizumab's safety has been approved by Phase 4 trials, so it was awarded a rating of 3."
Answered by AI
What is the scale of recruitment for this experiment?
"Hoffmann-La Roche is administering this trial with the aim of recruiting 120 patients that meet the criteria. The study will be conducted from two different sites, Oklahoma Children's Hospital - Jimmy Everest Center in Las Vegas and Hemostasis & Thrombosis Center of Nevada (HTCNV) located in Coral Gables."
Answered by AI
Is enrollment still available for the study?
"Affirmative. According to clinicaltrials.gov, this research trial launched on June 20th 2022 and is currently enrolling subjects. Seven sites are collaborating in the attempt to recruit 120 individuals for participation."
Answered by AI
What criteria must prospective participants fulfill to be eligible for this clinical trial?
"This study seeks 120 individuals suffering from hemophilia A, aged 13 to 69 years old. Most importantly, applicants must meet the following conditions: diagnosed with severe (FVIII <1%) or moderate (FVIII ≤5%) congenital haemophilia; no inhibitor in last 5 yrs.; successful immune tolerance induction at least 5 yr prior w/o recurrence of inhibitors; stable FVIII prophylaxis for min 24 weeks before entry; adequate hematologic, hepatic & renal functions and women of childbearing potential agree to contraception or abstinence during emicizumab treatment period + 24 weeks after"
Answered by AI
Does the current research include any participants above the age of seventy-five?
"This trial requires potential participants to be between 13 and 69 years old."
Answered by AI
How many healthcare facilities are facilitating this clinical trial?
"Participants can join this experiment at Oklahoma Children's Hospital - Jimmy Everest Center in Las Vegas, Hemostasis & Thrombosis Center of Nevada (HTCNV) in Coral Gables, or University of Miami in New Hyde Park. In addition to these three sites, there are 7 other enrolment hubs across the country."
Answered by AI
What other empirical investigations have been conducted involving Emicizumab?
"Since its initial research at Xiangya Hospital of Centre-South University in 2018, emicizumab has been subject to 7 completed clinical trials. Additionally, there are currently 9 active studies taking place with numerous sites located within the Las Vegas area of Nevada."
Answered by AI
Who else is applying?
What state do they live in?
Oklahoma
How old are they?
18 - 65
What site did they apply to?
Hemostasis & Thrombosis Center of Nevada (HTCNV)
What portion of applicants met pre-screening criteria?
Met criteria
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