| CTRI Number |
CTRI/2020/05/025142 [Registered on: 13/05/2020] Trial Registered Prospectively |
| Last Modified On: |
05/05/2020 |
| Post Graduate Thesis |
Yes |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Randomized, Parallel Group, Multiple Arm Trial |
|
Public Title of Study
|
LOW DOSE THALIDOMIDE VERSUS HYDROXYUREA IN TRANSFUSION DEPENDENT THALASSEMIA PATIENTS |
|
Scientific Title of Study
|
EFFICACY AND SAFETY OF LOW DOSE THALIDOMIDE VERSUS HYDROXYUREA IN TRANSFUSION DEPENDENT THALASSEMIA PATIENTS: A RANDOMIZED CONTROLLED TRIAL |
| Trial Acronym |
THY |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| NIL |
NIL |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
ALKA RANI KHADWAL |
| Designation |
ADDITIONAL PROFESSOR |
| Affiliation |
POSTGRADUATE INSTITUTE OF MEDICAL EDUCATION AND RESEARCH CHANDIGARH |
| Address |
DEPARTMENT OF INTERNAL MEDICINE, CLINICAL HEMATOLOGY UNIT, NEHRU HOSPITAL LEVEL 4 F BLOCK POSTGRADUATE INSTITUTE OF MEDICAL EDUCATION AND RESEARCH CHANDIGARH
Chandigarh
CHANDIGARH
160012
India |
| Phone |
7087008079 |
| Fax |
|
| Email |
alkakhadwal@hotmail.com |
|
Details of Contact Person
Scientific Query
|
| Name |
ALKA RANI KHADWAL |
| Designation |
ADDITIONAL PROFESSOR |
| Affiliation |
POSTGRADUATE INSTITUTE OF MEDICAL EDUCATION AND RESEARCH CHANDIGARH |
| Address |
DEPARTMENT OF INTERNAL MEDICINE, CLINICAL HEMATOLOGY UNIT, NEHRU HOSPITAL LEVEL 4 F BLOCK POSTGRADUATE INSTITUTE OF MEDICAL EDUCATION AND RESEARCH CHANDIGARH
CHANDIGARH
160012
India |
| Phone |
7087008079 |
| Fax |
|
| Email |
alkakhadwal@hotmail.com |
|
Details of Contact Person
Public Query
|
| Name |
URMIMALA BHATTACHARJEE |
| Designation |
SENIOR RESIDENT |
| Affiliation |
POSTGRADUATE INSTITUTE OF MEDICAL EDUCATION AND RESEARCH CHANDIGARH |
| Address |
DEPARTMENT OF CLINICAL HEMATOLOGY POSTGRADUATE INSTITUTE OF MEDICAL EDUCATION AND RESEARCH CHANDIGARH
Chandigarh
CHANDIGARH
160012
India |
| Phone |
6284059491 |
| Fax |
|
| Email |
drub200954@gmail.com |
|
|
Source of Monetary or Material Support
|
| POSTGRADUATE INSTITUTE OF MEDICAL EDUCATION AND RESEARCH CHANDIGARH |
|
|
Primary Sponsor
|
| Name |
ALKA RANI KHADWAL |
| Address |
DEPARTMENT OF INTERNAL MEDICINE NEHRU HOSPITAL LEVEL 4 F BLOCK POSTGRADUATE INSTITUTE OF MEDICAL EDUCATION AND RESEARCH CHANDIGARH |
| Type of Sponsor |
Other [Self] |
|
|
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 |
| URMIMALA BHATTACHARJEE |
POSTGRADUATE INSTITUTE OF MEDICAL EDUCATION AND RESEARCH CHANDIGARH |
POSTGRADUATE INSTITUTE OF MEDICAL EDUCATION AND RESEARCH CHANDIGARH
Chandigarh
CHANDIGARH |
6284059491
drub200954@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 1 |
| Name of Committee |
Approval Status |
| The Institutional Ethics Committee of PGIMER, Chandigarh |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: D569||Thalassemia, unspecified, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
1 |
HYDROXYUREA 500 mg O.D orally for 6 months |
| Intervention |
2 |
THALIDOMIDE 50 mg O.D orally along with Tab Aspirin 75mg O.D orally for 6 months |
| Comparator Agent |
3 |
STANDARD OF CARE |
|
|
Inclusion Criteria
|
| Age From |
18.00 Year(s) |
| Age To |
60.00 Year(s) |
| Gender |
Both |
| Details |
1. Treatment naive adult transfusion dependent thalassemia patients of either sex willing to give written informed consent.
2. Age more than 18 years.
|
|
| ExclusionCriteria |
| Details |
1. Those with active systemic comorbidity, with past personal or family history of thrombophilia and recent splenectomy (within 3 months).
2. Absolute neutrophil count (ANC) less than 2000/ mm3 in the 8 weeks before study entry or a history of chronic neutropenia, defined as an ANC less than 2000/mm3.
3. Platelet count less than 100,000/mm3 or greater than 1,000,000/mm3 in the 8 weeks before study entry.
4. Evidence of liver disease, as defined by one or more of the following conditions: Alanine aminotransferase (ALT) level greater than three times the upper limit of normal in the 8 weeks before study entry. Serum albumin level less than 3 g/dL in the 8 weeks before study entry.
5. Creatinine level more than twice the upper limit of normal for age or greater than 1.5mg/dl.
6. Pregnant, planning to become pregnant, or breastfeeding.
7. HCV/HBV/HIV infection.
8. Currently being treated with any other experimental or fetal hemoglobin-modulating agent.
9. Current participation in any other studies of investigational drugs or devices.
10. Baseline peripheral neuropathy symptoms equivalent to grade 2 Therapy-induced peripheral neuropathy (TiPN) will be excluded.
|
|
|
Method of Generating Random Sequence
|
Permuted block randomization, fixed |
|
Method of Concealment
|
Not Applicable |
|
Blinding/Masking
|
Open Label |
|
Primary Outcome
|
| Outcome |
TimePoints |
| 1. Change in hemoglobin levels |
at 3 months and 6 months |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
1. Average time to repeat transfusion over 6 months follow up.
2. Average volume of blood transfused over a period of 6 months compared to previous
6 months.
3. Percentage of patients with peak haemoglobin increase more than 2g/dl; 1-2 g/dl and less than 1g/dl.
4. Determinants of clinical (organomegaly and extramedullary hematopoiesis masses) and haematological correlates of response.
5. Incidence of severe adverse events or serious adverse events.
|
1. Average time to repeat transfusion over 6 months follow up.
2. Average volume of blood transfused over a period of 6 months compared to previous
6 months.
3. Percentage of patients with peak haemoglobin increase more than 2g/dl; 1-2 g/dl and less than 1g/dl.
4. Determinants of clinical (organomegaly and extramedullary hematopoiesis masses) and haematological correlates of response.
5. Incidence of severe adverse events or serious adverse events. |
|
|
Target Sample Size
|
Total Sample Size="90"
Sample Size from India="90"
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)
|
15/05/2020 |
| 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
|
Not published |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
|
|
Brief Summary
|
Transfusion dependent thalassemia (TDT) patients constitutes a major disease burden of Indian subcontinent. Till now regular transfusion and adequate chelation forms the main backbone of treatment. There is a worldwide search for newer therapies to decrease the transfusion dependence in TDTs nowadays. Out study is a randomized controlled trial comparing low dose thalidomide (50mg), hydroxyurea (500mg) and standard of care arm. Our study is based on the fact that low dose thalidomide and hydroxyurea have been found in multiple case reports, single arm retrospective trials individually and in combination to increase haemoglobin levels in thalassemia and decrease transfusion dependence with no serious side-effects causing hospitalisation or death. It will be a prospective open label randomized controlled trial with blinded end-point study design. Eligible patients with be randomized to either hydroxyurea, thalidomide or standard of care arm. Patients will be regularly followed up at monthly intervals for observing rise in haemoglobin and side-effects if any. Sample size required per group is 27 patients. Estimated duration of trial is 3 years. |