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L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Emmaus Medical, Inc.
ClinicalTrials.gov Identifier:
NCT00125788
First received: August 1, 2005
Last updated: March 15, 2013
Last verified: November 2011

August 1, 2005
March 15, 2013
March 2004
July 2008   (final data collection date for primary outcome measure)
Number of occurrences of painful sickle cell crises [ Time Frame: from Week 0 through Week 48 (cumulative) ] [ Designated as safety issue: No ]
Number of occurrences of painful sickle cell crises
Complete list of historical versions of study NCT00125788 on ClinicalTrials.gov Archive Site
  • Frequency of emergency room visits for sickle cell pain [ Time Frame: from Week 0 through Week 24 (cumulative), and from Week 0 through Week 48 (cumulative) ] [ Designated as safety issue: No ]
  • Number of days patients' usual daily activities are interrupted due to sickle cell pain [ Time Frame: from Week 0 through Week 48 (cumulative) ] [ Designated as safety issue: No ]
  • Growth curve for patients less than 18 years of age [ Time Frame: at each study visit (approximately every four weeks) ] [ Designated as safety issue: No ]
    At each study visit, the patient's height and weight will be compared to the standard growth curve. The patient will be classified as below the 5th, 10th, 25th, 50th, 75th, 90th and 95th percentile for height and weight (mutually exclusive groups).
  • Hematological parameters [ Time Frame: collected at Weeks 0, 4, 8, 12, 16, 20, 24, 32, 40, 48, 53 ] [ Designated as safety issue: No ]
  • Narcotic usage [ Time Frame: ongoing from Week 0 through Week 48 ] [ Designated as safety issue: No ]
  • Alcohol and tobacco use [ Time Frame: ongoing from Week 0 through Week 48 ] [ Designated as safety issue: No ]
  • Pain level [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
  • Energy level [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
  • Patient activity level [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
  • Patient appetite [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
  • Subjective exercise tolerance [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
  • Subjective quality of life [ Time Frame: assessed at each study visit from Week 0 through Week 48 (approximately every four weeks) ] [ Designated as safety issue: No ]
  • L-glutamine safety assessment based on adverse events, laboratory parameters, and vital signs [ Time Frame: ongoing from Week 0 through Week 53 ] [ Designated as safety issue: Yes ]
  • Number of occurrences of sickle cell crises [ Time Frame: from Week 0 through Week 24 (cumulative) ] [ Designated as safety issue: No ]
  • Frequency of hospitalizations for sickle cell pain
  • Frequency of emergency room visits for sickle cell pain
  • Number of days patients’ usual daily activities are interrupted due to sickle cell pain
  • Height and weight
  • Growth curve for patients less than 18 years of age
  • Hematological parameters
  • Narcotic usage
  • Alcohol and tobacco use
  • Pain level
  • Energy level
  • Patient activity level
  • Patient appetite
  • Subjective exercise tolerance
  • Subjective quality of life
  • L-glutaime safety assessment based on adverse events, laboratory parameters, and vital signs
Not Provided
Not Provided
 
L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
A Phase II, Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study of L Glutamine Therapy for Sickle Cell Anemia and Sickle ß0-Thalassemia

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 on pain; energy and appetite levels; narcotics usage; height and weight; and hospital and emergency room visits for sickle cell pain.

Not Provided
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Sickle Cell Anemia
  • Thalassemia
  • Drug: L-glutamine
    Approximately 0.3 g/kg total daily dose of L-glutamine will be orally administered (over two doses), with a maximum total daily dose of 30 grams.
  • Drug: maltodextrin
    Approximately 0.3 g/kg total daily dose of maltodextrin placebo will be orally administered (over two doses), with a maximum total daily dose of 30 grams.
  • Experimental: investigational product
    L-glutamine
    Intervention: Drug: L-glutamine
  • Placebo Comparator: placebo
    maltodextrin
    Intervention: Drug: maltodextrin
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
81
July 2008
July 2008   (final data collection date for primary outcome measure)

Inclusion Criteria:

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.

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) > 2.0.
  • 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.
Both
5 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00125788
10478
No
Emmaus Medical, Inc.
Emmaus Medical, Inc.
FDA Office of Orphan Products Development
Principal Investigator: Yutaka Niihara, MD CEO, Emmaus Medical, Inc
Emmaus Medical, Inc.
November 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP