Home » Trials » SLCTR/2018/024
Efficacy and safety of oral hydroxyurea in transfusion dependent beta-thalassaemia: a randomized double-blind placebo-controlled clinical trial
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SLCTR Registration Number
SLCTR/2018/024
Date of Registration
The date of last modification
Mar 03, 2019
Scientific Title of Trial
Efficacy and safety of oral hydroxyurea in transfusion dependent beta-thalassaemia: a randomized double-blind placebo-controlled clinical trial
Public Title of Trial
Effectiveness of hydroxyurea treatment compared to placebo in reducing transfusions in beta-thalassaemia patients.
Disease or Health Condition(s) Studied
Beta-thalassaemia
Scientific Acronym
None
Public Acronym
None
Brief title
None
Universal Trial Number
U1111-1214-3595
Any other number(s) assigned to the trial and issuing authority
Ref. No: P/116/05/2018 -Ethics review committee, Faculty of Medicine, University of Kelaniya
What is the research question being addressed?
Is hydroxyurea safe and effective in reducing transfusion requirement in patients with transfusion dependent beta-thalassaemia?
Type of study
Interventional
Study design
Allocation
Randomized controlled trial
Masking
Double blinded
Control
Placebo
Assignment
Parallel
Purpose
Treatment
Study Phase
Phase 2-3
Intervention(s) planned
Place of study will be Adolescent and Adult Thalassaemia Center of the Colombo North Teaching Hospital, Ragama, Sri Lanka.
Method of randomization will be simple randomization using an online simple randomization tool. Subjects will be randomized to two arms
Active arm treated with hydroxyurea
Control arm treated with placebo (This is a matching capsule which contains ingredients identical to hydroxyurea except for the active ingredient).
Investigational product [Active hydroxyurea] will be administered at a dose of 500mg (if body weight =<50kg) or 1000mg (if body weight >50kg) orally by subject once daily for six months. Placebo will also be administered once daily for six months.
The following will be blinded; participants, data collectors, outcome adjudicators and data analysts
Inclusion criteria
Exclusion criteria
Primary outcome(s)
|
1.
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. 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. Subjects will be evaluated monthly during the period of treatment by full blood count measurements. |
[ At the end of six months of initiating treatment. ] |
Secondary outcome(s)
|
1.
Compliance to hydroxyurea treatment (measured as actual number of tablets taken divided by the estimated number of tablets to be taken). |
[ Compliance to hydroxyurea treatment will be assessed monthly for 6 months after initiating treatment ] |
|
2.
Side effects of hydroxyurea 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. |
[ Side effects will be assessed monthly for 12 months after initiating treatment ] |
Target number/sample size
90 (45 in each arm)
Countries of recruitment
Sri Lanka
Anticipated start date
2018-09-01
Anticipated end date
2020-02-28
Date of first enrollment
Date of study completion
Recruitment status
Pending
Funding source
National Research Council- Sri Lanka. Investigator Driven Grant # 18-030
Regulatory approvals
Not applicable
Status
Approved
Date of Approval
2018-06-26
Approval number
P/116/05/2018
Details of Ethics Review Committee
| Name: | Ethics Review Committee, Faculty of Medicine, University of Kelaniya |
| Institutional Address: | PO Box 6, Thalagolla Road, Ragama Sri Lanka |
| Telephone: | +94-11-2961267 |
| Email: | erckelaniya@gmail.com |
Contact person for Scientific Queries/Principal Investigator
Dr Sachith Mettananda
Senior Lecturer and Consultant Paediatrician
Department of Paediatrics
Faculty of Medicine- University of Kelaniya
Thalagolla Road
Ragama
+94112961116
+94714805725
sachith.mettananda@kln.ac.lk
Contact Person for Public Queries
Dr Sachith Mettananda
Senior Lecturer and Consultant Paediatrician
Department of Paediatrics
Faculty of Medicine- University of Kelaniya
Thalagolla Road
Ragama
+94112961116
+94714805725
sachith.mettananda@kln.ac.lk
Primary study sponsor/organization
Faculty of Medicine - University of Kelaniya
Thalagolla Road, Ragama,
Sri Lanka.
Tel: +94112961116
Fax: 0112958337
Email: sachith.mettananda@kln.ac.lk
Website: http://www.kln.ac.lk
Do the investigators plan to share identified individual clinical trial participant-level data (IPD)?
IPD sharing plan description
Not Available
Study protocol available
Protocol version and date
Not Available
Protocol URL
Not Available
Results summary available
No
Date of posting results
Date of study completion
Final sample size
Date of first publication
Link to results
Brief summary of results
