L-Glutamine Therapy for Sickle Cell Anemia

• ClinicalTrials.gov Identifier: NCT00586209
• Recruitment Status: Terminated
• First Posted: January 4, 2008
• Results First Posted: February 2, 2021
• Last Update Posted: February 2, 2021
• Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
• Collaborator: Emmaus Medical, Inc.
• Information provided by (Responsible Party): yutaka niihara, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Tracking Information

• First Submitted Date: December 21, 2007
• First Posted Date: January 4, 2008
• Results First Submitted Date: September 21, 2020
• Results First Posted Date: February 2, 2021
• Last Update Posted Date: February 2, 2021
• Actual Study Start Date: February 6, 2004
• Actual Primary Completion Date: August 27, 2008 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 1, 2021)

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

• Original Primary Outcome Measures (submitted: January 3, 2008): Number of occurrences of painful sickle cell crises [ Time Frame: 4 (28 days) Follow-up after randomization ]

Current Secondary Outcome Measures (submitted: February 1, 2021)

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

Original Secondary Outcome Measures (submitted: January 3, 2008)

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

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: L-Glutamine Therapy for Sickle Cell Anemia
• 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

Brief Summary

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.

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
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Investigator, Outcomes Assessor); Primary Purpose: Treatment

Condition

• Sickle Cell Anemia
• Thalassemia

Intervention

• 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)
• Drug: Placebo
  Placebo (75% Maltodextrin, 24.5 % Starch, 0.5% Tricalcium Phosphate) - given at the same dosage as L-glutamine

Study Arms

• 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

  Intervention: Drug: L-Glutamine
• 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

  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Terminated
• Actual Enrollment (submitted: June 27, 2011): 15
• Original Estimated Enrollment (submitted: January 3, 2008): 50
• Actual Study Completion Date: November 2009
• Actual Primary Completion Date: August 27, 2008 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00586209
• Other Study ID Numbers: 10511-01RY
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: yutaka niihara, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
• Original Responsible Party: Yutaka Niihara, MD, LA Biomed at Harbor-UCLA Medical Center
• Current Study Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
• Original Study Sponsor: Same as current
• Collaborators: Emmaus Medical, Inc.

Investigators

• Principal Investigator: Yutaka Niihara, MD; LaBiomed At Harbor-UCLA Medical Center

• PRS Account: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
• Verification Date: February 2021