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CTRI Number  CTRI/2021/10/037406 [Registered on: 20/10/2021] Trial Registered Prospectively
Last Modified On: 17/05/2023
Post Graduate Thesis  No 
Type of Trial  Interventional 
Type of Study   Drug 
Study Design  Single Arm Study 
Public Title of Study   Safety of ADVATE in previously treated Indian patients for treatment of mild , moderate and severe bleeding episodes in Haemophilia A 
Scientific Title of Study   Phase 4, Multicenter, Prospective, Interventional, Post-Marketing Study in Hemophilia A Patients in India Receiving ADVATE as On-Demand or Prophylaxis Under Standard Clinical Practice 
Trial Acronym  Nil 
Secondary IDs if Any  
Secondary ID  Identifier 
TAK-761-4009, Dated 31 Jul 2019  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
Modification(s)  
Name  Dr Sandeep Arora 
Designation  Head Medical Affairs 
Affiliation  Takeda Biopharmaceuticals India Pvt. Ltd. 
Address  Takeda Biopharmaceuticals India Pvt. Ltd. 6th Floor, Tower C, Building No. 8, Cyber City,Gurgaon

Gurgaon
HARYANA
122002
India 
Phone  91-124-4559100  
Fax    
Email  sandeep.arora@takeda.com  
 
Details of Contact Person
Public Query
Modification(s)  
Name  Ruchi Sogarwal 
Designation  Head – Public Affairs & Patient Advocacy 
Affiliation  Takeda Biopharmaceuticals India Pvt. Ltd. 
Address  Takeda Biopharmaceuticals India Pvt. Ltd. 6th Floor, Tower C, Building No. 8, Cyber City, Gurgaon

Gurgaon
HARYANA
122002
India 
Phone  91-124-4559100  
Fax    
Email  ruchi.sogarwal@takeda.com  
 
Source of Monetary or Material Support  
1. Baxalta US Inc., 300 Shire Way, Lexington, MA 02421  
2. Baxalta Innovations GmbH, Industriestrasse 67, A-1221 Vienna  
 
Primary Sponsor  
Name  Baxalta US Inc 
Address  300 Shire Way, Lexington, MA 02421  
Type of Sponsor  Pharmaceutical industry-Global 
 
Details of Secondary Sponsor  
Name  Address 
Baxalta Innovations GmbH  Industriestrasse 67, A-1221 Vienna 
 
Countries of Recruitment     India  
Sites of Study  
No of Sites = 5  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Tulika Seth  All India Institute of Medical Sciences  Department of Haematology All India Institute of Medical Sciences, Ansari Nagar, New Delhi -110029
New Delhi
DELHI 		 91-9810493246

tuliseth@yahoo.com 
Dr Neeraj Siddhartan  Amrita Institute of Medical Sciences and Research Centre  Department of Haematology Amrita Institute of Medical Sciences, Peeliyadu Road, Ponekkara, Edappally, Ernakulam, Kerala 682041
Ernakulam
KERALA 		 91-8054959525

mjosephjohn@gmail.com 
Dr Savita Rangarajan  K. J. Somaiya Hospital and Research Centre  2nd Floor, College Building Somaiya Ayurvihar complex, Behind Everard Nagar, Eastern Express Highway Sion East, Mumbai-400022, Maharashtra
Mumbai
MAHARASHTRA 		 91-9619525341

rangarajansavita@gmail.com 
Dr Cecil Ross  St. Johns Medical College Hospital  Dept. of Medicine and Hematology, Sarjapur Road, Bangalore, Karnataka, India
Bangalore
KARNATAKA 		 91-9448493705

cecilrross@gmail.com 
Dr Sunil Devichand Lohade  Unique Childrens Hospital Pvt Ltd  Hira Moti Fortune, Mumbai Pune Road, Opp Chinchwad Police Station Chinchwad, Pune - 411019 Maharashtra
Pune
MAHARASHTRA 		 91-9049845551

sunil.lohade@gmail.com 
 
Details of Ethics Committee
Modification(s)  
No of Ethics Committees= 5  
Name of Committee  Approval Status 
Ethics Committee of Unique Childrens Hospitals Pvt Ltd., Hira Moti Fortune Opp. Police Station, Pune Mumbai Road Chinchwad Pune Maharashtra - 411019 India  Approved 
Ethics Committee, All India Institute of Medical Sciences-Ansari Nagar, New Delhi - 110029  Approved 
Institutional Ethics Committee K.J Somaiya Hospital Private Limited Ethics Committee  Approved 
Institutional Ethics Committee, Amrita Institute of Medical and Research Centre- Peeliyadu Road, Ponekkara, Edappally, Ernakulam, Kerala 682041  Submittted/Under Review 
Institutional Ethics Committee, St John’s Medical College & Hospital -Sarjapur Road, Bangalore- 560 034  Approved 
 
Regulatory Clearance Status from DCGI  
Status 
Approved/Obtained 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  , (1) ICD-10 Condition: D66||Hereditary factor VIII deficiency,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Intervention  ADVATE®-Coagulation Factor VIII (Recombinant) r FVIII I.P. (250 IU, 500 IU, 1000 IU and 1500 IU) and solvent for solution  1 vial contains Active Ingredient: coagulation factor VIII, Excipients: ,  - Trehalose, L- histidine, Tris – (Hydroxymethyl) Aminomethane,Sodium Chloride, , Calcium Chloride, Glutathione (Reduced), Polysorbate 80 (Vegetable derived), Mannitol Solvent: S.W. F. I. of 2 ml Dose and frequency: Subjects will be infused with a dose range of 20-50±5 IU/kg of ADVATE. The frequency of dosing can be either every second day or 3 times weekly. The individual regimen will be chosen by the investigator, taking into account the patient’s wishes, clinical status and readiness to comply with frequent dosing and adapted as per clinical response in the individual cases. Duration: 6 months ± 1 week Route of administration: Intravenous 
Comparator Agent  Not applicable  Not applicable 
 
Inclusion Criteria  
Age From  0.00 Year(s)
Age To  65.00 Year(s)
Gender  Both 
Details  1. The subject or legally authorized representative (in case of study participants <18 years of age) gave written informed consent to participate in the study.
2. Subject of any age with hemophilia A.
3. Subject is defined as previously treated patient (PTP):
• Subject aged ≥ 6 years that has been previously treated with plasma-derived and/or recombinant FVIII concentrate(s) for a minimum of 150 exposure days (EDs).
• Subject aged < 6 years that has been previously treated with plasma-derived and/or recombinant FVIII concentrate(s) for a minimum of 50 EDs.
4. Subject has negative history of FVIII inhibitors and negative inhibitor at screening defined as less than 0.6 Bethesda units (BU)/mL (Nijmegen-modified Bethesda assay).
5. Subject is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm3, as confirmed by central laboratory at screening.
6. Subject is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
7. The subject is willing and able to comply with the requirements of the protocol
 
 
ExclusionCriteria 
Details  1. Subject has known hypersensitivity to mouse or hamster proteins or to any of the excipients of FVIII concentrates.
2. Subject has been diagnosed with bleeding disorder(s) other than congenital hemophilia A, such as acquired hemophilia A, von Willebrand´s disease or thrombocytopenia (platelet count<100,000/mL).
3. Subject has received treatment for hemophilia A with non-FVIII products/concentrates (eg, emicizumab [Hemlibra®]) in the 6 months prior to screening.
4. Subject has severe chronic hepatic dysfunction [eg, ≥ 5 times upper limit of normal alanine aminotransferase (ALT), aspartate aminotransferase (AST) or INR > 1.5 as confirmed by central laboratory at screening].
5. Subject has planned, or is likely to have, surgery during the study period.
6. Subject has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject’s safety or compliance.
7. Subject is currently receiving or is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone greater than mg/day, or α-interferon) other than antiretroviral chemotherapy.
8. Subject has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
9. Subject is a family member or employee of the investigator.

 
 
Method of Generating Random Sequence   Not Applicable 
Method of Concealment   Not Applicable 
Blinding/Masking   Open Label 
Primary Outcome  
Outcome  TimePoints 
Incidence of SAEs (including FVIII inhibitor formation) that are at least possibly related to ADVATE  6 months ± 1 week  
 
Secondary Outcome  
Outcome  TimePoints 
Safety:
1. Incidence of non-serious AEs that are at least possibly related to ADVATE.
2. Clinically significant changes in clinical laboratory parameters (hematology and clinical chemistry)
 		 6 months ± 1 week 
Efficacy:
1. Annualized bleeding rate (ABR) with prophylactic use of ADVATE.
2. Total number of infusions and the average number of infusions per week per month during prophylactic treatment.
3. Total and average body mass adjusted consumption of ADVATE per week per month during prophylactic treatment.
4. Overall hemostatic efficacy rating for treatment of bleeding episodes.
5. Number of ADVATE infusions required to achieve bleed resolution.
6. Body mass adjusted consumption of ADVATE per bleeding episode.
 		 6 months ± 1 week 
 
Target Sample Size   Total Sample Size="50"
Sample Size from India="50" 
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 4 
Date of First Enrollment (India)   01/11/2021 
Date of Study Completion (India) Applicable only for Completed/Terminated trials 
Date of First Enrollment (Global)  Date Missing 
Date of Study Completion (Global) Applicable only for Completed/Terminated trials 
Estimated Duration of Trial   Years="1"
Months="9"
Days="0" 
Recruitment Status of Trial (Global)
Modification(s)  		 Not Applicable 
Recruitment Status of Trial (India)  Closed to Recruitment of Participants 
Publication Details    
Individual Participant Data (IPD) Sharing Statement

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

Response - NO
Brief Summary  

This is a single-country, multi-centre, prospective, interventional, open-label, post-marketing, non-randomised trial with a single treatment arm in previously treated haemophilia A patients (PTPs) in India receiving ADVATE under standard clinical practice.

50 PTPs  with congenital haemophilia A will receive a prophylactic or on demand regimen with ADVATE (Recombinant Coagulation Factor VIII -rFVIII; Octocog Alfa) for a period of 6 months ± 1 week

All enrolled subjects who have met the inclusion and exclusion criteria will be treated with ADVATE according to a regimen determined by the treating physician.The individual subjects duration of participation is expected to be approximately 7-8 months. The period of observation for each subject will be 6 months; another 10 to 15 days are anticipated for the study completion visit (“End-of-Treatment Visit”).

 
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