To evaluate the safety of BIVV001 in previously treated pediatric subjects with hemophilia A.
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
- Blood and lymphatic system disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Occurrence of inhibitor development
Secondary outcome
- Annualized bleeding rate (ABR), ABR by type of bleed and ABR by location of
bleed
- Percentage of participants who maintain FVIII activity above prespecified
levels
- Number of injection and dose of BIVV001 to treat a bleeding episode
- Percentage of bleeding episodes treated with a single injection of BIVV001
- Assessment of response to BIVV001 treatment of individual bleeding episodes
- Physician*s global assessment of the participant*s response based on BIVV001
treatment
- Total annualized BIVV001 consumption
- Annualized Joint Bleeding Rate (AJBR)
- Target joint resolution
- Change in Hemophilia Joint Health Score (HJHS) total score and domain scores
- Changes in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL)
total score and physical health domain scores from baseline to week 52
- Investigators* or Surgeons* assessment of participant*s hemostatic response
to BIVV001 treatment
- Number of injections and dose to maintain hemostasis during perioperative
period for major surgery
- Total BIVV001 consumption during perioperative period for major surgery
- Number of blood component transfusions used during perioperative period for
major surgery
- Type of blood component transfusions used during perioperative period for
major surgery
- Estimated blood loss during perioperative period for major surgery
- Number of participants with occurence of adverse events (AEs) and serious
adverse events (SAEs)
- Number of participants with occurrence of embolic and thrombotic events
- PK parameter: Maximum activity (Cmax), elimination half-life (t1/2), total
clearance (CL), total clearance at steady state (CLss), dose-normalized area
under the activity-time curve (DNAUC), area under the activity time curve
(AUC), volume of distribution at steady state (Vss), mean residence time (MRT),
incremental recovery (IR), trough activity (Ctrough), time above predefined
FVIII activity levels
Background summary
Hemophilia A is a congenital X-linked bleeding disorder that occurs
predominantly in males and is characterized by deficiency of functional FVIII.
Individuals with severe hemophilia experience frequent bleeding episodes into
major joints, soft tissue, and muscle, either spontaneously or following minor
trauma. The disease can be acutely life-threatening. Repeated bleeding can lead
to debilitating long-term complications, including
hemophilic arthropathy from bleeding into the joints.
BIVV001 is designed to be a new class of blood clotting FVIII. Preclinical and
clinical experience indicate that BIVV001 has an extended half-life, which can
achieve and maintain higher sustained factor activity levels than currently
available treatments, with less frequent administration.
Study objective
To evaluate the safety of BIVV001 in previously treated pediatric subjects with
hemophilia A.
Study design
Phase 3, open label, single arm.
Intervention
Weekly dose (intravenous) of BIVV001 for 52 weeks.
Study burden and risks
The risks are related to the blood sampling and possible side effects of the
study drug.
Paasheuvelweg 25
Amsterdam 1105BP
NL
Paasheuvelweg 25
Amsterdam 1105BP
NL
Listed location countries
Age
Inclusion criteria
- Participant must be younger than 12 years of age, at the time of signing the
informed consent
- Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII as documented
either by central laboratory testing at Screening or in historical medical
records from a clinical laboratory demonstrating <1% FVIII coagulant activity
(FVIII:C) or a documented genotype known to produce severe hemophilia A.
- Previous treatment for hemophilia A (prophylaxis or on-demand) with any
recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150
EDs for patients aged 6-11 years and above 50 EDs for patients aged below 6
years
- Weight above or equal to 10 kg.
Exclusion criteria
- History of hypersensitivity or anaphylaxis associated with any FVIII product.
- History of a positive inhibitor (to FVIII) test defined as >=0.6 BU/mL, or any
value greater than or equal to the lower sensitivity cut-off for laboratories
with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical
signs or symptoms of decreased response to FVIII administrations. Family
history of inhibitors will not exclude the participant.
- Positive inhibitor test result, defined as >=0.6 BU/mL at Screening.
Design
Recruitment
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2020-000769-18-NL |
| Other | na |
| CCMO | NL74679.018.20 |