L-Glutamine Therapy for Sickle Cell Anemia

NCT ID: NCT00586209

Last Updated: 2021-02-02

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2004-02-06
• Study Completion Date: 2009-11-30

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.

Conditions

Sickle Cell Anemia; Thalassemia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

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

Group Type: EXPERIMENTAL

L-Glutamine

Intervention Type: 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)

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

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

Interventions

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)

Intervention Type: DRUG

Placebo

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

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Emmaus Medical, Inc.

INDUSTRY

Sponsor Role: collaborator

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

OTHER

Sponsor Role: lead

Responsible Party

yutaka niihara

President, Emmaus Medical

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Yutaka Niihara, MD

Role: PRINCIPAL_INVESTIGATOR

LaBiomed At Harbor-UCLA Medical Center

Locations

LA Biomed at Harbor-UCLA Medical Center

Torrance, California, United States

Countries

United States

Other Identifiers

10511-01RY

Identifier Type: -

Identifier Source: org_study_id