CompletedPhase 4

Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia

Pakistan96 participantsStarted 2024-09-16

Plain-language summary

This clinical trial aims to evaluate and compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism mutations (SNPs) by DNA analysis. The main question to answer is: * Is there a significant difference in the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major? Participants will be divided into two treatment groups. Participants of group A will receive Tab. Metformin 500mg/dose twice daily orally for one year. Participants of group B will receive Tab. Hydroxyurea 10-20mg/kg once daily orally for one year. At the end of the study, Investigators will compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism mutations (SNPs) by DNA analysis.

Who can participate

Age range

10 Years – 25 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Diagnosed cases of beta-thalassemia major * Transfusion dependent patients and on iron chelating agents * Males and females 10 to 25 years of age * Creatinine ≤1.4 mg/dL * Written informed consent from the patients and up to 18 years consent from the Parents/Guardians. Exclusion Criteria: * Failure to meet inclusion criteria * Refusal to participate * Creatinine \>1.4mg/dL * Liver function tests (ALT and conjugated bilirubin) \> 4 times upper limits of normal * Massive splenomegaly \& Liver fibrosis * Patient on growth hormone * Sickle beta thalassemia * Severe osteoporosis * Hepatitis B \& C * HIV

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change in Fetal Haemoglobin (HbF) Percentage in responders and non-responders

Timeframe: 6 months to 1 year after the baseline

Change in frequency of blood transfusions

Timeframe: 6 months to 1 year after the baseline

Trial details

NCT IDNCT06606886

SponsorBahria University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-02-09

View on ClinicalTrials.gov More Transfusion Dependent Beta-thalassemia trials