Clinical trial NCT00586209: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Sickle Cell Anemia
Thalassemia
Keywords: sickle cell disease
sickle cell anemia
L-glutamine
Sickle Cell Anemia (homozygous)
Sickle β°-Thalassemia
Lead sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Sponsor class: OTHER
Collaborators: name: Emmaus Medical, Inc.
First posted: 2008-01-04
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Effect of L-glutamine Therapy on Exercise Endurance of Sickle Cell Anemia Patients
timeFrame: Baseline, Weeks 8 and 12
measure: Effect of L-glutamine Therapy on Breath by Breath Exercise of Sickle Cell Patients
timeFrame: Baseline, Weeks 18 and 12
Secondary outcomes:
measure: Effect of Oral L-glutamine on Incidence of Painful Crises
timeFrame: From Week 0 through Week 12
measure: Effect of Oral L-glutamine on Amount of Daily Requirement for Narcotics
timeFrame: From Week 0 through Week 20
measure: Effect of Oral L-glutamine on Level of Chronic Pain
timeFrame: From Week 0 to Week 12
Other outcomes:
Locations:
city: Torrance
country: United States
facility: LA Biomed at Harbor-UCLA Medical Center
state: California
Study officials:
affiliation: LaBiomed At Harbor-UCLA Medical Center
name: Yutaka Niihara, MD
role: PRINCIPAL_INVESTIGATOR
References: