Effectiveness of hydroxyurea treatment compared to placebo in reducing transfusions in beta-thalassaemia patients.

Phase 2

Conditions: Beta-thalassaemia

Registration Number: SLCTR/2018/024

Lead Sponsor: Faculty of Medicine - University of Kelaniya

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Pending

Sex: Not specified

Target Recruitment: Not specified

Inclusion Criteria

1. Male and female patients with confirmed genotype of homozygous or compound heterozygous beta-thalassaemia major or HbE beta-thalassaemia
2. Age above 12 years
3. Patients who required more than 10 transfusions during the preceding year

Exclusion Criteria

1. Patients who have undergone bone marrow transplantation
2. Patients with very high serum ferritin (>5000ng/ml)
3. Sickle beta-thalassaemia
4. Chronic liver disease
5. Chronic kidney disease
6. Patients with contraindications for hydroxyurea therapy (eg: hypersensitivity, severe anaemia with haemoglobin less than 5g/dl at the beginning of treatment, bone marrow depression, pregnancy and lactation)
7. Patients who are expecting to get pregnant during next 12 months
8. White blood cell count less than 4000/uL.
9. Platelet count less than 150,000/uL.
10. Evidence of active viral infection, including viral hepatitis
11. Patients on immunosuppressant therapy

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Response to treatment: Complete response to treatment is defined as complete cessation of blood transfusions with a baseline haemoglobin >9.0g/dl during the treatment period.<br>Partial response is defined as over 50% reduction in transfusion requirement during treatment period (=<3 transfusions during treatment 6 months) with baseline haemoglobin >7.0g/dl.<br><br>Subjects will be evaluated monthly during the period of treatment by full blood count measurements.<br><br><br><br> [At the end of six months of initiating treatment.]<br>

Secondary Outcome Measures

Name	Time	Method
Compliance to hydroxyurea treatment (measured as actual number of tablets taken divided by the estimated number of tablets to be taken).<br><br><br> [Compliance to hydroxyurea treatment will be assessed monthly for 6 months after initiating treatment<br><br><br>]<br>Side effects of hydroxyurea<br><br>Subjects will be evaluated monthly during the period of treatment. Evaluation includes an interview and physical examination by a trained medical officer to assess adverse effects of hydroxyurea. Following investigations will also be done to assess side effects of hydroxyurea; full blood count, serum creatinine and liver transaminases. <br> [ Side effects will be assessed monthly for 12 months after initiating treatment]<br>