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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

70 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-03-31

Study Completion Date

2008-07-31

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Sickle Cell Anemia Thalassemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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investigational product

L-glutamine

Group Type EXPERIMENTAL

L-glutamine

Intervention Type DRUG

Approximately 0.3 g/kg total daily dose of L-glutamine will be orally administered (over two doses), with a maximum total daily dose of 30 grams.

placebo

maltodextrin

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Approximately 0.3 g/kg total daily dose of maltodextrin placebo will be orally administered (over two doses), with a maximum total daily dose of 30 grams.

Interventions

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L-glutamine

Approximately 0.3 g/kg total daily dose of L-glutamine will be orally administered (over two doses), with a maximum total daily dose of 30 grams.

Intervention Type DRUG

Placebo

Approximately 0.3 g/kg total daily dose of maltodextrin placebo will be orally administered (over two doses), with a maximum total daily dose of 30 grams.

Intervention Type DRUG

Other Intervention Names

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oral L-glutamine maltodextrin

Eligibility Criteria

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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.

Minimum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No