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CTRI Number  CTRI/2023/02/049918 [Registered on: 21/02/2023] Trial Registered Prospectively
Last Modified On: 30/06/2023
Post Graduate Thesis  Yes 
Type of Trial  Interventional 
Type of Study   Biological
Preventive 
Study Design  Randomized, Parallel Group Trial 
Public Title of Study   Prophylactic Vs Routine Replacement Therapy with Factor VIII In Hemophilia A Patients 
Scientific Title of Study   A Comparative Study To Assess The Effect Of On Demand Vs Prophylactic Factor VIII Infusion On Annual Total Bleed Rate, Annual Joint Bleed Rate and Quality Of Life In Children Aged 12-18 Years With Severe Hemophilia A 
Trial Acronym   
Secondary IDs if Any  
Secondary ID  Identifier 
NIL  NIL 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)  
Name  Sugandh Sareen 
Designation  Junior Resident 
Affiliation  Guru Gobind Singh Medical College and Hospital 
Address  Hemophilia treatment centre, Department of Pediatrics, GGSMCH, Faridkot
Hemophilia treatment centre, Department of Pediatrics, GGSMCH, Faridkot
Faridkot
PUNJAB
151203
India 
Phone  8847085785  
Fax    
Email  sugandh9525@gmail.com  
 
Details of Contact Person
Scientific Query  
Name  Dr Varun Kaul 
Designation  Associate Professor 
Affiliation  Guru Gobind Singh Medical College and Hospital 
Address  Department of Pediatrics, Guru Gobind Singh Medical College and Hospital, Faridkot
Department of Pediatrics, Guru Gobind Singh Medical College and Hospital, Faridkot
Faridkot
PUNJAB
151203
India 
Phone  9876569622  
Fax    
Email  drvarunkaul2009@live.in  
 
Details of Contact Person
Public Query  
Name  Sugandh Sareen 
Designation  Junior Resident 
Affiliation  Guru Gobind Singh Medical College and Hospital 
Address  Hemophilia Treatment centre, Department of Pediatrics, GGSMCH, Faridkot
Hemophila Treatment centre, Department of Pediatrics, GGSMCH, Faridkot
Faridkot
PUNJAB
151203
India 
Phone  8847085785  
Fax    
Email  sugandh9525@gmail.com  
 
Source of Monetary or Material Support  
Guru Gobind Singh Medical College and Hospital, Faridkot, Punjab 
Intas Celestial division for factor support Mr Arun Chadha Associate Vice President Celestial Division, Intas Pharmaceuticals Limited(Corporate Office),Near Sola Bridge, S G Highway, Thaltej, Ahmedabad- 380054. 
 
Primary Sponsor  
Name  Dr Sugandh Sareen 
Address  Hemophilia treatment centre, Department of Pediatrics, Guru Gobind Singh Medical College and Hospital , Faridkot 
Type of Sponsor  Other [PG Resident] 
 
Details of Secondary Sponsor  
Name  Address 
NIL  NIL 
 
Countries of Recruitment     India  
Sites of Study  
No of Sites = 1  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Varun Kaul  Guru Gobind Singh Medical College and Hospital  2nd floor,Hemophilia treatment centre, Department of Pediatrics, GGSMCH
Faridkot
PUNJAB 		 9876569622

drvarunkaul2009@live.in 
 
Details of Ethics Committee  
No of Ethics Committees= 1  
Name of Committee  Approval Status 
Institutional ethics committee  Approved 
 
Regulatory Clearance Status from DCGI  
Status 
Not Applicable 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  (1) ICD-10 Condition: D66||Hereditary factor VIII deficiency,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Comparator Agent  On Demand Factor VIII  Factor VIII would be given on demand at the onset of any bleed . The factor would be given on demand based on the type of bleed as per the standard treatment guidelines of world federation of Hemophilia. e.g if the patient has a joint bleed then he will be given factor in the dose range to elevate the factor levels in the body upto 40%. The dose may be repeated depending upon the clinical response. 
Intervention  Prophylactic factor VIII  Prophylactic factor VIII would be given thrice weekly in the dose of 15 IU per Kg per dose to the randomized prophylactic study arm for one year and the effect on Annual bleed rate and quality of life would be assessed. They will also be given on demand treatment as comparator arm in case of any breakthrough bleed. 
 
Inclusion Criteria  
Age From  12.00 Year(s)
Age To  18.00 Year(s)
Gender  Male 
Details  1. Severe Hemophilia (Factor VIII levels <1% at diagnosis)
2. Age more than equal to 12 years to less than equal to 18 years at enrolment
3. more than equal to 6 treated joint bleeds in the previous one year
4. Inhibitor screen negavtive at enrolment
5. No evidence of hepatitis
6. Viral markers negative
7. Not on any prophylactic regimen in the last one year
8. Not on any non-factor replacement therapy 
 
ExclusionCriteria 
Details  1. Female Gender
2. Factor VIII levels >1% at diagnosis
3. Age <12 years or > 18 years at enrolment
4. <6 treated joint bleeds in the last one year.
5. Inhibitor screen positive at enrolment
6. Evidence of hepatitis 
 
Method of Generating Random Sequence   Computer generated randomization 
Method of Concealment   Sequentially numbered, sealed, opaque envelopes 
Blinding/Masking   Not Applicable 
Primary Outcome  
Outcome  TimePoints 
Annual Total Bleed Rate, Annual joint Bleed rate and quality of life  Baseline before the study initiation and after one year of study. 
 
Secondary Outcome  
Outcome  TimePoints 
Hemophilia Joint Health Score, Incidence of inhibitors, Pettersson Joint score, IPSG MRI score, HEAD US scoring system, HAL/Ped-Hal, Functional Impairment Scoring for Hemophilia (FISH), Health Related Quality of life  Inhibitor screening every 3 months
Rest all parameters, at the start of the study protocol and end of one year. 
 
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   N/A 
Date of First Enrollment (India)   01/03/2023 
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="6"
Days="0" 
Recruitment Status of Trial (Global)
Modification(s)  		 Not Applicable 
Recruitment Status of Trial (India)  Open to Recruitment 
Publication Details   Not yet published 
Individual Participant Data (IPD) Sharing Statement

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

Response - YES
  1. What data in particular will be shared?
    Response - Individual participant data that underlie the results reported in this article, after de-identification (text, tables, figures, and appendices).

  2. What additional supporting information will be shared?
    Response -  Study Protocol
    Response -  Statistical Analysis Plan
    Response - Informed Consent Form
    Response - Clinical Study Report

  3. Who will be able to view these files?
    Response - Researchers whose proposed use of the data has been approved by an independent review committee identified for this purpose.

  4. For what types of analyses will this data be available?
    Response - To achieve aims in the approved proposal.

  5. By what mechanism will data be made available?
    Response - Proposals should be directed to [sugandh9525@gmail.com].

  6. For how long will this data be available start date provided 01-01-2026 and end date provided 01-10-2029?
    Response - Beginning 9 months and ending 36 months following article publication.

  7. Any URL or additional information regarding plan/policy for sharing IPD? 
    Additional Information - NIL
Brief Summary   INTRODUCTION- Long term prophylaxis for treatment of Severe Hemophilia is the Gold Standard for significantly reducing symptoms in comparison to on demand treatment, with significant reduction in severity of bleeding episodes, improvement in quality of life in Hemophilic arthropathy. It is ideal to start primary prophylaxis to prevent arthropathy. Very little is known about the effects of late prophylaxis and we intend to see the response of initiation of secondary/ tertiary prophylaxis in children with severe Hemophilia A, aged 12-18 years.
AIMS AND OBJECTIVES- To assess the Annual Total Bleed Rate, Annual Joint Bleed Rate and Quality of Life in children aged 12 to 18 years with severe Hemophilia A, receiving on demand or prophylaxis with Factor VIII and then compare the effect of both the therapies on the same parameters.
MATERIALS AND METHODS- 50 males, in the age group 12- 18 years , diagnosed with severe Hemophilia A meeting the inclusion and exclusion criteria will be randomized and allocated to the two groups- on demand and prophylaxis. Effect of either of the therapies on the outcomes will be compared.
 
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