L-Glutamine Therapy for Sickle Cell Anemia - Full Text View

Sponsors and Collaborators:	Los Angeles Biomedical Research Institute Emmaus Medical, Inc.
Information provided by:	Los Angeles Biomedical Research Institute
ClinicalTrials.gov Identifier:	NCT00586209

Purpose

To primary purpose is to evaluate the effect of L-glutamine therapy on exercise endurance and breath by breath exercise response of sickle cell anemia patients

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

Condition	Intervention	Phase
Sickle Cell Anemia Thalassemia	Drug: L-Glutamine Drug: Placebo	Phase II

Genetics Home Reference related topics: beta thalassemia sickle cell disease

MedlinePlus related topics: Anemia Exercise and Physical Fitness Sickle Cell Anemia Thalassemia

Drug Information available for: Starch Glutamine

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Investigator, Outcomes Assessor), Parallel Assignment, Safety/Efficacy Study
Official Title:	A Phase II, Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Study of L Glutamine Therapy for Sickle Cell Anemia and Sickle ß0-Thalassemia

Further study details as provided by Los Angeles Biomedical Research Institute:

Primary Outcome Measures:

Number of occurrences of painful sickle cell crises [ Time Frame: 4 (28 days) Follow-up after randomization ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Frequency of hospitalizations for sickle cell pain [ Time Frame: 4 (28 days) Follow-up after randomization ] [ Designated as safety issue: Yes ]
Frequency of emergency room visits for sickle cell pain [ Time Frame: 4 (28 days) Follow-up after randomization ] [ Designated as safety issue: Yes ]
Number of days patients' usual daily activities are interrupted due to sickle cell pain [ Time Frame: 4 (28 days) Follow-up after randomization ] [ Designated as safety issue: Yes ]
Subjective exercise tolerance [ Time Frame: 4 (28 days) Follow-up after randomization ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	50
Study Start Date:	January 2003
Estimated Study Completion Date:	January 2009
Estimated Primary Completion Date:	November 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Active Comparator L-glutamine group will be given at the following dosage: 17-33.3 kg at 5 g 2x daily 33.4-66.6 kg at 10 g 2X daily >66.7 at 15 g 2X daily	Drug: L-Glutamine L-Glutamine at: 5 g 2X daily (17-33.3 Kg) 10 g 2x daily (33.4-66.6 Kg) 15 g 2x daily (>66.7 kg)
2: Placebo Comparator Placebo group will be given at the following dosage: 17-33.3 kg at 5 g 2x daily 33.4-66.6 kg at 10 g 2X daily >66.7 at 15 g 2X daily	Drug: Placebo Placebo (75% Maltodextrin, 24.5 % Starch, 0.5% Tricalcium Phosphate) - given at the same dosage as L-glutamine

Detailed Description:

This is a phase II, prospective, randomized, double-blind, placebo-controlled, parallel-group,study to evaluate the safety and efficacy of oral L-glutamine therapy for patients with sickle cell anemia or sickle IS°-thalassemia who are at least 18 years old with focus on the aspect of exercise endurance. In this study, the patients will orally take L-glutamine or placebo twice daily, and clinical and hematological parameters will be monitored. The adverse events,especially those attributable to L-glutamine therapy, will also be monitored. The data obtained will be essential in understanding the role of L-glutamine in therapy for sickle cell anemia and sickle B°-thalassemia.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

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

Patient is at least 18 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.
Patient is able to perform exercise tolerance test

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00586209

Locations

United States, California
LA BIomed At Harbor UCLA Medical Center
Torrance, California, United States, 90502
LA Biomed at Harbor-UCLA Medical Center
Torrance, California, United States, 90502

Sponsors and Collaborators

Los Angeles Biomedical Research Institute

Emmaus Medical, Inc.

Investigators

Principal Investigator:

Yutaka Niihara, MD

LaBiomed At Harbor-UCLA Medical Center

More Information

Responsible Party:	LA Biomed at Harbor-UCLA Medical Center ( Yutaka Niihara, MD )
Study ID Numbers:	10511-01RY
Study First Received:	December 21, 2007
Last Updated:	October 22, 2008
ClinicalTrials.gov Identifier:	NCT00586209
Health Authority:	United States: Food and Drug Administration

Keywords provided by Los Angeles Biomedical Research Institute:

sickle cell disease
sickle cell anemia
L-glutamine
Sickle Cell Anemia (homozygous and heterozygous)
Sickle ß0-Thalassemia

Study placed in the following topic categories:

Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
Anemia

Anemia, Hemolytic
Hemoglobinopathy
Thalassemia
Anemia, Sickle Cell
Sickle cell anemia

ClinicalTrials.gov processed this record on January 16, 2009