Comparing Hydroxyurea and Metformin for Beta-Thalassemia Treatment
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
This study is testing whether hydroxyurea or metformin works better for treating people with beta-thalassemia who need regular blood transfusions.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 96 (estimated) |
| Ages | 10 Years to 25 Years |
| Sex | All |
| Sponsor | Bahria University Academic / other |
| Locations | 1 site (Karachi, Sindh) |
| Trial ID | NCT06606886 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the effects and safety of hydroxyurea versus metformin in patients with transfusion-dependent beta-thalassemia major. Participants will be randomly assigned to receive either metformin or hydroxyurea for one year, with their responses correlated to specific genetic mutations. The study aims to determine if there is a significant difference in treatment outcomes between the two medications. Monthly assessments will monitor participants' health and adherence to dietary recommendations.
Who should consider this trial
Good fit: Ideal candidates for this study are males and females aged 10 to 25 years diagnosed with beta-thalassemia major who are transfusion-dependent.
Not a fit: Patients with conditions such as severe osteoporosis, liver fibrosis, or those with HIV or hepatitis B & C may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide new treatment options for patients with beta-thalassemia, potentially improving their quality of life and reducing dependence on blood transfusions.
How similar studies have performed: While there have been studies on hydroxyurea for beta-thalassemia, the combination with metformin is a novel approach that has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Karachi, Sindh
- Bahria University Health Sciences Campus — Karachi, Sindh, Pakistan (Recruiting)
Study contacts
- Principal investigator: Ayesha Khan, MBBS MPhil — Bahria University Islamabad
- Study coordinator: Ayesha Khan
- Email: dr.ayeshakhan85@gmail.com
- Phone: +923363258465
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.