A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia
Condition: Sickle Cell Anemia; Sickle ß0-Thalassemia
MeSH terms: Anemia; Thalassemia; Anemia, Sickle Cell
Intervention: L-glutamine; Placebo
Intervention description: 0.3 g/kg of L-glutamine will be administered twice a day orally to each patient for 48 weeks. The dosage will be in increments of 5 grams based on weight. The upper limit for daily dose of study medication will be set at 30 grams. Patients will be given verbal and written instructions for self-administration of the study medication at the Baseline visit. The powder can be mixed with water or most non-heated beverages other than alcohol, or can be mixed with most non-heated foods such as yogurt, applesauce, or cereal for administration. Mixing L-glutamine with soda or highly acidic juices (such as grapefruit juice or lemonade) is not recommended.; 0.3 g/kg of placebo (100% maltodextrin) will be administered twice a day orally to each patient for 48 weeks. The dosage will be in increments of 5 grams based on weight. The upper limit for daily dose of study medication will be set at 30 grams. Patients will be given verbal and written instructions for self-administration of the study medication at the Baseline visit. The powder can be mixed with water or most non-heated beverages other than alcohol, or can be mixed with most non-heated foods such as yogurt, applesauce, or cereal for administration.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 230.0
Sex: All
Minimum age: 5 Years
Healthy volunteers: No
Keywords: Sickle Cell Anemia; Sickle Cell Pain Crises; Painful Crises; Sickle Cell Pain; Vaso-occlusive Crises

Summary:

      The purpose of this research is to evaluate the effects of L-glutamine as a therapy for
      Sickle Cell Anemia or Sickle ß0 Thalassemia as evaluated by the number of occurrences of
      sickle cell crises.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patient is at least five years of age.

          -  Patient has been diagnosed with sickle cell anemia or sickle ß°-thalassemia
             (documented by hemoglobin electrophoresis).

          -  Patient has had at least two documented episodes of sickle cell 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 duration of the study.

          -  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 avoid pregnancy
             during the study and is willing and agrees to practice a recognized form of birth
             control during the course of the study (e.g. barrier, birth control pills,
             abstinence).

        Exclusion Criteria:

          -  Patient has a significant medical condition that required hospitalization (other than
             sickle cell crisis) within two months of the screening visit.

          -  Patient has prothrombin time 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 uncontrolled liver disease or renal insufficiency.

          -  Patient is pregnant or lactating or has the intention of becoming pregnant during the
             study (if female and of child-bearing potential).

          -  Patient is currently taking or has been treated with any form of glutamine supplement
             within 30 days of the screening visit.

          -  Patient has been treated with an experimental anti-sickling medication/ treatment
             within 30 days of the screening visit (with the exception of hydroxyurea in pediatric
             patients).

          -  Patient is currently taking or has been treated with an investigational drug within 30
             days of the screening visit (with the exception of hydroxyurea in pediatric patients).

          -  Patient is currently enrolled in an investigational drug or device study and/or has
             participated in such a study 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.