Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A
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Purpose
Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting process to be slowed and the person experiences bleeds causing serious problems that could lead to disability. The current standard treatment for severe hemophilia A is infusion of FVIII to stop bleeding, or regular scheduled treatment to prevent bleeds from occuring. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day.
In this trial safety and efficacy of a long-acting recombinant Factor VIII molecule is being evaluated in 50 male subjects, < 12 years of age, with severe Hemophilia A. These subjects will receive open label treatment with long-acting rFVIII for approximately 6 months (or longer until 50 exposure days) on a regular schedule at least once every 7-days. Doses and dose intervals may be adapted to the subject's clinical need. Patients will attend the treatment center for routine blood samples and will be required to keep an electronic diary.
Subjects will be offered participation in an optional extension study to collect observations for at least an additional 50 exposure days.
| Condition | Intervention | Phase |
|---|---|---|
|
Hemophilia A |
Biological: BAY94-9027 |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A |
- Annualized number of all bleeds [ Time Frame: Up to 11 months ] [ Designated as safety issue: No ]
- Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time period [ Time Frame: 6 time points from pre-infusion to 72 hours post-infusion ] [ Designated as safety issue: No ]Pharmacokinetics profile includes maximum concentration (Cmax), incremental recovery, mean residence time (MRT), apparent volume of distribution at steady state (Vss), half-life, area under the curve (AUC), and clearance, which are calculated values based on time-weighed blood concentration of drug.
- Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent) [ Time Frame: Up to 11 months ] [ Designated as safety issue: No ]
- Inhibitor development after 10 to 15 EDs (exposure days) [ Time Frame: Up to 15 weeks ] [ Designated as safety issue: No ]
- Inhibitor development after 50 EDs [ Time Frame: Up to 11 months ] [ Designated as safety issue: No ]
- Number of participants with adverse events as a measure of safety and tolerability [ Time Frame: Up to 11 months ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: | 50 |
| Study Start Date: | May 2013 |
| Estimated Study Completion Date: | February 2016 |
| Estimated Primary Completion Date: | March 2015 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: BAY94-9027 |
Biological: BAY94-9027
Prophylaxis treatment 25-60 IU/kg at least 1x/week.
|
Eligibility| Ages Eligible for Study: | up to 12 Years |
| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Males < 12 years of age
- Subjects with severe hemophilia A
- Previously treated with FVIII for > 50 exposure days
Exclusion Criteria:
- Subjects with current evidence of or history of inhibitors to FVIII
- Any other inherited or acquired bleeding disorder
- Platelet counts < 100,000/mm3
- Creatinine > 2x the upper limit of normal
- Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal
Contacts and Locations| Contact: Bayer Clinical Trials Contact | clinical-trials-contact@bayerhealthcare.com | |
| Contact: For trial location information (Phone Menu Options '3' or '4') | (+)1-888-84 22937 |
Show 33 Study Locations| Study Director: | Bayer Study Director | Bayer |
More Information
Additional Information:
No publications provided
| Responsible Party: | Bayer |
| ClinicalTrials.gov Identifier: | NCT01775618 History of Changes |
| Other Study ID Numbers: | 15912, 2012-004434-42 |
| Study First Received: | January 23, 2013 |
| Last Updated: | April 23, 2013 |
| Health Authority: | Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica Belgium: The Federal Public Service (FPS) Health, Food Chain Safety and Environment Bulgaria: Bulgarian Drug Agency Canada: Health Canada Netherlands: The Central Committee on Research Involving Human Subjects (CCMO) Romania: National Medicines Agency Israel: Ministry of Health Italy: Ethics Committee Lithuania: State Medicine Control Agency - Ministry of Health Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products United Kingdom: Medicines and Healthcare Products Regulatory Agency United States: Food and Drug Administration |
Keywords provided by Bayer:
|
Hemophilia A Factor VIII Prophylaxis Pediatric |
Additional relevant MeSH terms:
|
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases Coagulation Protein Disorders Hemorrhagic Disorders |
Genetic Diseases, Inborn Factor VIII Coagulants Hematologic Agents Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 16, 2013