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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

Sponsor
Tanta University (Other)
Overall Status
Recruiting
CT.gov ID
NCT05584956
Collaborator
(none)
44
Enrollment
1
Location
2
Arms
24
Anticipated Duration (Months)
1.8
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
44 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
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
Actual Study Start Date :
Jul 8, 2022
Anticipated Primary Completion Date :
Jul 8, 2023
Anticipated Study Completion Date :
Jul 8, 2024

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures

  1. Cardiopulmonary functions [3 months]

    change in the studied cardiopulmonary functions at the baseline and 3 months after intervention

Secondary Outcome Measures

  1. Biochemical parameters [3 months]

    Changes in Vascular Endothelial Growth Factor level (VEGF) at baseline and 3 months after the intervention

  2. Biochemical parameters [3 months]

    Changes in Nitric oxide level (NO) at the baseline and 3 months after the intervention

  3. Biochemical parameters [3 months]

    Changes in ferritin serum level at the baseline and 3 months after the intervention

Eligibility Criteria

Criteria

Ages Eligible for Study:
6 Years to 18 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Tanta University Hospital Tanta Gharbia Egypt 31511

Sponsors and Collaborators

  • Tanta University

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Mostafa Zaki Zedan, Clinical pharmacist, Tanta University
ClinicalTrials.gov Identifier:
NCT05584956
Other Study ID Numbers:
  • Beta thalassemia
First Posted:
Oct 18, 2022
Last Update Posted:
Oct 18, 2022
Last Verified:
Oct 1, 2022
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Thalassemia
beta-Thalassemia
Sildenafil Citrate

Study Results

No Results Posted as of Oct 18, 2022