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BAY81-8973 Pediatric Safety and Efficacy Trial

NCT01311648 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 94

Last updated 2023-12-05

Study results available
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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).

Conditions

  • Haemophilia A

Interventions

BIOLOGICAL

Recombinant Factor VIII (Kovaltry, BAY81-8973)

Main study: 25-50 IU/kg at least 2x/week for 6 months and at least 50 EDs, IV infusion; Extension study: 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study), IV infusion. Exposure day (ED): An ED is a unit of time (1 day) in which replacement treatment of Hemophilia is given to a patient.

BIOLOGICAL

Recombinant Factor VIII (Kovaltry, BAY81-8973)

Main study: 15-50 IU/kg at least 1x/week for at least 50 EDs or until inhibitor development, IV infusion; Extension study: For participants having reached at least 50 EDs in main study - Part B: 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part B and extension study), IV infusion. For participants who developed an inhibitor in main study - Part B: up to 200 IU/kg per day or 100 IU/kg twice a day at the discretion of the investigator and coordinating investigator until successful eradication of the inhibitor, or until failure, for up to18 months (treatment beyond 18 months required an agreement with the sponsor and coordinating investigator), IV infusion

Sponsors & Collaborators

Principal Investigators

  • Bayer Study Director · Bayer

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-06-09
Primary Completion
2019-09-09
Completion
2020-10-27
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Bulgaria
  • Canada
  • Denmark
  • Hungary
  • Ireland
  • Israel
  • Italy
  • Latvia
  • Lithuania
  • Mexico
  • Norway
  • Poland
  • Romania
  • Russia
  • Spain

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01311648 on ClinicalTrials.gov