Trial Condition(s):
Blood Coagulation Disorders, Hemophilia A
Trial to Evaluate the Efficacy and Safety of a new full length recombinant human FVIII for Hemophilia A (Leopold I)
Bayer Identifier:
12954
ClinicalTrials.gov Identifier:
EudraCT Number:
EU CT Number:
Not Available
Trial Purpose
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)
Inclusion Criteria
- Male, aged 12 to 65 years - Severe hemophilia A defined as < 1% FVIII:C - >/= 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
Trial Summary
Enrollment Goal
Trial Dates
Phase
Could I receive a placebo?
Products
Accepts Healthy Volunteers
Where to Participate
| Locations | |
|---|---|
Locations Investigative Site Oxford, United Kingdom, OX3 7LJ | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Sheffield, United Kingdom, S10 2JF | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Sacramento, United States, 95817 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Krakow, Poland, 31-501 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site A Coruña, Spain, 15006 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Valencia, Spain, 46026 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Göteborg, Sweden, 413 45 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Malmö, Sweden, 205 02 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Karolinska Universitetssjukhuset i Solna Stockholm, Sweden, 171 76 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Parktown, South Africa, 2132 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Wien, Austria, 1090 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Jakarta, Indonesia, 10430 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Bangalore, India, 34 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Pune, India, 411005 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Tampa, United States, 33607 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Boston, United States, 02115 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Cleveland, United States, 44106-2602 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Orange, United States, 92868 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Warszawa, Poland, 02-776 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Barcelona, Spain, 08035 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Santander, Spain, 39008 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Jaén, Spain, 23007 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site DK-Aarhus N, Denmark, 8200 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Oslo, Norway, 0027 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Pretoria, South Africa, 0001 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Ramat Gan, Israel, 5262000 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Mumbai, India, 400012 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Changhua, Taiwan, China, 500 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Hongkong, Hong Kong, China | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Bangkok, Thailand, 10400 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Karachi, Pakistan, 75300 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Taipei, Taiwan, China, 11217 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Taipei, Taiwan, China, 10016 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Bangkok, Thailand, 10330 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Bonn, Germany, 53127 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Mainz, Germany, 55131 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Frankfurt, Germany, 60596 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Magdeburg, Germany, 39112 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Dundee, United Kingdom, DD1 9SY | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site East Lansing, United States, 48823 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Kansas City, United States, 64108-9898 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Milano, Italy, 20122 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Catanzaro, Italy, 88100 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Vicenza, Italy, 36100 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Napoli, Italy, 80131 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Napoli, Italy, 80144 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Roma, Italy, 00168 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Oviedo, Spain, 33006 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Bahia Blanca, Argentina, B8001HXM | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Rosario, Argentina, S2000CKF | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Graz, Austria, 8036 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Izmir, Turkey, 35-100 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Adana, Turkey, 01330 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Antalya, Turkey, 07059 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Zagreb, Croatia, 10000 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Nis, Serbia, 18000 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Belgrade, Serbia, 11000 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Novi Sad, Serbia, 21000 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Beograd, Serbia, 11000 | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site London, United Kingdom, SE1 7EH | Contact Us: E-mail: [email protected] Phone: Not Available |
Locations Investigative Site Homburg, Germany, 66421 | Contact Us: E-mail: [email protected] Phone: Not Available |
Trial Design
A two Part Randomized Cross-Over Trial to Evaluate the Pharmacokinetics, Efficacy, and Safety Profile of Plasma Protein-Free Recombinant FVIII Formulated With Sucrose (BAY81-8973) in Previously Treated Subjects With Severe Hemophilia A Under Prophylaxis Therapy
Trial Type:
Interventional
Intervention Type:
Biological/Vaccine
Trial Purpose:
Treatment
Allocation:
Randomized
Blinding:
Open Label
Assignment:
Crossover Assignment
Trial Arms:
5
Primary Outcome
- Part A - Area under the drug concentration-time curve (AUC)To examine the Pharmacokinetic (PK) characteristics of BAY 81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.Timeframe: Samples taken at pre-injection, and at 0.25, 0.5, 1, 3, 6, 8, 24, 30 and 48 hours post injection. AUC calculated from time of injection to infinity.
- Part A - Half-life (t 1/2)To examine the PK characteristics of BAY81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.Timeframe: Samples taken at pre-injection, and at 0.25, 0.5, 1, 3, 6, 8, 24, 30 and 48 hours post injection.
- Part B - Annualized number of total bleedsThe annualized number of bleeds experienced by participantsTimeframe: 12 months after randomization
Secondary Outcome
- Part B - The in vivo recovery values of Human factor VIII (FVIII)The amount of Factor VIII found in blood samples taken after the injection of the study drug at the beginning of the CS/EP treatment period.Timeframe: 15-30 minutes after the injection
- Part B - Annualized number of bleeds in each 6-month potency assignment periodThe annualized number of bleeds experienced by participants in each of the two treatment periodsTimeframe: 6 months on each potency
- Part B - Control of bleeding as measured by the number of injections required to treat a bleedThe number of injections needed by participants to stop a bleedTimeframe: 6 months on each potency
- Part B - changes from baseline at 12 months in quality of life (QoL) as measured by Transformed Total Score of Haemo-QoL questionnaireA measure of how treatment with BAY81-8973 affected the daily life of participants. the scoring system has 100 points. 0 is the worst possible score. 100 is the best possible score. Positive changes from baseline indicate an improvement in quality of life and negative changes indicate a deterioration.Timeframe: Baseline and 12 months
- Part B - Changes from baseline at 12 months in Utility Index as measured by EQ–5D questionaireA measure of how treatment with BAY81-8973 affected the daily life of participants. 1.0 = Best possible score, -0.594 = Worst possible score. Positive changes from baseline indicate an improvement and negative changes indicate a deterioration.Timeframe: Baseline and 12 months
- Part A - Number of participants with inhibitory antibody formationA test to ensure that participants have not developed antibodies that will interfere with the action of BAY81-8973Timeframe: Up to 6 weeks after first injection of study drug
- Part B - Number of participants with incidence of inhibitory antibody formationA test to ensure that participants have not developed antibodies that will interfere with the action of BAY81-8973Timeframe: Up to 12 months after drug administration
- Part C - Number of participants with incidence of inhibitory antibody formationA test to ensure that participants have not developed antibodies that will interfere with the action of BAY81-8973Timeframe: before and 3 weeks after surgery
- Part A - Number of participants with incidence of antibody formation to heat-shock protein (HSP-70)A test to analyze the formation of antibodies to HSP-70Timeframe: Up to 6 weeks after drug administration
- Part B - Number of participants with incidence of antibody formation to heat-shock protein (HSP-70)A test to analyze the formation of antibodies to HSP-70Timeframe: Up to 12 months after drug administration
- Part C - Number of participants with incidence of antibody formation to heat-shock protein (HSP-70)A test to analyze the formation of antibodies to HSP-70Timeframe: before and 3 weeks after surgery
- Part A - Number of participants with incidence of antibody formation to host cell proteins (HCP)A test to ensure that participants have not developed antibodies to HCP during the studyTimeframe: Up to 4 weeks after drug administration
- Part B - Number of participants with incidence of antibody formation to host cell proteins (HCP)A test to ensure that participants have not developed antibodies to HCP during the studyTimeframe: Up to 12 months after drug administration
- Part C - Number of participants with incidence of antibody formation to host cell proteins (HCP)A test to ensure that participants have not developed antibodies to HCP during the studyTimeframe: before and 3 weeks after surgery
- Part B - Number of participants with assessment of the hemostasis during major surgeryAn assessment made by surgeons of how effective BAY81-8973 was in stopping bleeding during major operationsTimeframe: An average of 1 month after start of treatment
- Part C - Number of participants with assessment of the hemostasis during major surgeryAn assessment made by surgeons of how effective BAY81-8973 was in stopping bleeding during major operationsTimeframe: at the time of surgery
Additional Information
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