Phase 3
Completed N=94
BAY81-8973 Pediatric Safety and Efficacy Trial
Source: ClinicalTrials.gov NCT01311648 ↗Enrolled (actual)
94
Serious AEs
38.6%
Results posted
Nov 2020
Primary outcomePrimary: Annualized Number of Total Bleeds Within 48 h — 2.23; 1.86; 1.9 Bleeds
◆ Published Evidence
Established
40citations · ~4 / year
Pharmacokinetic properties of BAY 81-8973, a full-length recombinant factor VIII.
Summary
The primary objective was to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.
The secondary objectives were
* To assess the safety and efficacy of BAY81-8973 during surgeries.
* To assess incremental recovery of BAY81-8973.
* To assess pharmacokinetic (PK) parameters in a subset of children (Previously treated patients [PTPs] and previously untreated patients [PUPs] / minimally treated patients [MTPs] - participation in PK sampling was voluntary and required consent).
Linked Publications (5)
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Pharmacokinetic properties of BAY 81-8973, a full-length recombinant factor VIII.
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BAY 81-8973 safety and efficacy for prophylaxis and treatment of bleeds in previously treated children with severe haemophilia A: results of the LEOPOLD Kids Trial.
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Safety and efficacy of BAY 81-8973 for surgery in previously treated patients with haemophilia A: results of the LEOPOLD clinical trial programme.
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BAY 81-8973 (Octocog Alfa; Kovaltry<sup>®</sup>): A Review in Haemophilia A.
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BAY 81-8973, a full-length recombinant factor VIII: Human heat shock protein 70 improves the manufacturing process without affecting clinical safety.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annualized Number of Total Bleeds Within 48 h |
1.88; 0.00; 0.0 | — |
| SECONDARY Annualized Number of Total Bleeds During Prophylaxis Treatment |
2.03; 0.93; 4.7 | — |
| SECONDARY Hemostatic Control During Major and Minor Surgeries |
0; 0; 3; 0; 0; 1 | — |
| SECONDARY Number of Participants With Inhibitor Development in Main Study |
0; 0; 6; 0; 0; 17 | — |
| SECONDARY Number of Participants With New Inhibitor Development in Extension Study |
1; 0; 0; 0 | — |
| SECONDARY Factor VIII Recovery Values |
1.63; 1.72; 1.76; 0.86; 0.38 | — |
| SECONDARY Consumption of Factor VIII in All Infusions |
5499.1; 4679.1; 2195.8 | — |
| SECONDARY Consumption of FVIII in Infusions for Prophylaxis |
5224.8; 4492.7; 1486.6 | — |
| SECONDARY Consumption of FVIII in Infusions for the Treatment of Bleeds |
457.07; 391.64; 835.4 | — |
| SECONDARY Number of Infusions Per Bleed |
1.3; 1.4; 1.7 | — |
| SECONDARY Response to Treatment of Bleeds |
20; 12; 27; 23; 18; 56 | — |
| SECONDARY Half-life (t1/2) of BAY81-8973 in Plasma |
13.2; 12.1 | — |
Eligibility Criteria
Inclusion Criteria
- Male
- PTPs (previously treated patients): aged = 50 exposure days (EDs) with any FVIII concentrate, no current evidence of inhibitory antibody, and no history of FVIII inhibitor formation
- PUPs: no prior exposure to any FVIII product
- MTPs: having no more than 3 EDs with any FVIII product, no current evidence of inhibitory antibody and no history of FVIII inhibitor formation
Exclusion Criteria
- With another bleeding disorder that is different from Hemophilia A
- With thrombocytopenia (platelet count 2x upper limit of normal or Aspartate aminotransferase (AST)/Alanine aminotransferase (ALT) > 5x upper limit of normal
- Without a negative inhibitor testing at screening (except for PUPs)
- Receiving chemotherapy, immune modulatory drugs, has received another investigational FVIII product within the last month, or received another experimental drug within the last 3 months
- Requires any pre-medication to tolerate FVIII treatment
- Known hypersensitivity to active substance, mouse, or hamster protein
Data sourced from ClinicalTrials.gov (NCT01311648) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.