L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia

Emmaus Medical

Status and phase

Completed

Phase 2

Conditions

Thalassemia

Sickle Cell Anemia

Treatments

Drug: Placebo

Drug: L-glutamine

Study type

Interventional

Funder types

Industry

Other U.S. Federal agency

Identifiers

NCT00125788

10478

Details and patient eligibility

About

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.

Full description

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.

Enrollment

70 patients

Sex

All

Ages

5+ years old

Volunteers

No Healthy Volunteers

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.