L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia - Full Text View

Sponsor:	Emmaus Medical, Inc.
Collaborator:	FDA Office of Orphan Products Development
Information provided by (Responsible Party):	Emmaus Medical, Inc.
ClinicalTrials.gov Identifier:	NCT00125788

Purpose

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 on pain; energy and appetite levels; narcotics usage; height and weight; and hospital and emergency room visits for sickle cell pain.

Condition	Intervention	Phase
Sickle Cell Anemia Thalassemia	Drug: L-glutamine Drug: maltodextrin	Phase II

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

Resource links provided by NLM:

Genetics Home Reference related topics: sickle cell disease

MedlinePlus related topics: Anemia Sickle Cell Anemia Thalassemia

Drug Information available for: Glutamine

U.S. FDA Resources

Further study details as provided by Emmaus Medical, Inc.:

Primary Outcome Measures:

Number of occurrences of painful sickle cell crises [ Time Frame: from Week 0 through Week 48 (cumulative) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Frequency of emergency room visits for sickle cell pain [ Time Frame: from Week 0 through Week 24 (cumulative), and from Week 0 through Week 48 (cumulative) ] [ Designated as safety issue: No ]
Number of days patients' usual daily activities are interrupted due to sickle cell pain [ Time Frame: from Week 0 through Week 48 (cumulative) ] [ Designated as safety issue: No ]
Growth curve for patients less than 18 years of age [ Time Frame: at each study visit (approximately every four weeks) ] [ Designated as safety issue: No ]
At each study visit, the patient's height and weight will be compared to the standard growth curve. The patient will be classified as below the 5th, 10th, 25th, 50th, 75th, 90th and 95th percentile for height and weight (mutually exclusive groups).
Hematological parameters [ Time Frame: collected at Weeks 0, 4, 8, 12, 16, 20, 24, 32, 40, 48, 53 ] [ Designated as safety issue: No ]
Narcotic usage [ Time Frame: ongoing from Week 0 through Week 48 ] [ Designated as safety issue: No ]
Alcohol and tobacco use [ Time Frame: ongoing from Week 0 through Week 48 ] [ Designated as safety issue: No ]
Pain level [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
Energy level [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
Patient activity level [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
Patient appetite [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
Subjective exercise tolerance [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
Subjective quality of life [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
L-glutamine safety assessment based on adverse events, laboratory parameters, and vital signs [ Time Frame: ongoing from Week 0 through Week 53 ] [ Designated as safety issue: Yes ]
Number of occurrences of sickle cell crises [ Time Frame: from Week 0 through Week 24 (cumulative) ] [ Designated as safety issue: No ]

Enrollment:	81
Study Start Date:	March 2004
Study Completion Date:	July 2008
Primary Completion Date:	July 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: investigational product L-glutamine	Drug: 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.
Placebo Comparator: placebo maltodextrin	Drug: maltodextrin 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.

Eligibility

Ages Eligible for Study:	5 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 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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00125788

Locations

United States, California
Kaiser Permanente
Bellflower, California, United States, 90706
Harbor-UCLA Medical Center
Torrance, California, United States, 90502
United States, Georgia
Grady Memorial Hospital
Atlanta, Georgia, United States, 30303
United States, New Jersey
University of Medicine and Dentistry, New Jersey
New Brunswick, New Jersey, United States, 08903
United States, New York
Jacobi Medical Center
Bronx, New York, United States, 10461

Sponsors and Collaborators

Emmaus Medical, Inc.

FDA Office of Orphan Products Development

Investigators

Principal Investigator:

Yutaka Niihara, MD

CEO, Emmaus Medical, Inc

More Information

No publications provided

Responsible Party:	Emmaus Medical, Inc.
ClinicalTrials.gov Identifier:	NCT00125788 History of Changes
Obsolete Identifiers:	NCT00029887
Other Study ID Numbers:	10478
Study First Received:	August 1, 2005
Last Updated:	November 17, 2011
Health Authority:	United States: Food and Drug Administration

Keywords provided by Emmaus Medical, Inc.:

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

Additional relevant MeSH terms:

Anemia
Anemia, Sickle Cell
Thalassemia
Hematologic Diseases

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

ClinicalTrials.gov processed this record on February 09, 2012