| CTRI Number |
CTRI/2009/091/001059 [Registered on: 08/01/2010] |
| Last Modified On: |
04/09/2015 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
Type of Study
Modification(s)
|
Biological |
| Study Design |
Randomized, Parallel Group, Placebo Controlled Trial |
Public Title of Study
Modification(s)
|
Research Trial to Study inhibitor development in previously untreated or minimally exposed Children to two different commercially available types of Factor VIII.
This is Phase IV open lable , randomized,Study comparing Plasma derived Factor VIII with Recombinant Factor VIII. |
Scientific Title of Study
Modification(s)
|
Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomized, Open Label, Clinical Trial. |
| Trial Acronym |
Nil |
Secondary IDs if Any
Modification(s)
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| Secondary ID |
Identifier |
| ABB-09-001 |
Protocol Number |
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
Modification(s)
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| Name |
|
| Designation |
|
| Affiliation |
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| Address |
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| Phone |
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| Fax |
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| Email |
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Details of Contact Person
Scientific Query
Modification(s)
|
| Name |
Dr Subhranshu Nayak |
| Designation |
Medical Monitor |
| Affiliation |
Max Neeman International |
| Address |
Max Neeman International
Max House, 1st Floor
1, Dr. Jha Marg, Okhla Phase-III
New Delhi-110020
New Delhi
DELHI
110020
India |
| Phone |
011-40772100 |
| Fax |
91-11-26322846 |
| Email |
Subhranshu.Nayak@maxneeman.com |
|
Details of Contact Person
Public Query
Modification(s)
|
| Name |
Dr Shariq Anwar |
| Designation |
Head Operations |
| Affiliation |
Max Neeman International |
| Address |
Max Neeman International
Max House, 1st Floor
1, Dr. Jha Marg, Okhla Phase-III
Pin Code-110020
New Delhi
DELHI
110020
India |
| Phone |
91-11-40772100 |
| Fax |
91-11-40508168 |
| Email |
Shariq.Anwar@maxneeman.com |
|
Source of Monetary or Material Support
Modification(s)
|
| Fondazione Angelo Bianchi Bonomi (FABB) |
|
Primary Sponsor
Modification(s)
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| Name |
Sintesi Research |
| Address |
via Ripamonti 89
20141 Milano
Italy
|
| Type of Sponsor |
Contract research organization |
|
Details of Secondary Sponsor
Modification(s)
|
| Name |
Address |
| Max Neeman International |
Max House, 1, Dr. Jha Marg
Okhla Phase III
New Delhi - 110020
|
|
Countries of Recruitment
Modification(s)
|
Fiji
Argentina
Austria
Belgium
Brazil
Chile
Czech Republic
Denmark
Egypt
France
Germany
India
Iran (Islamic Republic of)
Italy
Mexico
Poland
Portugal
Romania
Saudi Arabia
Slovakia
South Africa
Spain
Switzerland
Turkey
United Kingdom
United States of America
Uruguay |
Sites of Study
Modification(s)
|
| No of Sites = 10 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Tulika Seth |
All India Institute of Medical Sciences |
Department of Hematology Ansari Nagar-110029, India
New Delhi
DELHI |
91-9868397236
91-11-26588031
tuliseth@yahoo.com |
| Dr Ramabadran Varadarajan |
Centre for Blood Disorder |
Habibullah Road, T. Nagar-600017, India
Chennai
TAMIL NADU |
91-9840119052
91-44-28344846
rvraj12345@gmail.com |
| Dr Mathew Thomas |
Health and Research Centre |
T.C.1/1668, Yamuna navarangam Lane-North-2, Medical College P.O. Pin Code-695011, India
Thiruvananthapuram
KERALA |
91-9447753533
91-471-2554913
amnavita@gmail.com |
| Dr Vijay Ramanan Madatha |
Jehangir Clinical Development Centre |
Jehangir Hospital Premises, 32, Sassoon Road, Pin Code-411001, India
Pune
MAHARASHTRA |
91-9325315471
91-11-26588031
mvijayr@gmail.com |
| Dr Suresh Hanagavadi |
Karnataka Hemophilia Society |
Karnataka Hemophilia Society , Davanagere Chapter No.1138, Behind KSFC, Ring Road, S.Nijalingappa Layout, Pin Code-577004
Davanagere
KARNATAKA |
91-8722209404
91-8192-231948
drhanagavadi@gmail.com |
| Dr Dinesh Nayak |
Kasturba Medical College |
Kasturba Medical College, Pin Code-576104
Udupi
KARNATAKA |
91-9845226437
91-820-2571934
dinesh_nayak@yahoo.com |
| Dr Mamta Vijay Manglani |
Lokmanya Tilak Municipal Medical college and general hospital |
Sion-400022, India
Mumbai
MAHARASHTRA |
91-9821322071
91-22-24086150
mmanglani@hotmail.com |
| Dr Shashikant Apte |
Sahyadri Speciality Hospital |
30 C,Erandawane, Karve Road, Pin Code-411004, India
Pune
MAHARASHTRA |
91-9822404983
91-20-25459117
shashikant.apte@gmail.com |
| Dr Anupam Sachdeva |
Sir Ganga Ram Hospital |
Sir Ganga Ram Marg, Rajinder Place, Pin Code-110060, India
New Delhi
DELHI |
91-9811043476
91-11-25861002
anupamace@yahoo.co.in |
| Dr Cecil Ross |
St. Johns Medical College & Hospital |
Sarjapur Road, Pin Code-560034, India
Bangalore
KARNATAKA |
91-9448493705
91-80-25501144
cecilross@bsnl.in |
|
Details of Ethics Committee
Modification(s)
|
| No of Ethics Committees= 10 |
| Name of Committee |
Approval Status |
| Ethics Committee, Sir Ganga Ram Hospital, Sir Ganga Ram Marg, Rajinder Nagar, New Delhi-110060, India |
Approved |
| CLINICOM, “SUSHRUTAâ€, No-1/1, Ist Temple road, 15th Cross, Malleswaram, Bangalore 560 003, Karnataka , India |
Approved |
| Ethics Committee All India Institute Of Medical Science, Ansari Nagar, New Delhi- 110029, India |
Approved |
| Independent Human Ethical Committee, Health and Research T.C.1/1668 , Yamuna navarangam Lane-North-2, Medical College P.O., Trivandrum-695011, India |
Approved |
| Institutional Ethical Review Board, St. Johns Medical College & Hospital, Sarjapur Road, Bangalore-560034, Karnataka, India |
Approved |
| Institutional Ethics Committee-Human Research LTMMC, LTMGH, L.T.M.Medical College building, 2nd Floor, Room Number 17, Sion, Mumbai-400022, India |
Approved |
| Jehangir Clinical Development Center Institutional Review Board– Jehangir Hospital Premises, 32 Sassoon Road, Pune – 411001, Maharashtra, India |
Approved |
| Karnataka Hemophilia Society Ethics Committee, Chapter No.1138, Behind KSFC, Ring Road, S.Nijalingappa Layout, Davanagere – 577004, Karnataka |
Approved |
| Manipal University Ethics Committee Madhav Nagar, Manipal-576104, Karnataka, India |
Approved |
| Sahyadri Ethics Committee, Sahyadri Speciality Hospital, 30 C, Erandawane, Karve Road, Pune- 411004 |
Approved |
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Regulatory Clearance Status from DCGI
Modification(s)
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Health Condition / Problems Studied
Modification(s)
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| Health Type |
Condition |
| Patients |
Severe Hemophilia A, |
|
Intervention / Comparator Agent
Modification(s)
|
| Type |
Name |
Details |
| Comparator Agent |
None |
None |
| Intervention |
Plasma Derived Product : Emoclot |
Therapeutic Regimen for on demand treatment - Administered through a dose of 40 IU/kg at bleeding occurrence,followed by 20IU/kg at 24 and 72 hours
Prophylaxis Dosage - Administered through a dose of 25-40 IU/kg, 3-4 times a week
Pereoperative Management Administered through a dose of 50IU/kg preoperatively followed by 30-50 IU/kg to repeat aiming to maintain Plasma FVIII levels above 40-50% |
| Intervention |
Recombinate Product: Recombinate |
Therapeutic Regimen for on demand treatment - Administered through a dose of 40 IU/kg at bleeding occurrence, followed by 20IU/kg at 24 and 72 hours
Prophylaxis Dosage - Administered through a dose of 25-40 IU/kg, 3-4 times a week
Preoperative Management Administered through a dose of 50IU/kg preoperatively followed by 30-50 IU/kg to repeat aiming to maintain Plasma FVIII levels above 40-50% |
|
Inclusion Criteria
Modification(s)
|
| Age From |
0.00 Year(s) |
| Age To |
6.00 Year(s) |
| Gender |
Male |
| Details |
1. Male subjects
2. Any ethnicity
3. Age 6 years
4. Severe haemophilia A (FVIII:C 1%), as confirmed by the central laboratory
5. A Patient with FVIII levels 1% and 2% will be separately recorded in the screening list
6. Previously untreated (0 EDs to any FVIII concentrate or blood products) or minimally treated (5 EDs) with blood components, namely whole blood, fresh frozen plasma, packed red blood cells, platelets or cryoprecipitate
7. A Patient not meeting these criteria will be separately recorded in the screening list.
8. Negative inhibitor measurement at both local and central laboratory at screening
9. Ability to comply with study requirements
10. Signed informed consent of legal tutors |
|
| ExclusionCriteria |
| Details |
1. Plasma FVIII level ≥1%, as assayed at the central laboratory those patients originally diagnosed locally as severe but subsequently found to have FVIII levels ranging from 1% to 2% on testing at the central laboratory will be separately recorded in the screening list.
2. Previous history of FVIII inhibitor
3. Other congenital or acquired bleeding defects
4. Concomitant congenital or acquired immunodeficiencies
5. Concomitant treatment with systemic immunosuppressive drugs
6. Concomitant treatment with any investigational drug |
|
Method of Generating Random Sequence
Modification(s)
|
Other |
Method of Concealment
Modification(s)
|
Pre-numbered or coded identical Containers |
Blinding/Masking
Modification(s)
|
Open Label |
Primary Outcome
Modification(s)
|
| Outcome |
TimePoints |
| To assess the immunogenicity of VWF/FVIII and of rFVIII concentrates by determining the frequency of inhibitor development in PUPs and MBCTs in the first 50 EDs or in the first 3 years from enrolment, whichever comes first. |
Enrollment visit,Every 3-4 EDs, Every 10 EDs,Every 3 months, Annual visit,Final visit |
|
Secondary Outcome
Modification(s)
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| Outcome |
TimePoints |
To evaluate the anamnestic response of inhibitor patients
|
Enrollment visit,Every 3-4 EDs, Every 10 EDs, Every 3 months, Annual visit, final visit. |
| To evaluate the frequency of transient inhibitors. |
Enrollment visit,Every 3-4 EDs, Every 10 EDs, Every 3 months, Annual visit, final visit. |
| To evaluate the modality of occurrence of inhibitors (number of EDs, titre at onset, etc) |
Enrollment visit,Every 3-4 EDs, Every 10 EDs, Every 3 months, Annual visit, final visit. |
| To evaluate the incidence of all other adverse events related and not related to the products used. |
Enrollment visit,Every 3-4 EDs, Every 10 EDs, Every 3 months, Annual visit, final visit. |
| To evaluate the bleeding history (age at first bleed, bleeding pattern) |
Enrollment visit,Every 3-4 EDs, Every 10 EDs, Every 3 months, Annual visit, final visit. |
|
Target Sample Size
Modification(s)
|
Total Sample Size="300"
Sample Size from India="100"
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
Modification(s)
|
Phase 4 |
Date of First Enrollment (India)
Modification(s)
|
10/02/2010 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
15/01/2010 |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
Estimated Duration of Trial
Modification(s)
|
Years="3"
Months="0"
Days="0" |
Recruitment Status of Trial (Global)
Modification(s)
|
Closed to Recruitment of Participants |
| Recruitment Status of Trial (India) |
Closed to Recruitment of Participants |
Publication Details
Modification(s)
|
None as yet |
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Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
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Brief Summary
Modification(s)
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The study is an international, multicenter, prospective, controlled, randomised, open-label clinical trial on inhibitor frequency in patients previously untreated (PUPs) or minimally blood component-treated (MBCTPs) when exposed to plasma-derived, von Willebrand factor-containing factor VIII (VWF/FVIII) concentrates or to recombinant factor VIII (rFVIII) concentrates. Patients meeting the enrolment criteria will be consecutively enrolled at each participating centre, randomised to be treated exclusively with a single FVIII product either plasma-derived and containing VWF or recombinant, and followed up until inhibitor development or until 50 exposure days (EDs) or 3 years from enrolment have elapsed, whichever comes first. Study products, belonging to the class of recombinant FVIII concentrates and to the class of VWF-containing FVIII concentrates, will be provided for free to the patients for all the duration of the study. A screening list will be held with consecutive patients that cannot be enrolled and with the reason of exclusion (refusal to enter the study, refusal of randomization, already exposed for 5 or more EDs to blood components or for less than 5 EDs to FVIII concentrates, FVIII levels ≥1). |