Clinical Study to Evaluate the Possible Efficacy and Safety of L- Carnitine and Sildenafil in Children Having Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity

Study Overview
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Brief Summary
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This study aims to investigate the possible efficacy and safety of L_Carnitine and Sildenafil on patient with Beta thalassemia complicated with increased Tricuspid Regurgitant Jet Velocity

Official Title
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Clinical Study to Evaluate the Possible Efficacy and Safety of L- Carnitine and Sildenafil in Children Having Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity

Conditions
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Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity

Intervention / Treatment
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* Drug: L_carnitine
* Drug: Sildenafil


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Children with Beta thalassemia major who have increased TRJV more than 2.5m/s. Children age from 6-18 years Exclusion Criteria: Others hemolytic anemia Young age before 6 years of age Allergy to Sildenafil or L-carnitine Patient with documented causes of pulmonary hypertension rather than caused by Beta thalassemia. Hepatic dysfunction: serum Alanine Aminotransferase (ALT) 3X. Renal dysfunction: Creatinine level greater than or equal to 1.2 mg/dl.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 18 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: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Group 1<br>n=22): Patients will receive traditional treatment and L- carnitine 50mg/kg/day orally (maximum dose 3g per day) | Drug: L_carnitine<br>* l-carnitine stabilizes red blood cell membranes and thus improves the anemic state[<br>|
| Active Comparator: Group 2<br>n=22): Patients will receive traditional treatment and Sildenafil 0.25mg/kg/dose every 6 h orally (maximum dose 60 mg per day) | Drug: Sildenafil<br>* selective and potent inhibitor of cGMP-specific phosphodiesterase 5 (PDE5), which promotes smooth muscle relaxation in lung vasculature, has been used successfully in the treatment of primary and secondary PH.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cardiopulmonary functions | change in the studied cardiopulmonary functions at the baseline and 3 months after intervention | 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Biochemical parameters | Changes in Vascular Endothelial Growth Factor level (VEGF) at baseline and 3 months after the intervention | 3 months | 
| Biochemical parameters | Changes in Nitric oxide level (NO) at the baseline and 3 months after the intervention | 3 months | 
| Biochemical parameters | Changes in ferritin serum level at the baseline and 3 months after the intervention | 3 months |