Glutamine Role in Preventing Vaso-occlusive Crisis Among SCD Patients

Study Overview
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Brief Summary
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Prospective phase IV interventional open label randomized controlled trial to assess safety and efficacy of glutamine in preventing vaso-occlusive crisis (VOC) episodes in sickle cell pediatrics and adolescents' patients

Detailed Description
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Vaso-occlusive crisis (VOC) episodes are considered to be the cause of 95% of hospitalizations for sickle cell disease (SCD) patients. Prior studies have described pain management in SCD patients with poor outcomes in the short term and decreased quality of life in patients over the long term. Although that L-glutamine has been recently approved by the FDA for the prevention of acute complications in sickle cell disease. However, there are many gaps in our understanding of its therapeutic implications in SCD. This study will assess the safety and efficacy of glutamine in preventing vaso-occlusive crisis (VOC) episodes in sickle cell pediatrics and adolescents' patients

Official Title
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Safety and Efficacy of Glutamine in Preventing Vaso-occlusive Crisis Among Sickle Cell Disease Patients: Randomized Controlled Study

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Drug: L-Glutamine, Oral Powder for Reconstitution
* Other: Standard of care


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Children and adolescents diagnosed with sickle cell disease by haemoglobin electrophoresis and had at least two pain crises (no upper limit) documented during the previous year; a pain crisis is defined as pain leading to treatment with a parenteral administered narcotic or ketolac in an emergency department (ED) (or outpatient treatment centre) or during hospitalization. Patients receiving hydroxyurea at a fixed dose for at least 3 months before screening. Exclusion Criteria: Patients with sickle cell trait and other hemoglobinopathy. -

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: cases<br>• 30 patients will receive glutamine in a dose of 0.3 gm /kg/dose twice daily orally (up to a maximum of 15 g/dose) for 24 weeks as an add on to the SOC | Drug: L-Glutamine, Oral Powder for Reconstitution<br>* Glutamine is an essential amino acid. It will be provided in a powder form. It will be dissolved in at least 8 ounces of hot or cold liquid. It can also be mixed with a soft food such as pudding, applesauce, or yogurt. Then it will be Stirred and then eaten or drunken The Glutamine will be as an add on to the Standard of care<br>* Other names: L-Glutamine Powder;|
| Active Comparator: control<br>30 patients will be assigned as a control group to receive standard of care therapy without glutamine intake. | Other: Standard of care<br>* Hydroxyurea 15-25 mg per kg per day and/ or blood transfusion therapy<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of pain crises | The number of pain crises will be counted from day 1 till end of treatment at week 24 | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in transcranial doppler | Calculate the change in transcranial doppler (TCD) time-averaged mean of the maximum velocity (TAMMV) arterial cerebral blood flow through the measuring of TCD flow velocity at day1 and at Week 24 | 24 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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glutamine, vasooclussive crisis, sickle cell disease, Egypt