L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
- Registration Number
- NCT00125788
- Lead Sponsor
- Emmaus Medical, Inc.
- Brief Summary
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia. as evaluated by the number of occurrences of sickle cell crises.
- Detailed Description
The primary purpose of this study is to evaluate the effectiveness of oral L-glutamine in the therapy of sickle cell anemia and sickle ß0-thalassemia.
The secondary purpose is to assess the effect of L-glutamine frequency of hospitalizations for sickle cell pain, frequency of emergency room visits for sickle cell pain; energy and appetite levels; narcotics usage.
Methodology:
By site, patients will be randomized to L-glutamine or placebo in a 1:1 ratio after a 4-week screening period. Patients will undergo 48 weeks of treatment with dosing BID orally, with dose calculated according to patient weight. Patient visits will occur every 4 weeks. After 48 weeks of treatment, dose will be tapered to zero within 3 weeks. A final evaluation visit will occur 2 weeks after last dose.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 70
To be eligible to participate in the study, a patient must meet all of the following inclusion criteria:
- Patient is at least five 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.
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description placebo Placebo maltodextrin investigational product L-glutamine L-glutamine
- Primary Outcome Measures
Name Time Method Number of Occurrences of Painful Sickle Cell Crises From Week 0 through Week 48 (cumulative) The mean number of painful sickle crisis through week 48
- Secondary Outcome Measures
Name Time Method Frequency of Hospitalizations for Sickle Cell Pain From Week 0 through Week 48 (cumulative) The mean number of hospitalizations through week 48
Frequency of Emergency Room Visits for Sickle Cell Pain From Week 0 through Week 48 (cumulative) The mean number of emergency room visits through week 48
Number of Participants With Narcotic Usage Week 24, Week 48 Analysis of narcotic usage was performed for the subset of patients with any narcotic use who completed the study. Changes in narcotic usage were determined by an independent consultant prior to database lock using morphine equivalents to determine relative use.
The Effect of Oral L-glutamine on Vital Signs - Blood Pressure Baseline, Weeks 4, 24, and 48 Patient's blood pressure will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
Effect of Oral L--glutamine on Height Baseline, Weeks 4, 24, and 48 Height will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Walk Without Rest Baseline, Weeks 4, 24, and 48. Minutes patient could walk without rest will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Walk Without Rest Baseline, Weeks 4, 24, and 48. Distance patient could walk without rest will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
The Effect of Oral L-glutamine on Hematological Parameters - Hemoglobin Baseline, Weeks 4, 24 and 40 Patient's hemoglobin will be collected at each visit.Change from Baseline will be reported at Weeks 4, 24 and 40.
The Effect of Oral L-glutamine on Hematological Parameters - Hematocrit Baseline, Weeks 4, 24, and 40 Patient's hematocrit will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24 and 40
The Effect of Oral L-glutamine on Vital Signs - Pulse Rate Baseline, Weeks 4, 24, and 48 Patient's pulse rate will be collected at each visit, Change from Baseline will be reported at Weeks 4, 24, and 48
Effect of L-glutamine on Alcohol Use Weeks 0, 8,16, 24, 32, 40 and 48 The patient's alcohol usage will be assessed at each visit. Alcohol usage will be reported at Weeks 0, 8,16, 24, 32, 40 and 48
The Effect of Oral L-glutamine on the Number of Days Patient's Daily Activities Are Interrupted Due to Sickle Cell Pain Weeks 0, 8,16, 24, 32, 40 and 48 Percentage of days a patient's daily activities were interrupted due to sickle pain calculated at each visit. Day's interrupted will be reported at Weeks 0, 8,16, 24, 32, 40 and 48
Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Run Without Rest Baseline, Weeks 4, 24, and 48. Minutes patient could run without rest will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
Patient Appetite (3-point Scale) Collected at Week 0, 8, 16, 24, 32, 40, 48 Patient's appetite level was evaluated at each visit using a 3 point scale: above average, average and below average. The parentages of patient at each visit whose appetite level was below, normal or above average were compared using CMH test (row mean scores) controlling for study center.
Effect of L-glutamine on Tobacco Use Weeks 0, 8,16, 24, 32, 40 and 48 Patient's alcohol usage will be assessed at each visit. Alcohol usage will be reported at Weeks 0, 8,16, 24, 32, 40 and 48
The Effect of Oral L-glutamine on Vital Signs - Respiration Baseline, Weeks 4, 24, and 48 Respiration will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
The Effect of Oral L-glutamine on Subjective Quality of Life Baseline and Week 24 (or at time of discontinuation) The subjective quality of life was evaluated using the scoring of the RAND 36-Item Health Survey Questionnaire. The subjective quality of life (Physical functioning, Physical health, Emotional problems, Energy/Fatigue, Emotional well being, Social functioning, Pain, General health) will be reported at Baseline and Week 24 (or at time of discontinuation). The range for Physical functioning, Physical health, Emotional problems, Emotional well being and Social functioning is 0-100, with a high score denotes a better quality of life. For Energy/Fatigue, Pain and General health the range is 0-100, with a lower score denotes better quality of life.
Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Run Without Rest Baseline, Weeks 4, 24, and 48. Distance patient could run without rest will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
The Effect of Oral L-glutamine on Hematological Parameters - Reticulocyte Count Baseline, Weeks 0, 4, 24, 40 Patient's reticulocyte count will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24 and 40
Energy Level (11-point Scale) Collected at Week 0, 8, 16, 24, 32, 40, 48 The patient's energy level was evaluated at each visit using an 11 point scale from 0=extremely tired to 10=extremely energetic
The Effect of Oral L-glutamine on Vital Signs - Temperature Baseline, Weeks 4, 24, and 48 Patient's temperature will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
Effect of Oral L--glutamine on Weight Baseline, Weeks 4, 24 and 48 Weight will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
Trial Locations
- Locations (5)
Kaiser Permanente
🇺🇸Bellflower, California, United States
Harbor-UCLA Medical Center
🇺🇸Torrance, California, United States
Jacobi Medical Center
🇺🇸Bronx, New York, United States
Grady Memorial Hospital
🇺🇸Atlanta, Georgia, United States
University of Medicine and Dentistry, New Jersey
🇺🇸New Brunswick, New Jersey, United States