We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

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:
ClinicalTrials.gov Identifier:
NCT01179217
First Posted: August 11, 2010
Last Update Posted: August 10, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Emmaus Medical, Inc.
Results First Submitted: December 14, 2016  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Sickle Cell Anemia
Sickle ß0-Thalassemia
Interventions: Drug: L-glutamine
Drug: Placebo

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
L-glutamine

Patients will be randomized to receive investigational product, L-Glutamine.

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

Patients will be randomized to receive Placebo.

Placebo (100% maltodextrin): 0.3 g/kg of placebo 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.


Participant Flow:   Overall Study
    L-glutamine   Placebo
STARTED   152   78 
COMPLETED   97   59 
NOT COMPLETED   55   19 



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
L-glutamine

Patients will be randomized to receive investigational product, L-Glutamine.

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

Patients will be randomized to receive Placebo.

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.

Total Total of all reporting groups

Baseline Measures
   L-glutamine   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 152   78   230 
Age 
[Units: Participants]
Count of Participants
     
<=18 years      75  49.3%      43  55.1%      118  51.3% 
Between 18 and 65 years      77  50.7%      35  44.9%      112  48.7% 
>=65 years      0   0.0%      0   0.0%      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 22.4  (12.32)   21.4  (12.42)   22.0  (12.33) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      79  52.0%      45  57.7%      124  53.9% 
Male      73  48.0%      33  42.3%      106  46.1% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      0   0.0%      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      144  94.7%      73  93.6%      217  94.3% 
White      0   0.0%      0   0.0%      0   0.0% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      8   5.3%      5   6.4%      13   5.7% 
Diagnosis 
[Units: Participants]
Count of Participants
     
Sickle Cell Anemia      136  89.5%      71  91.0%      207  90.0% 
Sickle Beta plus Thalassemia      2   1.3%      0   0.0%      2   0.9% 
Sickle Beta zero Thalassemia      14   9.2%      7   9.0%      21   9.1% 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   The Number of Occurrences of Sickle Cell Crises   [ Time Frame: 48 weeks ]

2.  Secondary:   The Number of Hospitalizations for Sickle Cell Pain   [ Time Frame: 48 weeks ]

3.  Secondary:   The Number of Emergency Room/Medical Facility Visits for Sickle Cell Pain   [ Time Frame: 48 weeks ]

4.  Secondary:   The Effect of Oral -L-glutamine on Hematological Parameters   [ Time Frame: Baseline, Week 4, 24 and 48 ]

5.  Secondary:   The Effect of Oral L-glutamine on Vital Signs   [ Time Frame: Baseline, Week 4, 24, and 48 ]

6.  Secondary:   The Effect of Oral L-glutamine on Hematological Parameters   [ Time Frame: Baseline, Week 4, 24 and 48 ]

7.  Secondary:   The Effect of Oral L-glutamine on Hematological Parameters   [ Time Frame: Baseline, Week 4, 24 and 48 ]

8.  Secondary:   The Effect of Oral L-glutamine on Vital Signs   [ Time Frame: Baseline, Week 4, Week 24 and Week 48 ]

9.  Secondary:   Effect of Oral L-glutamine on Vital Signs   [ Time Frame: Baseline, Week 4, Week 24 and Week 48 ]

10.  Secondary:   The Effect of Oral L-glutamine on Vital Signs   [ Time Frame: Baseline, Week 4, Week 24 and Week 48 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
  Hide More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Yutaka Niihara, MD, MPH
Organization: Emmaus Medical, Inc
phone: 310-214-0065
e-mail: yniihara@emmausmedical.com



Responsible Party: Emmaus Medical, Inc.
ClinicalTrials.gov Identifier: NCT01179217     History of Changes
Other Study ID Numbers: GLUSCC09-01
First Submitted: May 21, 2010
First Posted: August 11, 2010
Results First Submitted: December 14, 2016
Results First Posted: August 10, 2017
Last Update Posted: August 10, 2017