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

Tanta University

Status and phase: Enrolling; Phase 3

Conditions: Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity

Treatments: Drug: L_carnitine; Drug: Sildenafil

Study type: Interventional

Funder types: Other

Identifiers

NCT05584956

Beta thalassemia

Details and patient eligibility

About

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

Enrollment: 44 estimated patients

Sex: All

Ages: 6 to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

44 participants in 2 patient groups

Group 1

Active Comparator group

Description:

n=22): Patients will receive traditional treatment and L- carnitine 50mg/kg/day orally (maximum dose 3g per day)

Treatment:

Drug: L_carnitine

Group 2

Active Comparator group

Description:

n=22): Patients will receive traditional treatment and Sildenafil 0.25mg/kg/dose every 6 h orally (maximum dose 60 mg per day)

Treatment:

Drug: Sildenafil