Health Condition 1: D561- Beta thalassemia
Conditions
Interventions
Intervention1: Thalidomide plus Hydroxyurea add-on: Hydroxyurea up to 15 to 20 mg per kg per day [mkd] orally and Thalidomide up to 2- 3 mkd orally along with Aspirin 5mkd per day orally for 12 months
Sponsors
Indian Council of Medical research [ICMR], New Delhi
Eligibility
Inclusion criteria
Inclusion criteria: 1. Adults and children of any gender aged 12 years and above, willing to provide a written informed consent. 2. Diagnosis of TDT patients requiring regular blood transfusion for survival 3. Diagnosis of NTDT who is requiring at least 4-6 units of PRBC in the last 24 weeks working definition of NTDT for this study, which is jointly agreed by all investigators 4. Willing to follow the study protocol 5. Eastern Cooperative Oncology Group ECOG performance status 1-2
Exclusion criteria
Exclusion criteria: 1. Patients who are critically ill as judged by physician 2. Patients with comorbidities like severe heart or lung diseases, liver dysfunction, renal dysfunction, malignancy, cerebrovascular disease, or other serious primary diseases 3. Those with past personal or family history of thrombophilia and recent splenectomy within 3 months, 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. 4. Platelet count less than 100,000/mm3 or greater than 1,000,000/mm3 in the 8 weeks before study entry. 5. History of allergy to the drug ingredients 6. Participation in other drug clinical trials in the past 1 month 7. History of venous or arterial thrombosis 8. Known case of Human Immunodeficiency Virus, active Hepatitis C infection or Hepatitis B infection 9. Women who are pregnant or breast feeding or women of childbearing potential not willing to follow double contraceptive measures 10. Patients who are already receiving hydroxyurea/study drugs as standard of care 11. Any other reason that in the opinion of the investigator is likely to cause harm to the participant or will adversely affect the results of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| •Incidence of treatment emergent adverse events [TEAEs] of grade 3 or more severity at 12 monthsTimepoint: 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of patients with complete response (Maintenance of hemoglobin =9gm/dl without transfusion during the last 6 months while in the study)Timepoint: 6 months;Proportion of patients with partial response (At least 50% reduction in transfusion burden during the last 6 months)Timepoint: 6 months;Proportion of Non-responder (Patients with less than 50% transfusion burden reduction during the last 6 months of intervention)Timepoint: 6 months;To correlate response based on Genotype status & modifiers of Hemoglobin F inducersTimepoint: 12 months;Hemoglobin values & Ferritin levels at month 3, 6 & 12 monthsTimepoint: 12 months;Time to Transfusion after starting the intervention during the 12-month follow-upTimepoint: 12-month;Proportion of patients who are transfusion free for 12 weeks any time after 3 months of study interventionTimepoint: 3 months;Volume of blood transfused at the end ofTimepoint: 12 months.;The percentage of patients with peak hemoglobin increases more than 2g per dl 1-2g per dl & less than 1g per dl atTimepoint: 12 months;Change in cardiac relaxation time from baseline & at 12 months assessed using T2 Magnetic Resonance Imaging (MRI).Timepoint: 12 months;Determinants of clinical & hematological correlates of response at 12 monthsTimepoint: 12 months;The proportion of patients where iron chelator therapy could be reduced atTimepoint: 12 months;To correlate the trough concentrations of Hydroxyurea & Thalidomide at steady state with safety & efficacy parametersTimepoint: 12 months;Change in quality of life at 12 months as assessed by Transfusion-dependent Quality of Life (Tran QoL) questionnaire & the European Organisation for Research & Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) scoresTimepoint: 12 months | — |
Countries
India
Contacts
Public ContactDr Vijaya Chaudhari
Seth GSMC and KEM Hospital Mumbai
Outcome results
None listed