Clinical trial

Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia

Name

NIBD/IRB-266/01-2024

Description

The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing anemia. Conducted over two years at NIBD hospital, the study involves 54 transfusion-dependent patients aged 8-35. The primary objective is to correlate thalidomide doses with disease severity, adverse effects, and treatment response, aiming to optimize treatment strategies and reduce side effects. Data will be collected through clinical interviews and medical record reviews and analyzed using SPSS. Key variables include hemoglobin levels, leukocyte and reticulocyte counts, platelets, liver and spleen size, genetic modifiers, and transfusion frequency. Inclusion criteria are specific to beta thalassemia patients, while exclusion criteria rule out those with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis or fits.

Trial arms

Trial start

2024-04-22

Estimated PCD

2025-04-22

Trial end

2026-04-22

Status

Recruiting

Phase

Early phase I

Treatment

Thalidomide

Thalidomide is being investigated for its therapeutic efficacy and safety profile in transfusion-dependent beta thalassemia patients. Participants in this arm received thalidomide. Thalidomide was administered orally at a dosage of 5-6mg/kg/day. The medication was taken continuously or on a specified schedule (e.g., daily,) for the duration of the study period, which lasted 2 years. Participants were monitored for adherence to the medication regimen and for any adverse effects throughout the intervention period.

Arms:

Group 1 Thalidomide

Thalidomide

Thalidomide is being investigated for its therapeutic efficacy and safety profile in transfusion-dependent beta thalassemia patients. Participants in this arm received thalidomide. Thalidomide was administered orally at a dosage of 7-8mg/kg/day. The medication was taken continuously or on a specified schedule (e.g., daily,) for the duration of the study period, which lasted 2 years. Participants were monitored for adherence to the medication regimen and for any adverse effects throughout the intervention period.

Arms:

Group 2 Thalidomide

Size

Primary endpoint

transfusion dependency

1 year

Eligibility criteria

Inclusion Criteria: * Know case of beta thalassemia major/ intermediate ( transfusion dependent) * willing to give informed consent Exclusion Criteria: * Patients with comorbidities such as liver dysfunction * Married patients * Lactating mother * H/O thrombosis and fits

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'in this study three groups have been made. one is control group which received no intervention and on transfusion only. whereas other two groups are interventional both received thalidomide. one received thalidomide at 5-6mg/kg/day and second at 7-8 mg/kg/day.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 54, 'type': 'ESTIMATED'}}

Updated at

2024-07-08

1 organization

1 product

3 indications

Organization

National Institute of Blood and Marrow Transplant

Product

Indication

Indication

Indication