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

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

Study Overview

• Status: Recruiting
• Conditions: Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
• Intervention / Treatment: Drug: L_carnitine; Drug: Sildenafil

• Study Type: Interventional
• Enrollment (Anticipated): 44
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Mostafa Zaki Zedan, Bsc of pharmaceutical science; Phone Number: +0201554676330; Email: mostaf150776@pharm.tanta.edu.eg

Study Locations

• Egypt
  • Gharbia
    • Tanta, Gharbia, Egypt, 31511
      • Recruiting
      • Tanta University Hospital
      • Contact: Mohamed Ramadan El_shanshory, Professor

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 16 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Group 1; n=22): Patients will receive traditional treatment and L- carnitine 50mg/kg/day orally (maximum dose 3g per day)	Drug: L_carnitine; l-carnitine stabilizes red blood cell membranes and thus improves the anemic state[
Active Comparator: Group 2; 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; 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.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Tanta University
• Investigators: Principal Investigator: Sahar Kamal Hegazy, Professor, Tanta University

Study record dates

Study Major Dates

• Study Start (Actual): July 8, 2022
• Primary Completion (Anticipated): July 8, 2023
• Study Completion (Anticipated): July 8, 2024

Study Registration Dates

• First Submitted: October 13, 2022
• First Submitted That Met QC Criteria: October 14, 2022
• First Posted (Actual): October 18, 2022

Study Record Updates

• Last Update Posted (Actual): October 18, 2022
• Last Update Submitted That Met QC Criteria: October 14, 2022
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia; Molecular Mechanisms of Pharmacological Action; Vasodilator Agents; Urological Agents; Enzyme Inhibitors; Phosphodiesterase Inhibitors; Phosphodiesterase 5 Inhibitors; Sildenafil Citrate
• Other Study ID Numbers: Beta thalassemia

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No