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Recombinant Factor VIII
Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A (XTEND-Kids Trial)
Phase 3
Waitlist Available
Research Sponsored by Bioverativ, a Sanofi company
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up pre-dose, 0.25, 3, 24, 72, and 168 hours post-dose on day 1
Awards & highlights
Pivotal Trial
No Placebo-Only Group
Summary
This trial is testing BIVV001, a new treatment for children with hemophilia A. It aims to help their blood clot better by providing a necessary protein. The study will check if the treatment is safe and effective over several months. BIVV001 is a new type of treatment with a longer-lasting effect compared to traditional options.
Eligible Conditions
- Hemophilia A
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ pre-dose, 0.25, 3, 24, 72, and 168 hours post-dose on day 1
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~pre-dose, 0.25, 3, 24, 72, and 168 hours post-dose on day 1
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Number of Participants With Neutralising Antibodies (Development of Inhibitors) Directed Against Factor VIII
Secondary study objectives
Annualized Bleeding Rate (ABR): For Treated Bleeds
Annualized Bleeding Rate by Location of Bleed (Joint, Muscle, Internal and Skin/Mucosa)
Annualized Bleeding Rate by Type of Bleed (Spontaneous, Traumatic and Unknown Type)
+44 moreSide effects data
From 2022 Phase 3 trial • 159 Patients • NCT0416149520%
Headache
19%
Arthralgia
8%
Fall
6%
Back Pain
5%
Fatigue
2%
Haemophilic Arthropathy
1%
Cd4 Lymphocytes Decreased
1%
Mobility Decreased
1%
Ulnar Tunnel Syndrome
1%
Basal Cell Carcinoma
1%
Combined Tibia-Fibula Fracture
1%
Arthropathy
1%
Blood Glucose Increased
1%
Cubital Tunnel Syndrome
1%
Status Epilepticus
1%
Angina Pectoris
1%
Traumatic Haemorrhage
1%
Central Venous Catheter Removal
1%
Device Breakage
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm A: Prophylaxis
Arm B: On-demand
Arm B: Prophylaxis
BIVV001 (Efanesoctocog Alfa): All Participants
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.
Trial Design
2Treatment groups
Experimental Treatment
Group I: BIVV001: Participants aged <6 YearsExperimental Treatment1 Intervention
Participants aged less than (\<) 6 years received BIVV001 at a dose of 50 international units per kilogram (IU/kg) intravenous (IV) injection once-weekly (QW) prophylaxis for 52 weeks.
Group II: BIVV001: Participants aged 6 to <12 YearsExperimental Treatment1 Intervention
Participants aged 6 to \<12 years received BIVV001 at a dose of 50 IU/kg IV injection QW prophylaxis for 52 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
efanesoctocog alfa (BIVV001)
2021
Completed Phase 3
~240
Find a Location
Who is running the clinical trial?
Bioverativ, a Sanofi companyLead Sponsor
17 Previous Clinical Trials
962 Total Patients Enrolled
6 Trials studying Hemophilia A
593 Patients Enrolled for Hemophilia A
Clinical Sciences & OperationsStudy DirectorSanofi
877 Previous Clinical Trials
2,022,742 Total Patients Enrolled
9 Trials studying Hemophilia A
1,128 Patients Enrolled for Hemophilia A