L-Glutamine Therapy for Sickle Cell Anemia

A Phase II, Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Study of L Glutamine Therapy for Sickle Cell Anemia and Sickle ß0-Thalassemia

The 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 incidence of painful crises; level of chronic pain, and amount of daily requirement for narcotics.

Study Overview

• Status: Terminated
• Conditions: Sickle Cell Anemia; Thalassemia
• Intervention / Treatment: Drug: L-Glutamine; Drug: Placebo

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 β°-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 β°-thalassemia.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Torrance, California, United States, 90502
      • LA BioMed at Harbor-UCLA Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: L-glutamine; 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)
Placebo Comparator: Placebo; Maltodextrin 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effect of L-glutamine Therapy on Exercise Endurance of Sickle Cell Anemia Patients	Exercise endurance will be examined at each visit. Change from baseline will be reported at weeks 8 and 12	Baseline, Weeks 8 and 12
Effect of L-glutamine Therapy on Breath by Breath Exercise of Sickle Cell Patients	Breath by breath exercise will be examined at each visit. Change from baseline will be reported at weeks 8 and 12	Baseline, Weeks 18 and 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effect of Oral L-glutamine on Incidence of Painful Crises	Incidence of panful crises will be assessed at each visit.	From Week 0 through Week 12
Effect of Oral L-glutamine on Amount of Daily Requirement for Narcotics	Amount of daily requirement for narcotics will assessed at each visit.	From Week 0 through Week 20
Effect of Oral L-glutamine on Level of Chronic Pain	Level of chronic pain will be assessed at each visit.	From Week 0 to Week 12

Collaborators and Investigators

• Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
• Collaborators: Emmaus Medical, Inc.
• Investigators: Principal Investigator: Yutaka Niihara, MD, LABIOMED at Harbor-UCLA Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): February 6, 2004
• Primary Completion (Actual): August 27, 2008
• Study Completion (Actual): November 1, 2009

Study Registration Dates

• First Submitted: December 21, 2007
• First Submitted That Met QC Criteria: January 3, 2008
• First Posted (Estimate): January 4, 2008

Study Record Updates

• Last Update Posted (Actual): February 2, 2021
• Last Update Submitted That Met QC Criteria: February 1, 2021
• Last Verified: February 1, 2021

More Information

Terms related to this study

• Keywords: sickle cell disease; L-glutamine; sickle cell anemia; Sickle Cell Anemia (homozygous); Sickle β°-Thalassemia
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia; Anemia, Sickle Cell; Thalassemia
• Other Study ID Numbers: 10511-01RY