A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Emmaus Medical, Inc.
ClinicalTrials.gov Identifier:
NCT01179217
First received: May 21, 2010
Last updated: June 24, 2014
Last verified: June 2014
  Purpose

The purpose of this research is to test the effects of L-glutamine on red blood cells of patients with Sickle Cell Anemia or Sickle ß0-Thalassemia. Glutamine is a part of protein and is a part of a person's normal diet. It is often used as a nutritional supplement or as medication for various medical problems such as intestinal problems. The benefits of participating in this study may be a decrease in pain and other problems caused by sickle cell disease.


Condition Intervention Phase
Sickle Cell Anemia
Sickle ß0-Thalassemia
Drug: L-glutamine
Drug: 100% maltodextrin
Phase 3

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 III, 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:
  • The number of occurrences of sickle cell crises [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • The number of occurrences of sickle cell crises [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
    The number of occurrences of protocol-defined sickle cell crises that occur from Week 0 to Week 24 will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia.

  • Number of hospitalizations for sickle cell pain [ Time Frame: 24 weeks; 48 weeks ] [ Designated as safety issue: No ]
    The number of hospitalizations that occur from Week 0 to Week 24, and 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.

  • Number of emergency room/medical facility visits for sickle cell pain [ Time Frame: 24 weeks; 48 weeks ] [ Designated as safety issue: No ]
    The number of emergency room visits or medical facility visits that occur from Week 0 to Week 24, and 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.

  • Hematological parameters [ Time Frame: 24 weeks; 48 weeks ] [ Designated as safety issue: No ]
    Change from Baseline (Week 0) for hemoglobin, hematocrit, reticulocyte count will be assessed at 24 weeks and 48 weeks to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia.

  • Adverse events [ Time Frame: Week 0 - study exit ] [ Designated as safety issue: Yes ]
    All adverse events that occur between Baseline (Week 0) and the completion of study participation will be documented.

  • Laboratory parameters and vital signs [ Time Frame: Screening through study exit ] [ Designated as safety issue: Yes ]
    Complete Blood Count (CBC) and reticulocyte count will be collected at Screening, Baseline, and Weeks 4, 8, 12, 16, 20, 24, 32, 40, 48, and 53. Serum chemistry and hepatic panel will be collected at Screening and at study exit. Vital signs will be recorded at each study visit.


Enrollment: 230
Study Start Date: May 2010
Study Completion Date: March 2014
Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: L-glutamine
Patients will be randomized to receive investigational product, L-Glutamine.
Drug: L-glutamine
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.
Placebo Comparator: 100% maltodextrin
Patients will be randomized to receive Placebo.
Drug: 100% maltodextrin
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

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

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 (refer to Appendix 3 for guidelines).
  • 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.
  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: NCT01179217

  Show 31 Study Locations
Sponsors and Collaborators
Emmaus Medical, Inc.
  More Information

No publications provided

Responsible Party: Emmaus Medical, Inc.
ClinicalTrials.gov Identifier: NCT01179217     History of Changes
Other Study ID Numbers: GLUSCC09-01
Study First Received: May 21, 2010
Last Updated: June 24, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Emmaus Medical, Inc.:
Sickle Cell Anemia
Sickle Cell Pain Crises
Painful Crises
Sickle Cell Pain
Vaso-occlusive Crises

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

ClinicalTrials.gov processed this record on July 31, 2014