We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

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.
 
ClinicalTrials.gov Identifier: NCT00125788
Recruitment Status : Completed
First Posted : August 2, 2005
Results First Posted : January 29, 2021
Last Update Posted : January 29, 2021
Sponsor:
Collaborator:
FDA Office of Orphan Products Development
Information provided by (Responsible Party):
Emmaus Medical, Inc.

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
Thalassemia
Interventions Drug: L-glutamine
Drug: Placebo
Enrollment 70
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description

L-glutamine

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.

maltodextrin

Placebo: 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.
Period Title: Overall Study
Started 37 33
Completed 33 29
Not Completed 4 4
Arm/Group Title Investigational Product Placebo Total
Hide Arm/Group Description

L-glutamine

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.

maltodextrin

Placebo: 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.

 Total of all reporting groups
Overall Number of Baseline Participants 33 29 62
Hide Baseline Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62).
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants 29 participants 62 participants
<=18 years
3
   9.1%
3
  10.3%
6
   9.7%
Between 18 and 65 years
30
  90.9%
26
  89.7%
56
  90.3%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 33 participants 29 participants 62 participants
30.5  (10.09) 26.5  (9.42) 28.6  (13.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants 29 participants 62 participants
Female
22
  66.7%
10
  34.5%
32
  51.6%
Male
11
  33.3%
19
  65.5%
30
  48.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 33 participants 29 participants 62 participants
33 29 62
1.Primary Outcome
Title Number of Occurrences of Painful Sickle Cell Crises
Hide Description The mean number of painful sickle crisis through week 48
Time Frame From Week 0 through Week 48 (cumulative)
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: Crisis
4.5  (5.37) 10.8  (18.74)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Investigational Product, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.076
Comments [Not Specified]
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
2.Secondary Outcome
Title Frequency of Hospitalizations for Sickle Cell Pain
Hide Description The mean number of hospitalizations through week 48
Time Frame From Week 0 through Week 48 (cumulative)
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: Hosptalizations
1.5  (2.46) 2.3  (2.42)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Investigational Product, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.072
Comments [Not Specified]
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
3.Secondary Outcome
Title Frequency of Emergency Room Visits for Sickle Cell Pain
Hide Description The mean number of emergency room visits through week 48
Time Frame From Week 0 through Week 48 (cumulative)
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: Emergency room visits
3.7  (5.63) 9.4  (19.91)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Investigational Product, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.129
Comments [Not Specified]
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
4.Secondary Outcome
Title The Effect of Oral L-glutamine on Hematological Parameters - Hemoglobin
Hide Description Patient's hemoglobin will be collected at each visit.Change from Baseline will be reported at Weeks 4, 24 and 40.
Time Frame Baseline, Weeks 4, 24 and 40
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all patients who received at least one dose of study medication (N = 70).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 37 33
Mean (Standard Deviation)
Unit of Measure: g/dL
Baseline Number Analyzed 37 participants 33 participants
8.96  (1.477) 9.05  (1.219)
Change in Hemoglobin at Week 4 Number Analyzed 34 participants 27 participants
0.15  (0.831) -0.13  (0.861)
Change in Hemoglobin at Week 24 Number Analyzed 22 participants 21 participants
0.26  (0.641) 0.16  (0.607)
Change in Hemoglobin at Week 40 Number Analyzed 18 participants 12 participants
0.11  (0.749) -0.07  (0.650)
5.Secondary Outcome
Title The Effect of Oral L-glutamine on Hematological Parameters - Hematocrit
Hide Description Patient's hematocrit will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24 and 40
Time Frame Baseline, Weeks 4, 24, and 40
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all patients who received at least one dose of study medication (N = 70).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 37 33
Mean (Standard Deviation)
Unit of Measure: % RBC
Baseline Number Analyzed 37 participants 33 participants
25.90  (4.493) 26.22  (4.125)
Change in Hematocrit at Week 4 Number Analyzed 34 participants 27 participants
0.51  (2.784) -0.09  (2.279)
Change in Hematocrit at Week 24 Number Analyzed 22 participants 21 participants
1.08  (2.352) 0.58  (2.050)
Change in Hematocrit at Week 40 Number Analyzed 18 participants 12 participants
0.49  (2.786) 0.26  (1.869)
6.Secondary Outcome
Title The Effect of Oral L-glutamine on Hematological Parameters - Reticulocyte Count
Hide Description Patient's reticulocyte count will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24 and 40
Time Frame Baseline, Weeks 0, 4, 24, 40
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all patients who received at least one dose of study medication (N = 70).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 37 33
Mean (Standard Deviation)
Unit of Measure: 10^12 Reticulocytes/L
Baseline Number Analyzed 37 participants 33 participants
0.266  (0.1125) 0.295  (0.0979)
Change in Reticulocyte count at Week 4 Number Analyzed 19 participants 14 participants
-0.040  (0.0993) -0.034  (0.0689)
Change in Reticulocyte count at Week 24 Number Analyzed 12 participants 8 participants
-0.022  (0.0917) -0.032  (0.1032)
Change in Reticulocyte count at Week 40 Number Analyzed 10 participants 3 participants
-0.016  (0.1357) -0.020  (0.0908)
7.Secondary Outcome
Title Number of Participants With Narcotic Usage
Hide Description Analysis of narcotic usage was performed for the subset of patients with any narcotic use who completed the study. Changes in narcotic usage were determined by an independent consultant prior to database lock using morphine equivalents to determine relative use.
Time Frame Week 24, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Patients with any narcotic use who completed the visit
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 17 16
Measure Type: Count of Participants
Unit of Measure: Participants
Change in Narcotic Usage at Week 24 Change to weaker narcotics
6
  35.3%
2
  12.5%
No change
10
  58.8%
9
  56.3%
Change to stronger narcotics
1
   5.9%
5
  31.3%
Change in Narcotic Usage at Week 48 Change to weaker narcotics
6
  35.3%
2
  12.5%
No change
10
  58.8%
9
  56.3%
Change to stronger narcotics
1
   5.9%
5
  31.3%
8.Secondary Outcome
Title Energy Level (11-point Scale)
Hide Description The patient's energy level was evaluated at each visit using an 11 point scale from 0=extremely tired to 10=extremely energetic
Time Frame Collected at Week 0, 8, 16, 24, 32, 40, 48
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: score on a scale
Week 0 Number Analyzed 33 participants 29 participants
6.1  (1.92) 6.8  (1.99)
Week 8 Number Analyzed 26 participants 21 participants
6.9  (1.84) 7.1  (1.96)
Week 16 Number Analyzed 28 participants 21 participants
6.9  (2.02) 7.1  (2.70)
Week 24 Number Analyzed 21 participants 18 participants
7.3  (1.65) 7.8  (2.02)
Week 32 Number Analyzed 19 participants 14 participants
7.3  (1.67) 7.4  (1.95)
Week40 Number Analyzed 18 participants 13 participants
6.6  (1.54) 6.9  (2.29)
Week 48 Number Analyzed 24 participants 22 participants
6.8  (1.92) 7.3  (1.83)
9.Secondary Outcome
Title Patient Appetite (3-point Scale)
Hide Description Patient's appetite level was evaluated at each visit using a 3 point scale: above average, average and below average. The parentages of patient at each visit whose appetite level was below, normal or above average were compared using CMH test (row mean scores) controlling for study center.
Time Frame Collected at Week 0, 8, 16, 24, 32, 40, 48
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Measure Type: Count of Participants
Unit of Measure: Participants
Patient appetite since the last visit - Visit 0 Number Analyzed 33 participants 29 participants
Above average
4
  12.1%
6
  20.7%
Average
24
  72.7%
22
  75.9%
Below average
5
  15.2%
1
   3.4%
Not known
0
   0.0%
0
   0.0%
Patient appetite since the last visit - Visit 8 Number Analyzed 26 participants 22 participants
Above average
4
  15.4%
6
  27.3%
Average
19
  73.1%
14
  63.6%
Below average
2
   7.7%
2
   9.1%
Not known
1
   3.8%
0
   0.0%
Patient appetite since the last visit - Visit 16 Number Analyzed 28 participants 22 participants
Above average
3
  10.7%
9
  40.9%
Average
18
  64.3%
11
  50.0%
Below average
7
  25.0%
2
   9.1%
Not known
0
   0.0%
0
   0.0%
Patient appetite since the last visit - Visit 24 Number Analyzed 22 participants 20 participants
Above average
4
  18.2%
7
  35.0%
Average
17
  77.3%
11
  55.0%
Below average
1
   4.5%
2
  10.0%
Not known
0
   0.0%
0
   0.0%
Patient appetite since the last visit - Visit 32 Number Analyzed 19 participants 15 participants
Above average
3
  15.8%
3
  20.0%
Average
15
  78.9%
12
  80.0%
Below average
1
   5.3%
0
   0.0%
Not known
0
   0.0%
0
   0.0%
Patient appetite since the last visit - Visit 40 Number Analyzed 18 participants 13 participants
Above average
2
  11.1%
4
  30.8%
Average
13
  72.2%
7
  53.8%
Below average
3
  16.7%
2
  15.4%
Not known
0
   0.0%
0
   0.0%
Patient appetite since the last visit - Visit 48 Number Analyzed 17 participants 12 participants
Above average
2
  11.8%
2
  16.7%
Average
13
  76.5%
9
  75.0%
Below average
2
  11.8%
1
   8.3%
Not known
0
   0.0%
0
   0.0%
10.Secondary Outcome
Title The Effect of Oral L-glutamine on Vital Signs - Blood Pressure
Hide Description Patient's blood pressure will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
Time Frame Baseline, Weeks 4, 24, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all patients who received at least one dose of study medication (N = 70).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 37 33
Mean (Standard Deviation)
Unit of Measure: mm/Hg
Baseline SBP (mm/Hg) Number Analyzed 37 participants 33 participants
115.18  (14.30) 119.6  (13.64)
Change in SBP (mm/Hg) at Week 4 Number Analyzed 33 participants 26 participants
0.7  (10.98) -0.9  (10.81)
Change in SBP (mm/Hg) at Week 24 Number Analyzed 23 participants 20 participants
2.2  (10.48) -1.9  (13.06)
Change in SBP (mm/Hg) at Week 48 Number Analyzed 18 participants 13 participants
-1.2  (12.07) -1.5  (12.97)
Baseline DBP (mm/hg) Number Analyzed 37 participants 33 participants
63.7  (9.05) 64.9  (8.09)
Change in DBP (mm/Hg) at Week 4 Number Analyzed 33 participants 26 participants
1.2  (8.09) 2.2  (8.73)
Change in DBP (mm/Hg) at Week 24 Number Analyzed 23 participants 20 participants
1.2  (9.55) -0.4  (6.88)
Change in DBP (mm/Hg) at Week 48 Number Analyzed 18 participants 13 participants
-1.3  (7.98) 0.5  (7.91)
11.Secondary Outcome
Title The Effect of Oral L-glutamine on Vital Signs - Temperature
Hide Description Patient's temperature will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
Time Frame Baseline, Weeks 4, 24, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all patients who received at least one dose of study medication (N = 70).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 37 33
Mean (Standard Deviation)
Unit of Measure: deg C
Baseline Number Analyzed 37 participants 33 participants
36.40  (0.686) 36.29  (0.613)
Change in Temperature at Week 4 Number Analyzed 33 participants 27 participants
-0.05  (0.377) -0.06  (0.269)
Change Temperature at Week 24 Number Analyzed 23 participants 20 participants
-0.01  (0.261) -0.07  (0.268)
Change in Temperature at Week 48 Number Analyzed 18 participants 13 participants
0.08  (0.362) -0.09  (0.256)
12.Secondary Outcome
Title The Effect of Oral L-glutamine on Vital Signs - Respiration
Hide Description Respiration will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
Time Frame Baseline, Weeks 4, 24, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all patients who received at least one dose of study medication (N = 70).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 37 33
Mean (Standard Deviation)
Unit of Measure: resp/min
Baseline Number Analyzed 37 participants 33 participants
17.9  (2.11) 17.7  (1.94)
Change in Respiration at Week 4 Number Analyzed 33 participants 27 participants
0.3  (1.71) 0.6  (1.58)
Change in Respiration at Week 24 Number Analyzed 23 participants 20 participants
0.4  (1.73) -0.4  (1.93)
Change in Respiration at Week 48 Number Analyzed 18 participants 13 participants
0.4  (2.12) 0.6  (2.47)
13.Secondary Outcome
Title The Effect of Oral L-glutamine on Vital Signs - Pulse Rate
Hide Description Patient's pulse rate will be collected at each visit, Change from Baseline will be reported at Weeks 4, 24, and 48
Time Frame Baseline, Weeks 4, 24, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all patients who received at least one dose of study medication (N = 70).
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 37 33
Mean (Standard Deviation)
Unit of Measure: bpm
Baseline Number Analyzed 37 participants 33 participants
81.6  (11.37) 80.5  (11.95)
Change in Pulse Rate at Week 4 Number Analyzed 33 participants 27 participants
2.5  (12.88) 0.7  (12.11)
Change in Pulse Rate at Week 24 Number Analyzed 23 participants 20 participants
1.7  (13.69) -2.7  (12.81)
Change in Pulse Rate at Week 48 Number Analyzed 18 participants 13 participants
1.3  (14.53) 3.9  (10.07)
14.Secondary Outcome
Title Effect of L-glutamine on Alcohol Use
Hide Description The patient's alcohol usage will be assessed at each visit. Alcohol usage will be reported at Weeks 0, 8,16, 24, 32, 40 and 48
Time Frame Weeks 0, 8,16, 24, 32, 40 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Measure Type: Count of Participants
Unit of Measure: Participants
Week 0 Number Analyzed 33 participants 29 participants
NO - Alcohol Use
30
  90.9%
27
  93.1%
YES - Alcohol Use
3
   9.1%
2
   6.9%
Week 8 Number Analyzed 26 participants 22 participants
NO - Alcohol Use
25
  96.2%
20
  90.9%
YES - Alcohol Use
1
   3.8%
2
   9.1%
Week 16 Number Analyzed 28 participants 22 participants
NO - Alcohol Use
27
  96.4%
20
  90.9%
YES - Alcohol Use
1
   3.6%
2
   9.1%
Week 24 Number Analyzed 22 participants 20 participants
NO - Alcohol Use
22
 100.0%
19
  95.0%
YES - Alcohol Use
0
   0.0%
1
   5.0%
Week 32 Number Analyzed 19 participants 15 participants
NO - Alcohol Use
19
 100.0%
13
  86.7%
YES - Alcohol Use
0
   0.0%
2
  13.3%
Week 40 Number Analyzed 18 participants 13 participants
NO - Alcohol Use
18
 100.0%
11
  84.6%
YES - Alcohol Use
0
   0.0%
2
  15.4%
Week 48 Number Analyzed 17 participants 12 participants
NO - Alcohol Use
17
 100.0%
11
  91.7%
YES - Alcohol Use
0
   0.0%
1
   8.3%
15.Secondary Outcome
Title Effect of L-glutamine on Tobacco Use
Hide Description Patient's alcohol usage will be assessed at each visit. Alcohol usage will be reported at Weeks 0, 8,16, 24, 32, 40 and 48
Time Frame Weeks 0, 8,16, 24, 32, 40 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Measure Type: Count of Participants
Unit of Measure: Participants
Week 0 Number Analyzed 33 participants 29 participants
NO - Tobacco use
28
  84.8%
27
  93.1%
YES - Tobacco use
5
  15.2%
2
   6.9%
Week 8 Number Analyzed 26 participants 22 participants
NO - Tobacco use
23
  88.5%
21
  95.5%
YES - Tobacco use
3
  11.5%
1
   4.5%
Week 16 Number Analyzed 28 participants 22 participants
NO - Tobacco use
24
  85.7%
22
 100.0%
YES - Tobacco use
4
  14.3%
0
   0.0%
Week 24 Number Analyzed 22 participants 20 participants
NO - Tobacco use
20
  90.9%
19
  95.0%
YES - Tobacco use
2
   9.1%
1
   5.0%
Week 32 Number Analyzed 19 participants 15 participants
NO - Tobacco use
18
  94.7%
15
 100.0%
YES - Tobacco use
1
   5.3%
0
   0.0%
Week 40 Number Analyzed 18 participants 13 participants
NO - Tobacco use
17
  94.4%
13
 100.0%
YES - Tobacco use
1
   5.6%
0
   0.0%
Week 48 Number Analyzed 17 participants 12 participants
NO - Tobacco use
16
  94.1%
12
 100.0%
YES - Tobacco use
1
   5.9%
0
   0.0%
16.Secondary Outcome
Title The Effect of Oral L-glutamine on the Number of Days Patient's Daily Activities Are Interrupted Due to Sickle Cell Pain
Hide Description Percentage of days a patient's daily activities were interrupted due to sickle pain calculated at each visit. Day's interrupted will be reported at Weeks 0, 8,16, 24, 32, 40 and 48
Time Frame Weeks 0, 8,16, 24, 32, 40 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: % of days interrupted
Week 0 Number Analyzed 33 participants 29 participants
8.3  (11.19) 8.1  (11.94)
Week 8 Number Analyzed 25 participants 22 participants
11.9  (21.22) 24.2  (29.22)
Week 16 Number Analyzed 28 participants 22 participants
23.3  (28.56) 15.5  (21.55)
Week 24 Number Analyzed 22 participants 19 participants
11.4  (18.34) 11.7  (12.75)
Week 32 Number Analyzed 19 participants 15 participants
12.5  (17.96) 15.7  (17.33)
Week 40 Number Analyzed 18 participants 13 participants
13.2  (16.88) 28.0  (31.33)
Week 48 Number Analyzed 17 participants 11 participants
11.7  (19.17) 11.5  (17.58)
17.Secondary Outcome
Title The Effect of Oral L-glutamine on Subjective Quality of Life
Hide Description The subjective quality of life was evaluated using the scoring of the RAND 36-Item Health Survey Questionnaire. The subjective quality of life (Physical functioning, Physical health, Emotional problems, Energy/Fatigue, Emotional well being, Social functioning, Pain, General health) will be reported at Baseline and Week 24 (or at time of discontinuation). The range for Physical functioning, Physical health, Emotional problems, Emotional well being and Social functioning is 0-100, with a high score denotes a better quality of life. For Energy/Fatigue, Pain and General health the range is 0-100, with a lower score denotes better quality of life.
Time Frame Baseline and Week 24 (or at time of discontinuation)
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: score on a scale
Physical Functioning Baseline Number Analyzed 31 participants 27 participants
61.7  (25.38) 69.9  (26.68)
Physical Functioning Week 24 Number Analyzed 28 participants 25 participants
65.5  (23.18) 76.0  (17.38)
Physical Health - Baseline Number Analyzed 31 participants 27 participants
43.5  (38.17) 49.1  (40.72)
Physical Health - Week 24 Number Analyzed 28 participants 25 participants
55.6  (41.79) 46.7  (40.83)
Emotional Problems - Baseline Number Analyzed 31 participants 27 participants
52.7  (41.97) 66.7  (41.35)
Emotional Problems - Week 24 Number Analyzed 28 participants 25 participants
58.3  (40.20) 65.3  (40.24)
Energy/Fatigue - Baseline Number Analyzed 31 participants 27 participants
45.7  (12.29) 50.0  (17.82)
Energy/Fatigue - Week 24 Number Analyzed 28 participants 25 participants
49.8  (19.65) 53.4  (21.96)
Emotional Well Being - Baseline Number Analyzed 31 participants 27 participants
66.2  (19.37) 74.5  (16.02)
Emotional Well Being - Week 24 Number Analyzed 28 participants 25 participants
71.9  (17.83) 77.1  (21.92)
Social Functioning - Baseline Number Analyzed 31 participants 27 participants
61.3  (24.01) 62.5  (24.27)
Social Functioning - Week 24 Number Analyzed 20 participants 16 participants
72.5  (22.06) 65.6  (26.02)
Pain - Baseline Number Analyzed 31 participants 27 participants
53.4  (23.41) 55.5  (28.07)
Pain - Week 24 Number Analyzed 28 participants 25 participants
59.1  (24.60) 56.1  (30.76)
General Health - Baseline Number Analyzed 31 participants 27 participants
46.0  (17.42) 49.7  (21.5)
General Health - Week 24 Number Analyzed 28 participants 25 participants
53.9  (16.55) 57.0  (18.62)
18.Secondary Outcome
Title Effect of Oral L--glutamine on Height
Hide Description Height will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
Time Frame Baseline, Weeks 4, 24, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: cm
Baseline Number Analyzed 33 participants 29 participants
169.8  (9.49) 169.2  (11.87)
Change in Height at Week 4 Number Analyzed 31 participants 24 participants
-0.1  (1.18) 0.2  (2.87)
Change in Height at Week 24 Number Analyzed 22 participants 19 participants
0.1  (1.54) -0.1  (1.68)
Change in Height at Week 48 Number Analyzed 17 participants 12 participants
-1.7  (6.58) 0.4  (3.35)
19.Secondary Outcome
Title Effect of Oral L--glutamine on Weight
Hide Description Weight will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
Time Frame Baseline, Weeks 4, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: kg
Baseline Number Analyzed 33 participants 29 participants
67.1  (13.39) 70.7  (18.20)
Change in Weight at Week 4 Number Analyzed 31 participants 24 participants
0.8  (1.89) 0.1  (3.13)
Change in Weight at Week 24 Number Analyzed 22 participants 19 participants
1.2  (2.55) 1.4  (5.04)
Change in Weight at Week 48 Number Analyzed 17 participants 12 participants
-1.0  (10.79) 1.9  (7.39)
20.Secondary Outcome
Title Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Walk Without Rest
Hide Description Minutes patient could walk without rest will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
Time Frame Baseline, Weeks 4, 24, and 48.
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: mins
Baseline - Minutes patient could walk without rest Number Analyzed 33 participants 29 participants
32  (28.25) 28.6  (23.94)
Change in Minutes patient could walk without rest at Week 4 Number Analyzed 32 participants 24 participants
-2.1  (29.57) -1.8  (20.06)
Change in Minutes patient could walk without rest at Week 24 Number Analyzed 22 participants 20 participants
-1.9  (26.30) -3.5  (12.63)
Change in Minutes patient could walk without rest at Week 48 Number Analyzed 17 participants 12 participants
-5.8  (24.88) 6.7  (28.95)
21.Secondary Outcome
Title Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Run Without Rest
Hide Description Minutes patient could run without rest will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
Time Frame Baseline, Weeks 4, 24, and 48.
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: mins
Baseline - Minutes patient could run without rest Number Analyzed 33 participants 29 participants
5.1  (8.90) 7.4  (10.02)
Change in Minutes patient could run without rest at Week 4 Number Analyzed 32 participants 24 participants
-0.7  (4.75) -0.7  (8.64)
Change in Minutes patient could run without rest at Week 24 Number Analyzed 22 participants 20 participants
-0.3  (5.17) -1.2  (13.56)
Change in Minutes patient could run without rest at Week 48 Number Analyzed 17 participants 12 participants
-0.8  (5.14) -1.2  (12.78)
22.Secondary Outcome
Title Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Walk Without Rest
Hide Description Distance patient could walk without rest will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
Time Frame Baseline, Weeks 4, 24, and 48.
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: feet
Baseline - Distance patient could walk without rest Number Analyzed 33 participants 29 participants
4097.5  (2739) 5680.7  (7695)
Change in Distance patient could walk without rest at Week 4 Number Analyzed 32 participants 24 participants
41.9  (1976) 302.5  (2022)
Change in Distance patient could walk without rest at Week 24 Number Analyzed 22 participants 20 participants
74.5  (2116) -1719  (9569)
Change in Distance patient could walk without rest at Week 48 Number Analyzed 17 participants 12 participants
38.8  (2766) -4240  (11782)
23.Secondary Outcome
Title Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Run Without Rest
Hide Description Distance patient could run without rest will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
Time Frame Baseline, Weeks 4, 24, and 48.
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis dataset included all patients who received at least one dose of study medication and had been diagnosed with sickle cell anemia or sickle β°-thalassemia documented by hemoglobin electrophoresis and had at least 2 episodes of painful crises within 12 months prior to the screening visit (N = 62)
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description:

L-glutamine

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.

maltodextrin

Placebo: 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.
Overall Number of Participants Analyzed 33 29
Mean (Standard Deviation)
Unit of Measure: feet
Baseline - Distance patient could run without rest Number Analyzed 33 participants 29 participants
1059.5  (1688) 1482.1  (2160)
Change in Distance patient could run without rest at Week 4 Number Analyzed 32 participants 24 participants
-31.6  (1054) 124.2  (1226)
Change in Distance patient could run without rest at Week 24 Number Analyzed 22 participants 20 participants
215.9  (1411) 186.5  (2901)
Change in Distance patient could run without rest at Week 48 Number Analyzed 17 participants 12 participants
13.5  (1677) -586.7  (2698)
Time Frame Adverse events data were collected throughout the course of the study (53 weeks or about 1 year).
Adverse Event Reporting Description The Safety population will include all patients who received at least one dose of study medication.
 
Arm/Group Title Investigational Product Placebo
Hide Arm/Group Description

L-glutamine

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.

maltodextrin

Placebo: 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.
All-Cause Mortality
Investigational Product Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   1/37 (2.70%)      0/33 (0.00%)    
Hide Serious Adverse Events
Investigational Product Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   24/37 (64.86%)      21/33 (63.64%)    
Cardiac disorders     
Palpitations † 1  0/37 (0.00%)  0 1/33 (3.03%)  1
Congenital, familial and genetic disorders     
Sickle cell anaemia with crisis † 1  22/37 (59.46%)  22 17/33 (51.52%)  17
General disorders     
pyrexia † 1  0/37 (0.00%)  0 1/33 (3.03%)  1
Infections and infestations     
Pneumonia † 1  3/37 (8.11%)  3 1/33 (3.03%)  1
URTI † 1  0/37 (0.00%)  0 1/33 (3.03%)  1
Investigations     
Blood potassium increased † 1  1/37 (2.70%)  1 0/33 (0.00%)  0
Metabolism and nutrition disorders     
Hypoglycaemia † 1  1/37 (2.70%)  1 0/33 (0.00%)  0
Pregnancy, puerperium and perinatal conditions     
Pregnancy † 1  2/37 (5.41%)  2 1/33 (3.03%)  1
Renal and urinary disorders     
Renal failure acute † 1  1/37 (2.70%)  1 0/33 (0.00%)  0
Reproductive system and breast disorders     
Priapism † 1  0/37 (0.00%)  0 1/33 (3.03%)  1
Respiratory, thoracic and mediastinal disorders     
Asthma † 1  0/37 (0.00%)  0 2/33 (6.06%)  2
Surgical and medical procedures     
Hip Arthroplasty † 1  0/37 (0.00%)  0 1/33 (3.03%)  1
1
Term from vocabulary, MedDRA (7.1)
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Investigational Product Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   37/37 (100.00%)      33/33 (100.00%)    
Congenital, familial and genetic disorders     
Sickle cell anemia with crisis † 1  31/37 (83.78%)  31 26/33 (78.79%)  26
Gastrointestinal disorders     
Diarrhea † 1  3/37 (8.11%)  3 1/33 (3.03%)  1
Abdominal pain upper † 1  2/37 (5.41%)  2 2/33 (6.06%)  2
Constipation † 1  2/37 (5.41%)  2 1/33 (3.03%)  1
Toothache † 1  2/37 (5.41%)  2 0/33 (0.00%)  0
General disorders     
Chest pain † 1  2/37 (5.41%)  2 0/33 (0.00%)  0
Influenza-like illness † 1  0/37 (0.00%)  0 2/33 (6.06%)  2
Infections and infestations     
Pneumonia † 1  4/37 (10.81%)  4 1/33 (3.03%)  1
Nasopharyngitis † 1  3/37 (8.11%)  3 3/33 (9.09%)  3
Gastroenteritis † 1  2/37 (5.41%)  2 5/33 (15.15%)  5
URTI † 1  1/37 (2.70%)  1 3/33 (9.09%)  3
Ear infection † 1  0/37 (0.00%)  0 2/33 (6.06%)  2
Gastroentritis viral † 1  0/37 (0.00%)  0 2/33 (6.06%)  2
Musculoskeletal and connective tissue disorders     
Arthralgia † 1  2/37 (5.41%)  2 7/33 (21.21%)  7
Back pain † 1  2/37 (5.41%)  2 2/33 (6.06%)  2
Nervous system disorders     
Headache † 1  3/37 (8.11%)  3 3/33 (9.09%)  3
Convulsion † 1  2/37 (5.41%)  2 0/33 (0.00%)  0
Pregnancy, puerperium and perinatal conditions     
Pregnancy † 1  2/37 (5.41%)  2 1/33 (3.03%)  1
Respiratory, thoracic and mediastinal disorders     
Cough † 1  2/37 (5.41%)  2 0/33 (0.00%)  0
Asthma † 1  0/37 (0.00%)  0 2/33 (6.06%)  2
1
Term from vocabulary, MedDRA (7.1)
†
Indicates events were collected by systematic assessment
[Not Specified]
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.
PI agrees that any INFORMATION submitted to it by EMMAUS shall be maintained in secrecy for a period of seven (7) years from each disclosure of INFORMATION. PI will use the up most due diligence to prevent disclosure by it except to its employees, agents, and contractors necessary for evaluation, all of whom shall be bound by similar written obligations of confidentiality, and who agree not to use the INFORMATION for any purpose other than for evaluation purposes.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Yutaka Niihara, MD, MPH
Organization: Emmaus Medical, Inc.
Phone: 310-214-0065
EMail: yniihara@emmausmedical.com
Layout table for additonal information
Responsible Party: Emmaus Medical, Inc.
ClinicalTrials.gov Identifier: NCT00125788    
Obsolete Identifiers: NCT00029887
Other Study ID Numbers: 10478
First Submitted: August 1, 2005
First Posted: August 2, 2005
Results First Submitted: August 27, 2020
Results First Posted: January 29, 2021
Last Update Posted: January 29, 2021