- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01179217
A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia
A PHASE III, PROSPECTIVE, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTER STUDY OF L GLUTAMINE THERAPY FOR SICKLE CELL ANEMIA AND SICKLE ß0-THALASSEMIA
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Primary objective:
To evaluate the efficacy of oral L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia as evaluated by the number of occurrences of sickle cell crises.
Secondary objectives:
To assess the effect of oral L-glutamine on: (a) frequency of hospitalizations for sickle cell pain; (b) frequency of emergency room/medical facility visits for sickle cell pain; and (c) hematological parameters (hemoglobin, hematocrit, and reticulocyte count); and to assess the safety of L-glutamine as a therapy for sickle cell anemia as evaluated by adverse events, laboratory parameters, and vital signs.
Methodology:
This was a 2:1 randomized, double-blind, placebo-controlled, parallel-group, multicenter study in patients with sickle cell anemia and sickle ß0-thalassemia who were at least 5 years old. Informed consent was obtained up to four weeks prior to Week 0 (Baseline). Screening procedures were performed anytime between the date of consent and Week 0, as long as all eligibility criteria had been confirmed prior to Week 0. At Week 0, patients were randomized (to L-glutamine or placebo) and underwent 48 weeks of treatment (orally BID), with dose calculated according to patient weight. Patient clinic visits occurred every 4 weeks, and phone calls took place between visits to monitor compliance. After 48 weeks of treatment, the dose was tapered to 0 within 3 weeks. A final evaluation visit occurred 2 weeks after last dose for a total of 53 weeks on study.
Study Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
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Alabama
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Mobile, Alabama, United States, 36617
- University of South Alabama Medical Center
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Arizona
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Phoenix, Arizona, United States, 85016
- Phoenix Children's Hospital Center for Cancer and Blood Disorders
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California
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Inglewood, California, United States, 90301
- Kaiser Permanente
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Oakland, California, United States, 94609
- Children'S Hospital & Research Center At Oakland
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Orange, California, United States, 92868
- Children's Hospital of Orange County
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Torrance, California, United States, 90509
- Harbor-UCLA Medical Center
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Colorado
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Aurora, Colorado, United States, 80045
- University of Denver School of Medicine Sickle Cell Treatment & Research Center
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District of Columbia
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Washington, District of Columbia, United States, 20060
- Howard University Hospital & Howard University
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Florida
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Gainesville, Florida, United States, 32610-0296
- University of Florida
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Saint Petersburg, Florida, United States, 33701
- All Children's Hospital
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Georgia
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Atlanta, Georgia, United States, 30322
- Children's Healthcare of Atlanta at Egleston/Emory University
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Illinois
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Chicago, Illinois, United States, 60612
- University of Illinois at Chicago
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Kentucky
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Louisville, Kentucky, United States, 40202
- University of Louisville School of Medicine
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Louisiana
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New Orleans, Louisiana, United States, 70112
- Sickle Cell Center of S. Louisiana, Tulane University School of Medicine
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Maryland
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Baltimore, Maryland, United States, 21205
- Johns Hopkins University
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Massachusetts
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Boston, Massachusetts, United States, 02118
- Boston University Medical Center
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Michigan
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Detroit, Michigan, United States, 48201
- Children's Hospital of Michigan
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Mississippi
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Jackson, Mississippi, United States, 39216
- University of Mississippi Medical Center
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Missouri
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Kansas City, Missouri, United States, 64108
- Children's Mercy Hospitals and Clinics
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Nevada
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Las Vegas, Nevada, United States, 89109
- Children's Specialty Center of Nevada
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Las Vegas, Nevada, United States, 89109
- Comprehensive Cancer Center of Nevada
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New Jersey
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Camden, New Jersey, United States, 08103
- Cooper University Hospital
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New York
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Bronx, New York, United States, 11203
- Bronx Lebanon Hospital
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Brooklyn, New York, United States, 11212
- Brookdale University Hospital and Medical Center
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Brooklyn, New York, United States, 11201
- The Brooklyn Hospital Center
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Brooklyn, New York, United States, 11203
- SUNY - Downstate Medical Center
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Brooklyn, New York, United States, 11215
- New York Methodist Hospital - SC/Thalassemia Program
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Brooklyn, New York, United States, 11238
- Interfaith Medical Center
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North Carolina
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Charlotte, North Carolina, United States, 28204
- Presbyterian Blume Pediatric Hematology-Oncology Clinic
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Tennessee
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Memphis, Tennessee, United States, 38104
- University of Tennessee Cancer Institute
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Virginia
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Richmond, Virginia, United States, 23298-0306
- Virginia Commonwealth University
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Patient is at least five years of age.
- Patient has been diagnosed with sickle cell anemia or sickle ß°-thalassemia (documented by hemoglobin electrophoresis).
- Patient has had at least two documented episodes of sickle cell 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 duration of the study.
- 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 avoid pregnancy during the study and is willing and agrees to practice a recognized form of birth control during the course of the study (e.g. barrier, birth control pills, abstinence).
Exclusion Criteria:
- Patient has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit.
- Patient has prothrombin time 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 uncontrolled liver disease or renal insufficiency.
- Patient is pregnant or lactating or has the intention of becoming pregnant during the study (if female and of child-bearing potential).
- Patient is currently taking or has been treated with any form of glutamine supplement within 30 days of the screening visit.
- Patient has been treated with an experimental anti-sickling medication/ treatment within 30 days of the screening visit (with the exception of hydroxyurea in pediatric patients).
- Patient is currently taking or has been treated with an investigational drug within 30 days of the screening visit (with the exception of hydroxyurea in pediatric patients).
- Patient is currently enrolled in an investigational drug or device study and/or has participated in such a study 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 Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: L-glutamine
Patients will be randomized to receive investigational product, L-Glutamine.
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0.3 g/kg of L-glutamine will be administered twice a day orally to each patient for 48 weeks.
The dosage will be in increments of 5 grams based on weight.
The upper limit for daily dose of study medication will be set at 30 grams.
Patients will be given verbal and written instructions for self-administration of the study medication at the Baseline visit.
The powder can be mixed with water or most non-heated beverages other than alcohol, or can be mixed with most non-heated foods such as yogurt, applesauce, or cereal for administration.
Mixing L-glutamine with soda or highly acidic juices (such as grapefruit juice or lemonade) is not recommended.
Other Names:
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Placebo Comparator: 100% maltodextrin
Patients will be randomized to receive Placebo.
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0.3 g/kg of placebo (100% maltodextrin) will be administered twice a day orally to each patient for 48 weeks.
The dosage will be in increments of 5 grams based on weight.
The upper limit for daily dose of study medication will be set at 30 grams.
Patients will be given verbal and written instructions for self-administration of the study medication at the Baseline visit.
The powder can be mixed with water or most non-heated beverages other than alcohol, or can be mixed with most non-heated foods such as yogurt, applesauce, or cereal for administration.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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The Number of Occurrences of Sickle Cell Crises
Time Frame: 48 weeks
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The number of occurrences of protocol-defined sickle cell crises that occur from Week 0 to Week 48 will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia.
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48 weeks
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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The Number of Hospitalizations for Sickle Cell Pain
Time Frame: 48 weeks
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The number of hospitalizations that occur from Week 0 to Week 48, will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia.
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48 weeks
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The Number of Emergency Room/Medical Facility Visits for Sickle Cell Pain
Time Frame: 48 weeks
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The number of emergency room visits or medical facility visits that occur from Week 0 to Week 48, will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia.
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48 weeks
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The Effect of Oral -L-glutamine on Hematological Parameters
Time Frame: Baseline, Week 4, 24 and 48
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To assess the effect of oral L-glutamine on hematological parameters (hemoglobin), Change from Baseline will be reported at Weeks 4, 24 and 48.
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Baseline, Week 4, 24 and 48
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The Effect of Oral L-glutamine on Vital Signs
Time Frame: Baseline, Week 4, 24, and 48
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To assess the effect of oral L-glutamine on Vital signs (systolic and diastolic blood pressure).
Change from Baseline will be reported at Weeks 4, 24, and 48.
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Baseline, Week 4, 24, and 48
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The Effect of Oral L-glutamine on Hematological Parameters
Time Frame: Baseline, Week 4, 24 and 48
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To assess the effect of oral L-glutamine on hematological parameters (hematocrit), Change from Baseline will be reported at Weeks 4, 24 and 48.
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Baseline, Week 4, 24 and 48
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The Effect of Oral L-glutamine on Hematological Parameters
Time Frame: Baseline, Week 4, 24 and 48
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To assess the effect of oral L-glutamine on hematological parameters (reticulocyte count), Change from Baseline will be reported at Weeks 4, 24 and 48.
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Baseline, Week 4, 24 and 48
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The Effect of Oral L-glutamine on Vital Signs
Time Frame: Baseline, Week 4, Week 24 and Week 48
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To assess the effect of oral L-glutamine on Vital signs (pulse rate).
Change from Baseline will be reported at Weeks 4, 24, and 48.
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Baseline, Week 4, Week 24 and Week 48
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Effect of Oral L-glutamine on Vital Signs
Time Frame: Baseline, Week 4, Week 24 and Week 48
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To assess the effect of oral L-glutamine on Vital signs (temperature).
Change from Baseline will be reported at Weeks 4, 24, and 48.
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Baseline, Week 4, Week 24 and Week 48
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The Effect of Oral L-glutamine on Vital Signs
Time Frame: Baseline, Week 4, Week 24 and Week 48
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To assess the effect of oral L-glutamine on Vital signs (respiration).
Change from Baseline will be reported at Weeks 4, 24, and 48.
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Baseline, Week 4, Week 24 and Week 48
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Yutaka Niihara, MD, MPH, Chairman and CEO
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- GLUSCC09-01
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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