FULL DETAILS (Read-only)  -> Click Here to Create PDF for Current Dataset of Trial
CTRI Number  CTRI/2012/01/002390 [Registered on: 31/01/2012] Trial Registered Prospectively
Last Modified On: 02/02/2016
Post Graduate Thesis  No 
Type of Trial  Interventional 
Type of Study   Drug 
Study Design  Randomized, Parallel Group, Multiple Arm Trial 
Public Title of Study   A clinical trial to determine the distribution through the patient’s body, safety and effectiveness of rVWF and rFVIII in treatment of bleeding episodes in patient’s diagnosed with the Von Willebrand Disease. 
Scientific Title of Study   A Phase 3 Clinical Study to Determine the Pharmacokinetics, Safety and Efficacy of rVWF:rFVIII and rVWF in the Treatment of Bleeding Episodes in Subjects Diagnosed with Von Willebrand Disease 
Trial Acronym   
Secondary IDs if Any  
Secondary ID  Identifier 
2010-024108-84  EudraCT 
Protocol 071001 dated 8 Apr 2011  Protocol Number 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)  
Name   
Designation   
Affiliation   
Address 




 
Phone    
Fax    
Email    
 
Details of Contact Person
Scientific Query  
Name  Dr Ashok Moharana 
Designation  Director, Regulatory & Medical Affairs, India/SEA 
Affiliation  Baxter (India) Private Limited 
Address  2nd Floor, Tower-C Building No. 8 DLF Cyber City, DLF Phase II Gurgaon, Haryana India

Gurgaon
HARYANA
122002
India 
Phone  01244500200  
Fax  01244263505  
Email  ashok_moharana@baxter.com  
 
Details of Contact Person
Public Query  
Name  Dr Ashok Moharana 
Designation  Director, Regulatory & Medical Affairs, India/SEA 
Affiliation  Baxter (India) Private Limited 
Address  2nd Floor, Tower-C Building No. 8 DLF Cyber City, DLF Phase II Gurgaon, Haryana India


HARYANA
122002
India 
Phone  01244500200  
Fax  01244263505  
Email  ashok_moharana@baxter.com  
 
Source of Monetary or Material Support  
Baxter Healthcare Corporation 
 
Primary Sponsor  
Name  Baxter Healthcare Corporation 
Address  One Baxter Way Westlake Village, CA 91362  
Type of Sponsor  Other [Healthcare industry] 
 
Details of Secondary Sponsor  
Name  Address 
NIL  NIL 
 
Countries of Recruitment     Canada
United States of America
Australia
Austria
Belgium
Bulgaria
France
Germany
India
Italy
Netherlands
Sweden
United Kingdom
Japan  
Sites of Study  
No of Sites = 3  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Alok Srivastava  Christian Medical College  Department of Hematology, IDA Scudder Road
Vellore
TAMIL NADU 		 0416228235
0416222644
aloks@cmcvellore.ac.in 
Dr Vijay M Ramanan  Jehangir Clinical Development Centre  Department of Hematology, Jehangir Hospital Premises, 32 Sasoon Road Department of Medical Oncology Jahangir Hospital Building
Pune
MAHARASHTRA 		 02026059318
02026059319
mvijayr@gmail.com 
Dr Shashikant J Apte  Sahyadri Specialty Hospital  Hematology and BMT Department, Plot No. 30C, Karve Road
Pune
MAHARASHTRA 		 09822404983
02025459117
shashikant.apte@gmail.com 
 
Details of Ethics Committee
Modification(s)  
No of Ethics Committees= 3  
Name of Committee  Approval Status 
Ethics Committee, Institutional Review Board, Christine Medical College  Approved 
HIrabai Cawasji Jehangir Medical Research Institute and Jehangir Clinical Development Centre Ethics Committee  Approved 
Sahaydri Hospitals Limited Ethics Committee, Sahaydri Speciality Hospital  Approved 
 
Regulatory Clearance Status from DCGI  
Status 
Approved/Obtained 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  severe hereditary Von Willebrand Disease,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Intervention  PK50 only arm  2 PK infusions of 50 IU/kg rVWF separated by approximately 18 days.  
Comparator Agent  PK50 plus treatment of bleeding  2 PK infusions of 50 IU/kg rVWF separated by approximately 18 days, followed by treatment of bleeding episodes with rVWF for approximately 12 months. 
Intervention  PK80 plus treatment of bleeding   2 PK infusions of 80 IU/kg rVWF separated by approximately 6 months, followed by treatment of bleeding episodes with rVWF for approximately 12 months. 
Comparator Agent  rVWF Treatment of bleeding only  Treatment of bleeding episodes with rVWF for approximately 12 months. 
 
Inclusion Criteria  
Age From  18.00 Year(s)
Age To  65.00 Year(s)
Gender  Both 
Details 
•The subject has been diagnosed with:
o type 3 VWD (VWF:Ag ≤3 IU/dl) or
o severe non-type 3 VWD (VWF:RCo <20 IU/dL) or
o type 2N VWD (FVIII:C <10% and historically documented genetics)
•The subject, who participates for the treatment for bleeding episodes, has had a minimum of 6 documented bleeds (medical history) requiring VWF coagulation factor replacement therapy during the previous3 years prior to enrollment.
•The subject has a Karnofsky score ≥60.
•If female of childbearing potential, subject presents with a negative pregnancy test
•The subject agrees to employ adequate birth control measures for the duration of the study.
•Subject is willing and able to comply with the requirements of the protocol.
 
 
ExclusionCriteria 
Details  • The subject has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (eg qualitative and quantitative platelet disorders or elevated PT/international normalized ratio [INR] >1.4).
• The subject has a documented history of a VWF:RCo half-life of <6 hours.
• The subject has a history or presence of a VWF inhibitor at screening.
• The subject has a history or presence of a factor VIII (FVIII) inhibitor with a titer ≥0.4 BU (by Nijmegen assay) or ≥0.6 BU (by Bethesda assay).
• The subject has a known hypersensitivity to any of the components of the study drugs, such as to mouse or hamster proteins.
• The subject has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies.
• The subject has a medical history of a thromboembolic event.
• The subject is HIV positive with an absolute CD4 count <200/mm3.
• The subject has been diagnosed with cardiovascular disease (New York Heart Association [NYHA] classes 1-4)
• The subject has an acute illness (eg, influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, non-seasonal asthma) at screening.
• The subject has been diagnosed with significant liver disease as evidenced by any of the following: serum alanine aminotransferase (ALT) 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (eg, presence of otherwise unexplained splenomegaly, history of esophageal varices).
• The subject has been diagnosed with renal disease, with a serum creatinine level ≥2 mg/dL.
• In the judgment of the investigator, the subject has another clinically significant concomitant disease (eg, uncontrolled hypertension) that may pose additional risks for the subject.
• The subject has been treated with an imunomodulatory drug, excluding topical treatment (eg, ointments, nasal sprays), within 30 days prior to signing the informed consent.
• Subject is pregnant or lactating at the time of enrollment.
• Subject has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an investigational product or investigational device during the course of this study.
• The subject has a history of drug or alcohol abuse within the 2 years prior to enrollment.
• The subject has a progressive fatal disease and/or life expectancy of less than 3 months.
• The subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
• The subject suffers from a mental condition rendering him/her unable to understand the nature, scope and possible consequences of the study and/or evidence of an uncooperative attitude.
• The subject is in prison or compulsory detention by regulatory and/or juridical order
 
 
Method of Generating Random Sequence   Computer generated randomization 
Method of Concealment   Centralized 
Blinding/Masking   Double Blind Double Dummy 
Primary Outcome  
Outcome  TimePoints 
Number of subjects with a treatment success for treated bleeding episodes.  12 month 
 
Secondary Outcome  
Outcome  TimePoints 
Number of treated bleeding episodes with an efficacy rating of excellent or good  12 month 
Number of infusions and rVWF:rFVIII and/or rVWF units per bleeding episode  12 month 
Development of inhibitory and total binding anti-VWF antibodies  12 month 
Development of inhibitory antibodies to FVIII, Time-points  12 month 
Development of antibodies to Chinese hamster ovary (CHO) proteins, mouse immunoglobulin G (IgG) and rFurin  12 month 
Occurrence of thrombotic events, Time-points  12 month 
Other IP related AEs, such as clinically significant changes in routine laboratory parameters (hematology and clinical chemistry) and vital signs  12 month 
Area under the plasma concentration/time curve from time 0 to infinity (AUC0-∞/Dose); area under the plasma concentration/time curve from time 0 to 96 hours (AUC0-96h/Dose); mean residence time (MRT); clearance (CL); incremental recovery (IR), elimination phase half-life (T1/2); volume of distribution at steady state (Vss) of VWF Ristocetin cofactor (VWF:RCo), VWF antigen (VWF:Ag), VWF collagen-binding (VWF:CB), and FVIII  6 month 
In vivo recovery (IVR) of VWF:RCo, VWF:Ag and VWF:CB  12 month 
Comparison of intra-subject PK of VWF:RCo, VWF:CB and VWF:Ag at baseline and after 6 months in a subset of at least 20 subjects with severe VWD (minimum 6 subjects with type 3 VWD)  6 month 
 
Target Sample Size   Total Sample Size="45"
Sample Size from India="6" 
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" 
Phase of Trial   Phase 3 
Date of First Enrollment (India)   15/02/2012 
Date of Study Completion (India) Applicable only for Completed/Terminated trials 
Date of First Enrollment (Global)  17/11/2011 
Date of Study Completion (Global) Applicable only for Completed/Terminated trials 
Estimated Duration of Trial   Years="2"
Months="0"
Days="0" 
Recruitment Status of Trial (Global)
Modification(s)  		 Completed 
Recruitment Status of Trial (India)  Other (Terminated) 
Publication Details    
Individual Participant Data (IPD) Sharing Statement

Will individual participant data (IPD) be shared publicly (including data dictionaries)?  

Brief Summary  

This is a Phase 3, multi-center, open-label study to evaluate Pharmacokinetics, safety and effectiveness in the treatment of bleeding episodes in patients with severe type 3 VWD, non-type 3 WVD and type 2N VWD disease. The study will be divided in 2 parts (A&B). Approximately 45 patients will be enrolled. The primary endpoint is a pharmacokinetic measurement.

 
Close