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Emicizumab for Hemophilia A

Phase-Based Progress Estimates
3
Effectiveness
3
Safety
The Jimmy Everest Center for Cancer & Blood Disorders in Children, Oklahoma City, OKHemophilia A+1 MoreEmicizumab - Drug
Eligibility
13 - 69
All Sexes
What conditions do you have?
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Study 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.

Eligible Conditions
  • Hemophilia A
  • Moderate Hemophilia A
  • Severe Hemophilia A

Treatment Effectiveness

Effectiveness Progress

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

Similar Trials

1
Factor IX
1
SPK-8011
1
emicizumab

Study Objectives

28 Primary · 7 Secondary · Reporting Duration: From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)

12 Months
Clinical Joint Status at 12 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment
Joint Status at 12 Months, Based on Centrally Reviewed Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) Scores with a Specific Focus on the Synovitis Score in Participants with Synovitis
Number of Problem Joints at 12 Months
Percentage of Joints That are Problem Joints at 12 Months
24 Months
Clinical Joint Status at 24 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment
Joint Status at 24 Months, Based on Centrally Reviewed Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) Scores with a Specific Focus on the Synovitis Score in Participants with Synovitis
Number of Problem Joints at 24 Months
Percentage of Joints That are Problem Joints at 24 Months
36 Months
Clinical Joint Status at 36 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment
Joint Status at 36 Months, Based on Centrally Reviewed Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) Scores with a Specific Focus on the Synovitis Score in Participants with Synovitis
Joint Status at 36 Months, Based on Centrally Reviewed International Prophylaxis Study Group (IPSG) Score (with MRI)
Number of Problem Joints at 36 Months
Percentage of Joints That are Problem Joints at 36 Months
6 Months
Clinical Joint Status at 6 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment
Joint Status at 6 Months, Based on Centrally Reviewed Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) Scores with a Specific Focus on the Synovitis Score in Participants with Synovitis
Number of Problem Joints at 6 Months
Percentage of Joints That are Problem Joints at 6 Months
Month 3
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
Month 6
Number of Participants with Anti-Drug Antibodies (ADAs) Against Emicizumab at Baseline and During the Study
At Month 6
Number of Participants who Prefer Emicizumab SC Treatment, Their Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Month 6
Month 6
Number of Participants who Develop Anti-FVIII Inhibitors During the Study
Year 5
Number of Participants with at Least One Adverse Event, with Severity Determined According to the World Health Organization (WHO) Toxicity Scale
Number of Participants with at Least One Event of Thrombotic Microangiopathy (TMA)
Number of Participants with at Least One Injection-Site Reaction
Number of Participants with at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event
Number of Participants with at Least One Thromboembolic Event
Month 36
Daily Active Minutes of Physical Activity Over Time, as Measured with a Wearable Activity Tracker
Daily Metabolic Equivalents of Tasks (METs) Over Time, as Measured with a Wearable Activity Tracker
Daily Step Count Over Time, as Measured with a Wearable Activity Tracker
Daily Time Spent in Moderate to Vigorous Physical Activity (MVPA) Over Time, as per the Activity Tracker Default Categorization
Mean Calculated Annualized Bleed Rates for All Bleeds, Treated Bleeds, Spontaneous Bleeds, Joint Bleeds, Treated Joint Bleeds, and Target Joint Bleeds
Median Calculated Annualized Bleed Rates for All Bleeds, Treated Bleeds, Spontaneous Bleeds, Joint Bleeds, Treated Joint Bleeds, and Target Joint Bleeds
Model-Based Annualized Bleed Rates for All Bleeds, Treated Bleeds, Spontaneous Bleeds, Joint Bleeds, Treated Joint Bleeds, and Target Joint Bleeds

Trial Safety

Safety Progress

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

Similar Trials

1
Factor IX
1
SPK-8011
2
emicizumab

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%BACK PAIN
15%COVID-19
15%JOINT INJURY
15%PAIN IN EXTREMITY
12%Headache
12%TOOTHACHE
10%INFLUENZA
10%PYREXIA
10%HYPERTENSION
10%Pain in extremity
7%LIGAMENT SPRAIN
7%NECK PAIN
7%PAIN
7%LIMB INJURY
7%FALL
7%UPPER RESPIRATORY TRACT INFECTION
7%Upper respiratory tract infection
5%ABDOMINAL PAIN
5%DIARRHOEA
5%HYPERCHOLESTEROLAEMIA
5%SYNOVITIS
5%RASH
5%HEAD INJURY
5%MYALGIA
5%ANXIETY
5%Fatigue
5%Pyrexia
5%Neck pain
5%Dizziness
5%Synovitis
5%Nephrolithiasis
2%Otitis media
2%BLADDER NEOPLASM
2%TUMOUR COMPRESSION
2%Insomnia
2%DEVICE LOOSENING
2%DIFFUSE LARGE B-CELL LYMPHOMA
2%CHEST PAIN
2%VITAMIN D DEFICIENCY
2%OSTEOARTHRITIS
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%ATRIAL FIBRILLATION
2%MUSCULOSKELETAL CHEST PAIN
2%Splenomegaly
2%Joint injury
2%Oral herpes
2%HAEMORRHAGE
2%Fall
2%SEASONAL ALLERGY
2%ARTHRITIS
2%PARAESTHESIA
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%CELLULITIS
2%RHABDOMYOLYSIS
2%NEPHROLITHIASIS
2%URINARY TRACT INFECTION
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

3 Treatment Groups

Cohort 2, Hemophilia A and with Synovitis Only: Emicizumab
1 of 3
Cohort 1, Hemophilia A and Without Arthropathy: Emicizumab
1 of 3
Cohort 3, Hemophilia A and with Osteochondral Damage: Emicizumab
1 of 3

Experimental Treatment

120 Total Participants · 3 Treatment Groups

Primary Treatment: Emicizumab · No Placebo Group · Phase 4

Cohort 2, Hemophilia A and with Synovitis Only: Emicizumab
Drug
Experimental Group · 1 Intervention: Emicizumab · Intervention Types: Drug
Cohort 1, Hemophilia A and Without Arthropathy: Emicizumab
Drug
Experimental Group · 1 Intervention: Emicizumab · Intervention Types: Drug
Cohort 3, Hemophilia A and with Osteochondral Damage: Emicizumab
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: from baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)

Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor
2,371 Previous Clinical Trials
1,068,494 Total Patients Enrolled
1 Trials studying Severe Hemophilia A
55 Patients Enrolled for Severe Hemophilia A
Clinical TrialsStudy DirectorHoffmann-La Roche
2,152 Previous Clinical Trials
876,134 Total Patients Enrolled
1 Trials studying Severe Hemophilia A
55 Patients Enrolled for Severe Hemophilia A

Eligibility Criteria

Age 13 - 69 · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:

Who else is applying?

What state do they live in?
Oklahoma100.0%
How old are they?
18 - 65100.0%
What site did they apply to?
Hemostasis & Thrombosis Center of Nevada (HTCNV)100.0%
What portion of applicants met pre-screening criteria?
Met criteria100.0%
References

Frequently Asked Questions

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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" - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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