A Phase II, Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Study of L Glutamine Therapy for Sickle Cell Anemia and Sickle ß0-Thalassemia
Trial Snapshot
- Phase
- Phase 2
- Status
- Terminated
- Enrollment
- 15
- Locations
- 1
- Primary Endpoint
- Effect of L-glutamine Therapy on Exercise Endurance of Sickle Cell Anemia Patients
Study Overview
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 Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
- •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.
Arms & Interventions
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: L-Glutamine (Drug)
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: Placebo (Drug)
Outcomes
Primary Outcomes
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
Secondary Outcomes
- 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)
Investigators
yutaka niihara
President, Emmaus Medical
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center