Thalidomide for patients with Thalassemia major

Study of efficacy of Low dose vs Standard dose of Thalidomide in patients with transfusion dependent thalassemia (TDT): a non-inferiority trial

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

CTRI

Registry ID

CTRI/2022/05/042781

Enrollment

226

Registered

2022-05-24

Start date

Unknown

Completion date

Unknown

Last updated

2022-10-17

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: Thalidomide: Whether low dose of thalidomide (1mg/kg/day) is as efficacious as standard dose (2mg/kg/day) Intervention2: Thalidomide : Standard dose arm (2mg/kg) per orally once at bed

Eligibility

Inclusion criteria

Inclusion criteria: 1. Any diagnosed patient of TDT who is between age 12-25 years 2. With no current or recent (in past 6 months) of any regular specific drug therapy for hemoglobinopathy 3. Records available to ascertain mean Hb, transfusion frequency and transfusion volumes recieved in preceding 12 weeks period

Exclusion criteria

Exclusion criteria: 1. Weight less than 25 kg or more than 75 kg 2. Known case with HIV, active HCV or Hepatitis B infection 3. Those with any other known systemic illness or known thromboembolic phenomenon 4. Current evidence of hypersplenism 5. Not consenting for contraception if cohabiting 6. Not willing for MTP in case get pregnant while on therapy

Design outcomes

Primary

Measure	Time frame
compare the efficacy of Low dose (1 mg/kg daily) with standard dose (2 mg/kg daily) of thalidomide in reducing transfusion requirement in patients with TDTTimepoint: Baseline Hemoglobin, at 12 weeks and at 24 weeks	—

Secondary

Measure	Time frame
Compare ferritin level at 24 weeks and 72 weeks with baseline value in either armsTimepoint: at 24 weeks and 72 weeks;Compare proportion of patients necessitating temporary or permanent cessation of drugsTimepoint: at 24 weeks and 72 weeks;Compare proportion of patients with Gr 3/ 4 AEs in both groupsTimepoint: at 24 weeks and 72 weeks;Proportion of patients where iron chelator therapy could be reduced in either arm.Timepoint: at 24 weeks;Proportion of subjects achieving a fair or good response in the two arms at the end of 72 weeksTimepoint: end of 72 weeks;Proportion of subjects having no or poor response in the two arms at the end of 72 weeks Timepoint: end of 72 weeks;Time to response in either group (when the treating centre decides to withhold due date transfusion will be considered beginning of response).Timepoint: at 24 weeks	—

Measure

Time frame

Compare ferritin level at 24 weeks and 72 weeks with baseline value in either armsTimepoint: at 24 weeks and 72 weeks;Compare proportion of patients necessitating temporary or permanent cessation of drugsTimepoint: at 24 weeks and 72 weeks;Compare proportion of patients with Gr 3/ 4 AEs in both groupsTimepoint: at 24 weeks and 72 weeks;Proportion of patients where iron chelator therapy could be reduced in either arm.Timepoint: at 24 weeks;Proportion of subjects achieving a fair or good response in the two arms at the end of 72 weeksTimepoint: end of 72 weeks;Proportion of subjects having no or poor response in the two arms at the end of 72 weeks Timepoint: end of 72 weeks;Time to response in either group (when the treating centre decides to withhold due date transfusion will be considered beginning of response).Timepoint: at 24 weeks

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Countries

India

Contacts

Public ContactJagdish Chandra

Postgraduate Institute of Medical Sciences and Research & ESIC Model Hospital

jchandra55@gmail.com9968568905

Outcome results

None listed

Source: CTRI (via WHO ICTRP) · Data processed: Sep 3, 2026