L-Glutamine Therapy for Sickle Cell Anemia

NCT00586209 | Terminated Phase 2 Results DMC

• 1 other identifier: 10511-01RY
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Sickle Cell Anemia; Thalassemia

Keywords

sickle cell disease; sickle cell anemia; L-glutamine; Sickle Cell Anemia (homozygous); Sickle β°-Thalassemia

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (3)

• Effect of Oral L-glutamine on Incidence of Painful Crises

  From Week 0 through Week 12

• Effect of Oral L-glutamine on Amount of Daily Requirement for Narcotics

  From Week 0 through Week 20

• Effect of Oral L-glutamine on Level of Chronic Pain

  From Week 0 to Week 12

Study Arms (2)

L-glutamine

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

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

Interventions

L-Glutamine DRUG

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)

L-glutamine

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center: lead
• Emmaus Medical, Inc.: collaborator

Study Sites (1)

LA Biomed at Harbor-UCLA Medical Center

Torrance, California, 90502, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Thalassemia

Interventions

Glutamine

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Amino Acids, Basic; Amino Acids; Amino Acids, Peptides, and Proteins; Amino Acids, Diamino; Amino Acids, Neutral

Results Point of Contact

• Title: Yutaka Niihara, MD, MPH
• Organization: Emmaus Medical, Inc.

yniihara@emmausmedical.com

310-214-0065

Study Officials

• Yutaka Niihara, MD

  LaBiomed At Harbor-UCLA Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: President, Emmaus Medical

Study Record Dates

• First Submitted: December 21, 2007
• First Posted: January 4, 2008
• Study Start: February 6, 2004
• Primary Completion: August 27, 2008
• Study Completion: November 1, 2009
• Last Updated: February 2, 2021
• Results First Posted: February 2, 2021

Record last verified: 2021-02

Locations

US (1)