A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia
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ClinicalTrials.gov Identifier: NCT01179217 |
Recruitment Status
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Completed
First Posted
: August 11, 2010
Results First Posted
: August 10, 2017
Last Update Posted
: August 10, 2017
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Tracking Information | ||||
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First Submitted Date ICMJE | May 21, 2010 | |||
First Posted Date ICMJE | August 11, 2010 | |||
Results First Submitted Date | December 14, 2016 | |||
Results First Posted Date | August 10, 2017 | |||
Last Update Posted Date | August 10, 2017 | |||
Study Start Date ICMJE | May 2010 | |||
Actual Primary Completion Date | March 2014 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
The Number of Occurrences of Sickle Cell Crises [ Time Frame: 48 weeks ] 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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Original Primary Outcome Measures ICMJE | Same as current | |||
Change History | Complete list of historical versions of study NCT01179217 on ClinicalTrials.gov Archive Site | |||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE | Not Provided | |||
Current Other Outcome Measures ICMJE | Not Provided | |||
Original Other Outcome Measures ICMJE | Not Provided | |||
Descriptive Information | ||||
Brief Title ICMJE | A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia | |||
Official Title ICMJE | 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 | |||
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. | |||
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. |
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Study Type ICMJE | Interventional | |||
Study Phase | Phase 3 | |||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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Condition ICMJE |
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Intervention ICMJE |
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Study Arms |
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Publications * | Not Provided | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Completed | |||
Actual Enrollment ICMJE |
230 | |||
Original Estimated Enrollment ICMJE |
225 | |||
Actual Study Completion Date | March 2014 | |||
Actual Primary Completion Date | March 2014 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender |
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Ages | 5 Years and older (Child, Adult, Senior) | |||
Accepts Healthy Volunteers | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT01179217 | |||
Other Study ID Numbers ICMJE | GLUSCC09-01 | |||
Has Data Monitoring Committee | No | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement |
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Responsible Party | Emmaus Medical, Inc. | |||
Study Sponsor ICMJE | Emmaus Medical, Inc. | |||
Collaborators ICMJE | Not Provided | |||
Investigators ICMJE |
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PRS Account | Emmaus Medical, Inc. | |||
Verification Date | July 2017 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |