| CTRI Number |
CTRI/2025/08/093906 [Registered on: 29/08/2025] Trial Registered Prospectively |
| Last Modified On: |
28/08/2025 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Randomized, Parallel Group, Active Controlled Trial |
|
Public Title of Study
|
To see the effect of drug Thalidomide in thalassemia |
|
Scientific Title of Study
|
A prospective study to understand the clinical efficacy, safety, and molecular mechanisms of thalidomide-induced erythropoiesis in patients with non-transfusion dependent thalassemia (NTDT) |
| Trial Acronym |
NA |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| NOT APPLICABLE |
NIL |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Biju George |
| Designation |
Professor |
| Affiliation |
Christian Medical College Vellore Ranipet campus, |
| Address |
Department of Haematology,
Room no 25, 5th Floor, A Block,
Ranipet
Vellore
TAMIL NADU
632517
India |
| Phone |
04172224480 |
| Fax |
|
| Email |
biju@cmcvellore.ac.in |
|
Details of Contact Person
Scientific Query
|
| Name |
Biju George |
| Designation |
Professor |
| Affiliation |
Christian Medical College Vellore Ranipet campus, |
| Address |
Department of Haematology,
Room no 25, 5th Floor, A Block,
Ranipet
TAMIL NADU
632517
India |
| Phone |
04172224480 |
| Fax |
|
| Email |
biju@cmcvellore.ac.in |
|
Details of Contact Person
Public Query
|
| Name |
Biju George |
| Designation |
Professor |
| Affiliation |
Christian Medical College Vellore Ranipet campus, |
| Address |
Department of Haematology,
Room no 25, 5th Floor, A Block,
Ranipet
TAMIL NADU
632517
India |
| Phone |
04172224480 |
| Fax |
|
| Email |
biju@cmcvellore.ac.in |
|
|
Source of Monetary or Material Support
|
| Indian Council of Medical Research,
V. Ramalingaswami Bhawan, P.O. Box No. 4911, Ansari Nagar, New Delhi - 110029 |
|
|
Primary Sponsor
|
| Name |
ndian Council of Medical Research |
| Address |
V. Ramalingaswami Bhawan, P.O. Box No. 4911, Ansari Nagar, New Delhi - 110029 |
| Type of Sponsor |
Other [Charitable trust hospital] |
|
|
Details of Secondary Sponsor
|
| Name |
Address |
| Not applicable |
Not applicable |
|
|
Countries of Recruitment
|
India |
|
Sites of Study
|
| No of Sites = 3 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Biju George |
Christian Medical College Vellore Ranipet campus |
Department of Haematology,
Room no 25, 5th Floor, A Block
Vellore
TAMIL NADU |
04172224480
biju@cmcvellore.ac.in |
| Dr Tuphan Kanti Dolai |
NRS Medical College and Hospital |
Hematology Department
NRS Medical College and Hospital
Kolkata-700014
Kolkata
WEST BENGAL |
9874890275
tkdolai75@gmail.com |
| Dr R K Jena |
S.C.B. Medical College, Hospital |
Department of Clinical Hematology,
S.C.B. Medical College
Hospital, Cuttack
Cuttack
ORISSA |
9437022343
rkjena@msn.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 3 |
| Name of Committee |
Approval Status |
| Ethics Committee NRS Medical College and Hospital |
Submittted/Under Review |
| Institutional Ethics Committee, SCB Medical College and Hospital |
Submittted/Under Review |
| Institutional Review Board, CMC Vellore |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: D758||Other specified diseases of bloodand blood-forming organs, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
Cap Thalidomide |
Dose: [50 mg/day for adults and 2 mg/kg/day for children] with prospective increase to 100 mg/day for adults and 5 mg/kg/day for children.
Duration: 12 months |
| Comparator Agent |
Hydroxyurea |
Dose: 500 mg daily or 10 mg/kg/day] with prospective dose increase to 20 mg/kg/day
Duration: 12 months |
|
|
Inclusion Criteria
|
| Age From |
1.00 Year(s) |
| Age To |
70.00 Year(s) |
| Gender |
Both |
| Details |
1. Patients diagnosed to have non-transfusion dependent thalassemia [NTDT]
2. Patients not on treatment/on treatment for less than 1 month
3. No major organ dysfunction [grade 3-4 heart, lungs, liver, kidneys] |
|
| ExclusionCriteria |
| Details |
1. Diagnosis of thalassemia major
2. Patients on treatment for more than 1 month
3. Unwilling to sign consent
|
|
|
Method of Generating Random Sequence
|
Computer generated randomization |
|
Method of Concealment
|
Centralized |
|
Blinding/Masking
|
Open Label |
|
Primary Outcome
|
| Outcome |
TimePoints |
| Improvement of hematological parameters and reducing transfusion frequency in patients with NTDT. |
Response assessments at 3,6 ,9 and 12 months of treatment |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| Identification of genetic markers that predict Thalidomide responsiveness in NTDT patients through comparative genomic analysis of responders and non-responders. |
Response assessments at 3,6 ,9 and 12 months of treatment |
|
|
Target Sample Size
|
Total Sample Size="194"
Sample Size from India="194"
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/ Phase 3 |
|
Date of First Enrollment (India)
|
10/09/2025 |
| 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="3"
Months="0"
Days="0" |
|
Recruitment Status of Trial (Global)
|
Not Applicable |
| 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
|
Introduction:
Non-transfusion dependent thalassemia [NTDT] is a milder form of thalassemia in
which patients require transfusions every 3-4 months. Hydroxyurea shows improvement
in 30-40% while thalidomide used as second line treatment has shown improvement
in 50%.
Methodology:
We would like to do perform a Phase II randomized controlled trial in which
newly diagnosed patients with NTDT will be randomized to receive either
Hydroxyurea or Thalidomide for a period of 12 months. At the end of 12 months,
patients will be classified as responders or non-responders. Patients will also
have samples collected to identify genetic markers that predict response to
thalidomide as well as study mechanisms
of thalidomide induced erythropoiesis.
Results:
We will try to study if thalidomide is a better drug than hydroxyurea for
reducing transfusion frequency in NTDT. In the same study, we will try and
identify predictors of response to thalidomide.
Conclusions: We hope to show
improved efficacy of thalidomide in patients with NTDT with a reduction in
transfusion frequency. We also hope to elucidate mechanisms for response to
thalidomide in patients with NTDT.
|