| CTRI Number |
CTRI/2025/11/096854 [Registered on: 03/11/2025] Trial Registered Prospectively |
| Last Modified On: |
02/11/2025 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Randomized, Parallel Group, Active Controlled Trial |
|
Public Title of Study
|
A study comparing Emicizumab and Extended Half-Life Factor VIII for reducing bleeding episodes in Hemophilia A patients without inhibitors.
|
|
Scientific Title of Study
|
A pilot Randomised Control Trial comparing the efficacy and safety of Standard Dose and Low dose Emicizumab prophylaxis with Extended Half Life Factor VIII concentrate in children with severe Hemophilia A without Inhibitors |
| Trial Acronym |
NIL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| NIL |
NIL |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Aswathy V Nair |
| Designation |
Senior Resident |
| Affiliation |
Shyam Shah Medical College and Associated SGMH, GMH |
| Address |
Room number 102
Hematology ward
Department of Pediatrics
First Floor
Gandhi Memorial Hospital
Shyam Shah Medical College
Rewa
Rewa
MADHYA PRADESH
486001
India |
| Phone |
07358956806 |
| Fax |
|
| Email |
aswathyvnair1995@gmail.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Gaurav Tripathi |
| Designation |
Assistant Professor |
| Affiliation |
Shyam Shah Medical College and Associated SGMH, GMH |
| Address |
Room number 102
Hematology ward
Department of Pediatrics
First Floor
Gandhi Memorial Hospital
Shyam Shah Medical College
Rewa
Rewa
MADHYA PRADESH
486001
India |
| Phone |
9630132488 |
| Fax |
|
| Email |
drgtripathi@gmail.com |
|
Details of Contact Person
Public Query
|
| Name |
Aswathy V Nair |
| Designation |
Senior Resident |
| Affiliation |
Shyam Shah Medical College and Associated SGMH, GMH |
| Address |
Room number 102
Hematology ward
Department of Pediatrics
First Floor
Gandhi Memorial Hospital
Shyam Shah Medical College
Rewa
Rewa
MADHYA PRADESH
486001
India |
| Phone |
07358956806 |
| Fax |
|
| Email |
aswathyvnair1995@gmail.com |
|
|
Source of Monetary or Material Support
|
| Department of Pediatrics
Gandhi Memorial Hospital
Shyam Shah Medical College
Rewa, Madhya Pradesh |
|
|
Primary Sponsor
|
| Name |
NIL |
| Address |
NIL |
| Type of Sponsor |
Government medical college |
|
|
Details of Secondary Sponsor
|
|
|
Countries of Recruitment
|
India |
|
Sites of Study
|
| No of Sites = 1 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Aswathy V Nair |
Gandhi Memorial Hospital, Shyam Shah Medical College |
Room Number 102
Hematology Ward
First Floor
Department of Pediatrics
Rewa
MADHYA PRADESH |
07358956806
aswathyvnair1995@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 1 |
| Name of Committee |
Approval Status |
| Institutional Ethics Committee , Shyam Shah Medical College and associated SGMH, GMH, Rewa |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
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 |
Extended Half Life Factor VIII concentrate prophylaxis |
Patients will receive prophylaxis with extended half-life recombinant factor VIII (EHL FVIII) concentrate, administered intravenously at standard prophylactic doses 25–50 IU/kg twice weekly for 24 weeks. |
| Intervention |
Low Dose Emicizumab prophylaxis |
Patients will receive prophylaxis with low-dose emicizumab, administered subcutaneously at 50% of the standard maintenance dose 1.5 mg/kg every two weeks for 24 weeks |
| Intervention |
Standard Dose Emicizumab prophylaxis |
Patients will receive prophylaxis with emicizumab, administered subcutaneously at standard dose regimen (loading dose 3 mg/kg weekly for 4 weeks followed by maintenance dose 3 mg/kg every two weeks) for 24 weeks. |
|
|
Inclusion Criteria
|
| Age From |
2.00 Year(s) |
| Age To |
18.00 Year(s) |
| Gender |
Male |
| Details |
Severe Hemophilia A without Inhibitors and has not been on prophylactic therapy with either Extended Half Life Factor VIII or Emicizumab |
|
| ExclusionCriteria |
| Details |
Presence of FVIII inhibitors
Known case of coagulopathies and bleeding disorders other than Hemophilia A |
|
|
Method of Generating Random Sequence
|
Random Number Table |
|
Method of Concealment
|
Sequentially numbered, sealed, opaque envelopes |
|
Blinding/Masking
|
Open Label |
|
Primary Outcome
|
| Outcome |
TimePoints |
| To assess and compare Annualized Bleeding Rate ( spontaneous, Traumatic, Joint bleeds) between various treatment groups |
At baseline and end of 6 months of prophylaxis |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| Hemophilia Joint Health Score (HJHS) |
At baseline and and of 6 months |
| Haemo-QoL score |
At baseline and end of 6 months |
| Adverse events monitoring |
Any time during the study |
|
|
Target Sample Size
|
Total Sample Size="30"
Sample Size from India="30"
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 2 |
|
Date of First Enrollment (India)
|
15/11/2025 |
| 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="0"
Months="6"
Days="0" |
|
Recruitment Status of Trial (Global)
|
Not Yet Recruiting |
| Recruitment Status of Trial (India) |
Not Yet Recruiting |
|
Publication Details
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
This is a pilot randomized controlled open label trial comparing the efficacy and safety of prophylaxis with extended half life recombinant factor VIII and two dosing regimens of emicizumab in patients with hemophilia A without inhibitors. A total of 30 participants will be enrolled, with 10 participants in each group. Randomization will be done using random number tables, and allocation concealment will be maintained using sequentially numbered sealed envelopes. Each participant will receive the assigned prophylactic regimen for six months. The primary outcome will be the annualized bleeding rate. Secondary outcomes will include Hemophilia Joint Health Score, Haemo QoL score assessed at end of 6 months and adverse events recorded at any time during the study period.
|