Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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
  Purpose

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.


Condition Intervention Phase
Sickle Cell Anemia
Thalassemia
Drug: L-glutamine
Drug: maltodextrin
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Emmaus Medical, Inc.:

Primary Outcome Measures:
  • Number of occurrences of painful sickle cell crises [ Time Frame: from Week 0 through Week 48 (cumulative) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ]

Enrollment: 81
Study Start Date: March 2004
Study Completion Date: July 2008
Primary Completion Date: July 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: investigational product
L-glutamine
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.
Placebo Comparator: placebo
maltodextrin
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.

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00125788

Locations
United States, California
Kaiser Permanente
Bellflower, California, United States, 90706
Harbor-UCLA Medical Center
Torrance, California, United States, 90502
United States, Georgia
Grady Memorial Hospital
Atlanta, Georgia, United States, 30303
United States, New Jersey
University of Medicine and Dentistry, New Jersey
New Brunswick, New Jersey, United States, 08903
United States, New York
Jacobi Medical Center
Bronx, New York, United States, 10461
Sponsors and Collaborators
Emmaus Medical, Inc.
Investigators
Principal Investigator: Yutaka Niihara, MD CEO, Emmaus Medical, Inc
  More Information

No publications provided

Responsible Party: Emmaus Medical, Inc.
ClinicalTrials.gov Identifier: NCT00125788     History of Changes
Obsolete Identifiers: NCT00029887
Other Study ID Numbers: 10478
Study First Received: August 1, 2005
Last Updated: March 15, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Emmaus Medical, Inc.:
sickle cell disease
sickle cell anemia
L-glutamine
Sickle Cell Anemia (homozygous)
Sickle ß0-Thalassemia

Additional relevant MeSH terms:
Anemia
Anemia, Sickle Cell
Thalassemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies

ClinicalTrials.gov processed this record on November 27, 2014