Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia (Thal-Thalido)

Blood Diseases Clinic

Status and phase: Completed; Phase 3; Phase 2

Conditions: Thalassemia

Treatments: Drug: Thalidomide and Hydroxyurea

Study type: Interventional

Funder types: Other

Identifiers

NCT03651102

TDT

Details and patient eligibility

About

Thalidomide is known to have hypnosedative, immuno-modulatory and anti-angiogenic effects. The drug is widely used in several neoplastic disorders (e.g. multiple myeloma and malignant melanoma), inflammatory conditions (e.g. Crohn's disease) and skin disorders (e.g. leprosy). Thalidomide has been successfully used in adult thalassemia patients. The current study explores its role in transfusion-dependent thalassemia patients.

Enrollment: 654 patients

Sex: All

Ages: 1+ year old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Transfusion dependent thalassemia patients refractory to Hydroxyurea

Exclusion criteria

• Those with active systemic comorbidity, with past personal or family history of thrombophilia, recent fracture or recent major surgery

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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654 participants in 1 patient group

Thalidomide in combination with hydroxyurea

Experimental group

Description:

All the study patients refractory to hydroxyurea will be given thalidomide at an average dose of 2mg/kg/day (range 1-4mg/kg/day). Participants will also be continued on hydroxyurea at a dose of 10-20 mg/kg/day.

Treatment:

Drug: Thalidomide and Hydroxyurea