Investigating thalidomide's effects on beta thalassemia
Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
This study is testing whether low doses of thalidomide can help young people with beta thalassemia manage their condition better and reduce the need for blood transfusions.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 54 (estimated) |
| Ages | 8 Years to 35 Years |
| Sex | All |
| Sponsor | National Institute of Blood and Marrow Transplant (NIBMT), Pakistan Government |
| Locations | 1 site (Karachi, Sindh) |
| Trial ID | NCT06490627 on ClinicalTrials.gov |
What this trial studies
This study examines the safety and efficacy of low-dose thalidomide in managing transfusion-dependent beta thalassemia, a genetic blood disorder. Conducted over two years at the National Institute of Blood Disease and Bone Marrow Transplant in Karachi, it involves 54 patients aged 8-35. The primary goal is to correlate different doses of thalidomide with disease severity, treatment response, and adverse effects. Data will be collected through clinical interviews and medical record reviews, focusing on key health indicators such as hemoglobin levels and transfusion frequency.
Who should consider this trial
Good fit: Ideal candidates are transfusion-dependent patients diagnosed with beta thalassemia major or intermediate who are willing to provide informed consent.
Not a fit: Patients with liver dysfunction, married individuals, lactating mothers, or those with a history of thrombosis or seizures may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option that improves management of beta thalassemia and reduces the need for blood transfusions.
How similar studies have performed: While thalidomide has been used in various contexts, this specific approach in beta thalassemia is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Know case of beta thalassemia major/ intermediate ( transfusion dependent) * willing to give informed consent Exclusion Criteria: * Patients with comorbidities such as liver dysfunction * Married patients * Lactating mother * H/O thrombosis and fits
Where this trial is running
Karachi, Sindh
- National Institute of blood disease and bone marrow transplant — Karachi, Sindh, Pakistan (Recruiting)
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.