Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major

Study Overview
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Brief Summary
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This study aims to investigate the use of amlodipine, a drug that blocks the uptake of calcium into cells, in the prevention and treatment of iron overload in patients with thalassemia major. Since iron uses the same calcium channels to enter the heart, pancreas and other organs, blocking these channels might help to prevent the accumulation of iron in these tissues. The study will follow 10 patients with thalassemia major: 5 will openly receive amlodipine and 5 will serve as controls, not receiving any additional drugs. Patients will be monitored through one year with an additional year of follow up after the group using amlodipine stops its use. Monitoring will occur through the measurement of blood ferritin as well as live and heart T2* by MRI.

Official Title
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Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major

Conditions
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Thalassemia Major

Intervention / Treatment
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* Drug: Amlodipine


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: patients with thalassemia major over 6 years of age in use of iron chelating therapy. Exclusion Criteria: pregnancy; advanced heart failure with NYHA III/IV or LVEF < 35%; formal contra-indications to an MRI study; patient with advanced heart block.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 90 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Crossover Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Amlodipine<br>amlodipine 5mg qd | Drug: Amlodipine<br>* 5mg po for 12 months<br>|
| Placebo Comparator: Controls<br>placebo | Drug: Amlodipine<br>* 5mg po for 12 months<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Myocardium T2* | Heart T2* by MRI at 12 months | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Liver T2* | Liver T2* by MRI every 6 months for 2 years. First year with active treatment; second year with withdrawal. | 24 months | 
| Heart volumes | Diastolic and systolic volumes by MRI every 6 months for 2 years. First year with active treatment; second year with withdrawal. | 24 months | 
| Left ventricle ejection fraction | LVEF by MRI every 6 months for 2 years. First year with active treatment; second year with withdrawal. | 24 months | 
| Serum ferritin | Serum ferritin every 6 months for 2 years. First year with active treatment; second year with withdrawal. | 24 months | 
| Myocardium T2* | Heart T2* by MRI every 6 months until 24 months | 6-18-24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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thalassemia major, calcium channel blockade, magnetic resonance imaging