Back to resultsClinical Study to Evaluate the Possible Efficacy and Safety of L- Carnitine and Sildenafil in Children Having Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
Primary Purpose
Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
Status
Recruiting
Phase
Phase 3
Locations
Egypt
Study Type
Interventional
Intervention
L_carnitine
Sildenafil
Sponsored by
About this trial
This is an interventional treatment trial for Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
Eligibility Criteria
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.
Sites / Locations
- Tanta University HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Active Comparator
Arm Label
Group 1
Group 2
Arm Description
n=22): Patients will receive traditional treatment and L- carnitine 50mg/kg/day orally (maximum dose 3g per day)
n=22): Patients will receive traditional treatment and Sildenafil 0.25mg/kg/dose every 6 h orally (maximum dose 60 mg per day)
Outcomes
Primary Outcome Measures
Cardiopulmonary functions
change in the studied cardiopulmonary functions at the baseline and 3 months after intervention
Secondary Outcome Measures
Biochemical parameters
Changes in Vascular Endothelial Growth Factor level (VEGF) at baseline and 3 months after the intervention
Biochemical parameters
Changes in Nitric oxide level (NO) at the baseline and 3 months after the intervention
Biochemical parameters
Changes in ferritin serum level at the baseline and 3 months after the intervention
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05584956
Brief 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
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Recruiting
Study Start Date
July 8, 2022 (Actual)
Primary Completion Date
July 8, 2023 (Anticipated)
Study Completion Date
July 8, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Tanta University
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. 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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
44 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Group 1
Arm Type
Active Comparator
Arm Description
n=22): Patients will receive traditional treatment and L- carnitine 50mg/kg/day orally (maximum dose 3g per day)
Arm Title
Group 2
Arm Type
Active Comparator
Arm Description
n=22): Patients will receive traditional treatment and Sildenafil 0.25mg/kg/dose every 6 h orally (maximum dose 60 mg per day)
Intervention Type
Drug
Intervention Name(s)
L_carnitine
Intervention Description
l-carnitine stabilizes red blood cell membranes and thus improves the anemic state[
Intervention Type
Drug
Intervention Name(s)
Sildenafil
Intervention Description
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.
Primary Outcome Measure Information:
Title
Cardiopulmonary functions
Description
change in the studied cardiopulmonary functions at the baseline and 3 months after intervention
Time Frame
3 months
Secondary Outcome Measure Information:
Title
Biochemical parameters
Description
Changes in Vascular Endothelial Growth Factor level (VEGF) at baseline and 3 months after the intervention
Time Frame
3 months
Title
Biochemical parameters
Description
Changes in Nitric oxide level (NO) at the baseline and 3 months after the intervention
Time Frame
3 months
Title
Biochemical parameters
Description
Changes in ferritin serum level at the baseline and 3 months after the intervention
Time Frame
3 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Mostafa Zaki Zedan, Bsc of pharmaceutical science
Phone
+0201554676330
Email
mostaf150776@pharm.tanta.edu.eg
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sahar Kamal Hegazy, Professor
Organizational Affiliation
Tanta University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Tanta University Hospital
City
Tanta
State/Province
Gharbia
ZIP/Postal Code
31511
Country
Egypt
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mohamed Ramadan El_shanshory, Professor
12. IPD Sharing Statement
Learn more about this trial
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
We'll reach out to this number within 24 hrs