Use of medicines- Hydroxyurea and thalidomide for children suffering from thalassemia

Safety and efficacy of combination of thalidomide and hydroxyurea in children with transfusion dependent thalassemia: A phase III, multicentric, double-blind randomized placebo-controlled trial. - NIL

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

CTRI

Registry ID

CTRI/2023/12/060945

Enrollment

150

Registered

2023-12-29

Start date

Unknown

Completion date

Unknown

Last updated

2025-08-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Health Condition 1: D561- Beta thalassemia

Interventions

Intervention1: Hydroxyurea, thalidomide, aspirin: Drugs and dose: Thalidomide (2.5-3.5 mg/kg/day), Hydroxyurea (15 mg/ kg/ day) and Aspirin (3-4 mg/ kg/ day)- rounded off to nearest available dose Rou

Eligibility

Inclusion criteria

Inclusion criteria: a. Children aged 2-18 years. b. Diagnosis of transfusion dependent thalassemia, requiring regular blood transfusions. c. Willing for regular follow up as required for the study d. Informed consent given e. Patients with child bearing potential must be willing for contraception (wherever applicable)

Exclusion criteria

Exclusion criteria: a. Patients with hypersplenism and planning splenectomy during the next year b. Patients planning for hematopoietic stem cell transplantation (HSCT) c. Retroviral infection d. Chronic active hepatitis (Hepatitis B or Hepatitis C) e. Elevation of transaminases more than 5 times of upper limit of normal f. Renal failure g. Symptomatic cardiac failure and LVEF h. Past history of stroke i. Received hydroxyurea, thalidomide or luspatercept in the previous 6 months j. Pregnancy or lactation

Design outcomes

Primary

Measure	Time frame
Proportion of patients with transfusion dependent thalassemia achieving erythroid response (Defined as at least 40% reduction in transfusion requirement from baseline during 3 consecutive months)Timepoint: 3 months, 6 months, 9 months and 12 months	—

Secondary

Measure	Time frame
Safety of Hydroxyurea and thalidomide in children with transfusion dependent thalassemiaTimepoint: 3 months, 6 months, 9 months and 12 months;Proportion of patients who are transfusion free for a period of at least 3 months.Timepoint: 3 months, 6 months, 9 months and 12 months;Determinants and predictors of patients having response to hydroxyurea and thalidomideTimepoint: 3 months, 6 months, 9 months and 12 months	—

Measure

Time frame

Safety of Hydroxyurea and thalidomide in children with transfusion dependent thalassemiaTimepoint: 3 months, 6 months, 9 months and 12 months;Proportion of patients who are transfusion free for a period of at least 3 months.Timepoint: 3 months, 6 months, 9 months and 12 months;Determinants and predictors of patients having response to hydroxyurea and thalidomideTimepoint: 3 months, 6 months, 9 months and 12 months

—

Countries

India

Contacts

Public ContactDr DEBASISH SAHOO

AIIMS, Bhubaneswar

debasish0712@gmail.com8872028232

Outcome results

None listed

Source: CTRI (via WHO ICTRP) · Data processed: Feb 4, 2026