Phase 3
N=28
Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A
Blood Coagulation Disorders · Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT01029340 ↗Enrolled (actual)
28
Serious AEs
10.5%
Results posted
Nov 2013
Primary outcome: Primary: Part A - Area Under the Drug Concentration-time Curve (AUC) — 1889.23; 1583.91 Int.units x hours/deciliters (IU*h/dL)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Recombinant Factor VIII (BAY81-8973) (Biological); Recombinant Factor VIII (Kogenate FS, BAY14-2222) (Biological)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- Male
- Sponsor
- Bayer
- Primary completion
- Jun 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part A - Area Under the Drug Concentration-time Curve (AUC) |
1889.23; 1583.91 | — |
| PRIMARY Part A - Half-life (t 1/2) |
13.77; 12.00 | — |
| PRIMARY Part B - Annualized Number of Total Bleeds |
1.03 | — |
| SECONDARY Part B - The in Vivo Recovery Values of Human Factor VIII (FVIII) |
2.50 | — |
| SECONDARY Part B - Annualized Number of Bleeds in Each 6-month Potency Assignment Period |
1.9; 1.9 | — |
| SECONDARY Part B - Control of Bleeding as Measured by the Number of Injections Required to Treat a Bleed |
1.0; 1.0 | — |
| SECONDARY Part B - Changes From Baseline at 12 Months in Quality of Life (QoL) as Measured by Transformed Total Score of Haemo-QoL Questionnaire |
2.02 | — |
| SECONDARY Part B - Changes From Baseline at 12 Months in Utility Index as Measured by EQ-5D Questionaire |
0.00 | — |
| SECONDARY Part A - Number of Participants With Inhibitory Antibody Formation |
— | — |
| SECONDARY Part B - Number of Participants With Incidence of Inhibitory Antibody Formation |
— | — |
| SECONDARY Part C - Number of Participants With Incidence of Inhibitory Antibody Formation |
— | — |
| SECONDARY Part A - Number of Participants With Incidence of Antibody Formation to Heat-shock Protein (HSP-70) |
— | — |
| SECONDARY Part B - Number of Participants With Incidence of Antibody Formation to Heat-shock Protein (HSP-70) |
1 | — |
| SECONDARY Part C - Number of Participants With Incidence of Antibody Formation to Heat-shock Protein (HSP-70) |
— | — |
| SECONDARY Part A - Number of Participants With Incidence of Antibody Formation to Host Cell Proteins (HCP) |
— | — |
| SECONDARY Part B - Number of Participants With Incidence of Antibody Formation to Host Cell Proteins (HCP) |
— | — |
| SECONDARY Part C - Number of Participants With Incidence of Antibody Formation to Host Cell Proteins (HCP) |
— | — |
| SECONDARY Part B - Number of Participants With Assessment of the Hemostasis During Major Surgery |
1; 4; 0; 0 | — |
| SECONDARY Part C - Number of Participants With Assessment of the Hemostasis During Major Surgery |
1; 4; 0; 0 | — |
Summary
The study will assess the pharmacokinetics (part A) safety, tolerability, and efficacy of prophylaxis treatment (2 to 3 times a week) (part B) with BAY81-8973 over a one year period (split into two six month treatment periods). The study will compare 2 different methods (assays) for measuring the amount of study drug, the chromogenic substrate assay per European Pharmacopeia (CS/EP) with the classical assay (Chromogenic Substrate Adjusted, CS/ADJ). During one six month period patients will receive the study drug where the dose has been measured using the" (CS/EP) and during the other six months period the dose will be measured based on the Chromogenic Substrate Adjusted assay CS/ADJ)
Eligibility Criteria
Inclusion Criteria
- Male, aged 12 to 65 years
- Severe hemophilia A defined as /= 150 days of previous treatment with FVIII in lifetime
- Currently receiving on-demand or any type of prophylaxis treatment regimen with any FVIII product
- No history of or current FVIII inhibitors
Exclusion Criteria
- Presence of another bleeding disease that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B)
- Low platelet count, abnormal kidney function, or liver disease
- Received treatment with immune suppressing drugs within the last 3 months prior or requires treatment during the study. (Some drugs for hepatitis C, Human immunodeficiency virus (HIV), and steroids are allowed)
- Receiving or has received other experimental drugs within 3 months prior to study entry
- Allergy to Factor VIII or hamsters or mouse protein
Data sourced from ClinicalTrials.gov (NCT01029340). 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.