L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for
 sickle cell anemia and sickle ß0-thalassemia. as evaluated by the number of occurrences of
 sickle cell crises.
The primary purpose of this study is to evaluate the effectiveness of oral L-glutamine in the
 therapy of sickle cell anemia and sickle ß0-thalassemia.

 The secondary purpose is to assess the effect of L-glutamine frequency of hospitalizations
 for sickle cell pain, frequency of emergency room visits for sickle cell pain; energy and
 appetite levels; narcotics usage.

 Methodology:

 By site, patients will be randomized to L-glutamine or placebo in a 1:1 ratio after a 4-week
 screening period. Patients will undergo 48 weeks of treatment with dosing BID orally, with
 dose calculated according to patient weight. Patient visits will occur every 4 weeks. After
 48 weeks of treatment, dose will be tapered to zero within 3 weeks. A final evaluation visit
 will occur 2 weeks after last dose.
Inclusion Criteria:

 To be eligible to participate in the study, a patient must meet all of the following
 inclusion criteria:

 -  Patient is at least five years of age.

 -  Patient has been diagnosed with sickle cell anemia or sickle ß0-thalassemia
 (documented by hemoglobin electrophoresis).

 -  Patient has had at least two episodes of painful crises within 12 months of the
 screening visit.

 -  If the patient has been treated with an anti-sickling agent within three months of the
 screening visit, the therapy must have been continuous for at least three months with
 the intent to continue for the next 14 months.

 -  Patient or the patient's legally authorized representative has given written informed
 consent.

 -  If the patient is a female of child-bearing potential, she agrees to practice a
 recognized form of birth control during the course of the study.

 Exclusion Criteria:

 If the patient meets any of the following criteria, the patient must not be enrolled:

 -  Patient has a significant medical condition that required hospitalization (other than
 sickle painful crisis) within two months of the screening visit.

 -  Patient has diabetes mellitus with untreated fasting blood sugar >115 mg/dL.

 -  Patient has prothrombin time International Normalized Ratio (INR) > 2.0.

 -  Patient has serum albumin < 3.0 g/dl.

 -  Patient has received any blood products within three weeks of the screening visit.

 -  Patient has a history of uncontrolled liver disease or renal insufficiency.

 -  Patient is pregnant or lactating.

 -  Patient has been treated with an experimental anti-sickling medication/treatment
 (except hydroxyurea) within 30 days of the screening visit.

 -  Patient has been treated with an experimental drug within 30 days of the screening
 visit.

 -  There are factors that would, in the judgment of the investigator, make it difficult
 for the patient to comply with the requirements of the study.