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Trial record 2 of 3 for:    Emmaus + Sickle Cell Anemia

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: August 8, 2017
Last verified: July 2017
  Purpose
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.

Condition Intervention Phase
Sickle Cell Anemia Sickle ß0-Thalassemia Drug: L-glutamine Drug: Placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, 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 ]
    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 Hospitalizations for Sickle Cell Pain [ Time Frame: 48 weeks ]
    The number of hospitalizations 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.

  • The Number of Emergency Room/Medical Facility Visits for Sickle Cell Pain [ Time Frame: 48 weeks ]
    The number of emergency room visits or medical facility visits 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.

  • The Effect of Oral -L-glutamine on Hematological Parameters [ Time Frame: Baseline, Week 4, 24 and 48 ]
    To assess the effect of oral L-glutamine on hematological parameters (hemoglobin), Change from Baseline will be reported at Weeks 4, 24 and 48.

  • The Effect of Oral L-glutamine on Vital Signs [ Time Frame: Baseline, Week 4, 24, and 48 ]
    To assess the effect of oral L-glutamine on Vital signs (systolic and diastolic blood pressure). Change from Baseline will be reported at Weeks 4, 24, and 48.

  • The Effect of Oral L-glutamine on Hematological Parameters [ Time Frame: Baseline, Week 4, 24 and 48 ]
    To assess the effect of oral L-glutamine on hematological parameters (hematocrit), Change from Baseline will be reported at Weeks 4, 24 and 48.

  • The Effect of Oral L-glutamine on Hematological Parameters [ Time Frame: Baseline, Week 4, 24 and 48 ]
    To assess the effect of oral L-glutamine on hematological parameters (reticulocyte count), Change from Baseline will be reported at Weeks 4, 24 and 48.

  • The Effect of Oral L-glutamine on Vital Signs [ Time Frame: Baseline, Week 4, Week 24 and Week 48 ]
    To assess the effect of oral L-glutamine on Vital signs (pulse rate). Change from Baseline will be reported at Weeks 4, 24, and 48.

  • Effect of Oral L-glutamine on Vital Signs [ Time Frame: Baseline, Week 4, Week 24 and Week 48 ]
    To assess the effect of oral L-glutamine on Vital signs (temperature). Change from Baseline will be reported at Weeks 4, 24, and 48.

  • The Effect of Oral L-glutamine on Vital Signs [ Time Frame: Baseline, Week 4, Week 24 and Week 48 ]
    To assess the effect of oral L-glutamine on Vital signs (respiration). Change from Baseline will be reported at Weeks 4, 24, and 48.


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.
Other Name: oral L-glutamine
Placebo Comparator: 100% maltodextrin
Patients will be randomized to receive Placebo.
Drug: Placebo
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.
Other Name: Maltodextrin

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.

  Eligibility

Ages Eligible for Study:   5 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
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.
  • 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.
Investigators
Study Director: Yutaka Niihara, MD, MPH Chairman and CEO
  More Information

Responsible Party: Emmaus Medical, Inc.
ClinicalTrials.gov Identifier: NCT01179217     History of Changes
Other Study ID Numbers: GLUSCC09-01
Study First Received: May 21, 2010
Results First Received: December 14, 2016
Last Updated: August 8, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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
Thalassemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on September 21, 2017