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
1 other identifier
interventional
230
1 country
31
Brief Summary
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for Sickle Cell Anemia or Sickle ß0 Thalassemia as evaluated by the number of occurrences of sickle cell crises.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started May 2010
Typical duration for phase_3
31 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 1, 2010
CompletedFirst Submitted
Initial submission to the registry
May 21, 2010
CompletedFirst Posted
Study publicly available on registry
August 11, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2014
CompletedResults Posted
Study results publicly available
August 10, 2017
CompletedAugust 19, 2020
July 1, 2017
3.8 years
May 21, 2010
December 14, 2016
August 17, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The Number of Occurrences of Sickle Cell Crises
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.
48 weeks
Secondary Outcomes (9)
The Number of Hospitalizations for Sickle Cell Pain
48 weeks
The Number of Emergency Room/Medical Facility Visits for Sickle Cell Pain
48 weeks
The Effect of Oral -L-glutamine on Hematological Parameters
Baseline, Week 4, 24 and 48
The Effect of Oral L-glutamine on Vital Signs
Baseline, Week 4, 24, and 48
The Effect of Oral L-glutamine on Hematological Parameters
Baseline, Week 4, 24 and 48
- +4 more secondary outcomes
Study Arms (2)
L-glutamine
EXPERIMENTALPatients will be randomized to receive investigational product, L-Glutamine.
100% maltodextrin
PLACEBO COMPARATORPatients will be randomized to receive Placebo.
Interventions
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.
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.
Eligibility Criteria
You may qualify if:
- 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).
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (31)
University of South Alabama Medical Center
Mobile, Alabama, 36617, United States
Phoenix Children's Hospital Center for Cancer and Blood Disorders
Phoenix, Arizona, 85016, United States
Kaiser Permanente
Inglewood, California, 90301, United States
Children's Hospital & Research Center at Oakland
Oakland, California, 94609, United States
Children's Hospital of Orange County
Orange, California, 92868, United States
Harbor-UCLA Medical Center
Torrance, California, 90509, United States
University of Denver School of Medicine Sickle Cell Treatment & Research Center
Aurora, Colorado, 80045, United States
Howard University Hospital & Howard University
Washington D.C., District of Columbia, 20060, United States
University of Florida
Gainesville, Florida, 32610-0296, United States
All Children's Hospital
St. Petersburg, Florida, 33701, United States
Children's Healthcare of Atlanta at Egleston/Emory University
Atlanta, Georgia, 30322, United States
University of Illinois at Chicago
Chicago, Illinois, 60612, United States
University of Louisville School of Medicine
Louisville, Kentucky, 40202, United States
Sickle Cell Center of S. Louisiana, Tulane University School of Medicine
New Orleans, Louisiana, 70112, United States
Johns Hopkins University
Baltimore, Maryland, 21205, United States
Boston University Medical Center
Boston, Massachusetts, 02118, United States
Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
University of Mississippi Medical Center
Jackson, Mississippi, 39216, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
Children's Specialty Center of Nevada
Las Vegas, Nevada, 89109, United States
Comprehensive Cancer Center of Nevada
Las Vegas, Nevada, 89109, United States
Cooper University Hospital
Camden, New Jersey, 08103, United States
The Brooklyn Hospital Center
Brooklyn, New York, 11201, United States
SUNY - Downstate Medical Center
Brooklyn, New York, 11203, United States
Brookdale University Hospital and Medical Center
Brooklyn, New York, 11212, United States
New York Methodist Hospital - SC/Thalassemia Program
Brooklyn, New York, 11215, United States
Interfaith Medical Center
Brooklyn, New York, 11238, United States
Bronx Lebanon Hospital
The Bronx, New York, 11203, United States
Presbyterian Blume Pediatric Hematology-Oncology Clinic
Charlotte, North Carolina, 28204, United States
University of Tennessee Cancer Institute
Memphis, Tennessee, 38104, United States
Virginia Commonwealth University
Richmond, Virginia, 23298-0306, United States
Related Publications (2)
Bolarinwa AB, Oduwole O, Okebe J, Ogbenna AA, Otokiti OE, Olatinwo AT. Antioxidant supplementation for sickle cell disease. Cochrane Database Syst Rev. 2024 May 22;5(5):CD013590. doi: 10.1002/14651858.CD013590.pub2.
PMID: 38775255DERIVEDNiihara Y, Miller ST, Kanter J, Lanzkron S, Smith WR, Hsu LL, Gordeuk VR, Viswanathan K, Sarnaik S, Osunkwo I, Guillaume E, Sadanandan S, Sieger L, Lasky JL, Panosyan EH, Blake OA, New TN, Bellevue R, Tran LT, Razon RL, Stark CW, Neumayr LD, Vichinsky EP; Investigators of the Phase 3 Trial of l-Glutamine in Sickle Cell Disease. A Phase 3 Trial of l-Glutamine in Sickle Cell Disease. N Engl J Med. 2018 Jul 19;379(3):226-235. doi: 10.1056/NEJMoa1715971.
PMID: 30021096DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Yutaka Niihara, MD, MPH
- Organization
- Emmaus Medical, Inc
Study Officials
- STUDY DIRECTOR
Yutaka Niihara, MD, MPH
Chairman and CEO
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 21, 2010
First Posted
August 11, 2010
Study Start
May 1, 2010
Primary Completion
March 1, 2014
Study Completion
March 1, 2014
Last Updated
August 19, 2020
Results First Posted
August 10, 2017
Record last verified: 2017-07
Data Sharing
- IPD Sharing
- Will not share