NCT04684381

Pharmacokinetics and Safety of Endari (L-glutamine) in Sickle Cell Disease Patients

A Phase 4, Open-Label, Single-Center Study to Assess Pharmacokinetic Characteristics and Safety of Endari in Patients With Sickle Cell Disease

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Emmaus Medical, Inc. · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Accepted

Summary

L-glutamine has been approved in the US to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients 5 years of age and older. The purpose of this single-center, open-label, phase 4 study is to evaluate the pharmacokinetic characteristics and safety of L-glutamine in patients with SCD.

Detailed description

Sickle cell disease (SCD) is associated with a mutation in the β-hemoglobin gene that results in abnormal polymerization of hemoglobin. Polymerization of hemoglobin causes the red blood cell to sickle, leading to a cascade of events which cause acute complications for SCD patients. L-glutamine has been approved in the US to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients 5 years of age and older. The purpose of this single-center, open-label, phase 4 study is to evaluate the pharmacokinetic characteristics and safety of L-glutamine in patients with SCD. 8 SCD patients and 4 healthy volunteers will receive weight-based dosing of L-glutamine for 3 weeks. Doses will be changed weekly: 0.1 g/kg administered twice daily during week 1, 0.3 g/kg administered twice daily during week 2, and 0.6 g/kg administered once daily during week 3. The primary objective is to evaluate the pharmacokinetic characteristics of L-glutamine in SCD patients compared with healthy volunteers.

Conditions

• Sickle Cell Disease
• Pharmacokinetics

Interventions

Type	Name	Description
DRUG	L-glutamine	Pharmacokinetic study

Timeline

• Start date: 2021-01-04
• Primary completion: 2021-06-30
• Completion: 2021-06-30
• First posted: 2020-12-24
• Last updated: 2022-08-02

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study