Home » Trials » SLCTR/2025/034
Efficacy of Hydroxyurea and Thalidomide in the Management of Beta Thalassemia
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SLCTR Registration Number
SLCTR/2025/034
Date of Registration
The date of last modification
Aug 29, 2025
Scientific Title of Trial
Efficacy of Hydroxyurea and Thalidomide in the Management of Beta Thalassemia
Public Title of Trial
Effectiveness of combined Hydroxyurea and Thalidomide in Transfusion-Dependent Beta Thalassemia Patients for Reducing Blood Transfusion Requirements: A Prospective Single-Arm Trial.
Disease or Health Condition(s) Studied
Beta Thalassemia
Scientific Acronym
None
Public Acronym
None
Brief title
Efficacy of Hydroxyurea and Thalidomide in the Management of Beta Thalassemia.
Universal Trial Number
U1111-1318-1358
Any other number(s) assigned to the trial and issuing authority
ERC/2025/PAEDS/14 (PNS Shifa hospital)
What is the research question being addressed?
What is the comparative efficacy of Hydroxyurea and Thalidomide in the management of beta-thalassemia in terms of clinical outcomes, transfusion dependency, and quality of life?
Type of study
Interventional
Study design
Allocation
Single arm study
Masking
Masking not used
Control
Uncontrolled
Assignment
Single
Purpose
Treatment
Study Phase
Phase 4
Intervention(s) planned
Participants in the single-arm study will receive combination therapy with Hydroxyurea plus Thalidomide. Hydroxyurea will be given via oral route [capsule form], once daily every 24 hours at a dose of 15mg/kg/day. Thalidomide will be given via oral route [capsule form], once daily at night every 24 hours at a dose of 50mg/day for patients aged 10 to 13 years, and 100mg/day for patients aged above 13 years. Both medications will be administered concurrently for a total duration of 6 months.
Inclusion criteria
Male and female patients with major thalassemia B type within the age range of 20 to 50 years.
Exclusion criteria
Primary outcome(s)
|
1.
Change in Hb gm/dl. |
[ 3 and 6 months after initiation of treatment ] |
|
2.
Change in Blood transfusion frequency. |
[ 3 and 6 months after initiation of treatment ] |
|
3.
Change in Hb F. |
[ 3 months after initiation of treatment ] |
Secondary outcome(s)
|
1.
Transfusion dependence will be assessed by calculating the total Packed Red Blood Cell volume (mL/kg) transfused during the 6-month period before treatment initiation and during the 6-month treatment period. Participants will be classified as good, partial, or non-responders based on the percentage reduction in transfusion volume, using previously published thresholds (>50%, 25–50%, <25%) |
[ Before treatment initiation, 6 months after starting treatment. ] |
|
2.
Quality of life will be assessed using the TranQoL (Transfusion-dependent Quality of Life Questionnaire), a validated disease-specific tool for transfusion-dependent thalassemia patients. It will be administered at baseline and after 6 months of therapy. The validated Urdu version will be used for participants who prefer it. |
[ baseline and at 6 months ] |
Target number/sample size
67
Countries of recruitment
Pakistan
Anticipated start date
2025-10-30
Anticipated end date
2025-06-30
Date of first enrollment
Date of study completion
Recruitment status
Pending
Funding source
None
Regulatory approvals
Status
Approved
Date of Approval
2025-05-07
Approval number
ERC/2025/PAEDS/14
Details of Ethics Review Committee
| Name: | Ethical committee PNS Shifa |
| Institutional Address: | Main Korangi Rd? near Kala Pul, Clifton, Karachi |
| Telephone: | (021) 48506500, +922148506777 |
| Email: | admin@pnsshifa.gov.pk |
Contact person for Scientific Queries/Principal Investigator
Inshal Jawed
General Physician
Dow University of Health Sciences, Mission Rd, New Labour Colony Nanakwara, Karachi, Karachi City, Sindh, Pakistan
+92 341 2636484
inshaljwd@gmail.com
Contact Person for Public Queries
Inshal Jawed
General Physician
Dow University of Health Sciences, Mission Rd, New Labour Colony Nanakwara, Karachi, Karachi City, Sindh, Pakistan
+92 341 2636484
inshaljwd@gmail.com
Do the investigators plan to share identified individual clinical trial participant-level data (IPD)?
No
IPD sharing plan description
Study protocol available
No
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
