Sickle Cell Disease
Conditions
Keywords
glutamine, vasooclussive crisis, sickle cell disease, Egypt
Brief summary
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
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
Interventions
DRUGL-Glutamine, Oral Powder for Reconstitution
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
OTHERStandard of care
Hydroxyurea 15-25 mg per kg per day and/ or blood transfusion therapy
Sponsors
Ain Shams University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
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. \-
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of pain crises | 24 weeks | The number of pain crises will be counted from day 1 till end of treatment at week 24 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in transcranial doppler | 24 weeks | 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 |
Countries
Egypt
Outcome results
None listed