L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
Sponsor
Emmaus Medical, Inc. (Industry)
Overall Status
Completed
CT.gov ID
NCT00125788
Collaborator
FDA Office of Orphan Products Development (U.S. Fed)
70
5
2
52
14
0.3
Study Details
Study Description
Brief Summary
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia. as evaluated by the number of occurrences of sickle cell crises.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Detailed Description
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 frequency of hospitalizations for sickle cell pain, frequency of emergency room visits for sickle cell pain; energy and appetite levels; narcotics usage.
Methodology:
By site, patients will be randomized to L-glutamine or placebo in a 1:1 ratio after a 4-week screening period. Patients will undergo 48 weeks of treatment with dosing BID orally, with dose calculated according to patient weight. Patient visits will occur every 4 weeks. After 48 weeks of treatment, dose will be tapered to zero within 3 weeks. A final evaluation visit will occur 2 weeks after last dose.
Study Design
Study Type:
Interventional
Actual Enrollment
:
70 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, 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
Study Start Date
:
Mar 1, 2004
Actual Primary Completion Date
:
Jul 1, 2008
Actual Study Completion Date
:
Jul 1, 2008
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| 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.
Other Names:
|
| Placebo Comparator: placebo maltodextrin |
Drug: 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.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Number of Occurrences of Painful Sickle Cell Crises [From Week 0 through Week 48 (cumulative)]
The mean number of painful sickle crisis through week 48
Secondary Outcome Measures
- Frequency of Hospitalizations for Sickle Cell Pain [From Week 0 through Week 48 (cumulative)]
The mean number of hospitalizations through week 48
- Frequency of Emergency Room Visits for Sickle Cell Pain [From Week 0 through Week 48 (cumulative)]
The mean number of emergency room visits through week 48
- The Effect of Oral L-glutamine on Hematological Parameters - Hemoglobin [Baseline, Weeks 4, 24 and 40]
Patient's hemoglobin will be collected at each visit.Change from Baseline will be reported at Weeks 4, 24 and 40.
- The Effect of Oral L-glutamine on Hematological Parameters - Hematocrit [Baseline, Weeks 4, 24, and 40]
Patient's hematocrit will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24 and 40
- The Effect of Oral L-glutamine on Hematological Parameters - Reticulocyte Count [Baseline, Weeks 0, 4, 24, 40]
Patient's reticulocyte count will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24 and 40
- Number of Participants With Narcotic Usage [Week 24, Week 48]
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.
- Energy Level (11-point Scale) [Collected at Week 0, 8, 16, 24, 32, 40, 48]
The patient's energy level was evaluated at each visit using an 11 point scale from 0=extremely tired to 10=extremely energetic
- Patient Appetite (3-point Scale) [Collected at Week 0, 8, 16, 24, 32, 40, 48]
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.
- The Effect of Oral L-glutamine on Vital Signs - Blood Pressure [Baseline, Weeks 4, 24, and 48]
Patient's blood pressure will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
- The Effect of Oral L-glutamine on Vital Signs - Temperature [Baseline, Weeks 4, 24, and 48]
Patient's temperature will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
- The Effect of Oral L-glutamine on Vital Signs - Respiration [Baseline, Weeks 4, 24, and 48]
Respiration will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
- The Effect of Oral L-glutamine on Vital Signs - Pulse Rate [Baseline, Weeks 4, 24, and 48]
Patient's pulse rate will be collected at each visit, Change from Baseline will be reported at Weeks 4, 24, and 48
- Effect of L-glutamine on Alcohol Use [Weeks 0, 8,16, 24, 32, 40 and 48]
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
- Effect of L-glutamine on Tobacco Use [Weeks 0, 8,16, 24, 32, 40 and 48]
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
- The Effect of Oral L-glutamine on the Number of Days Patient's Daily Activities Are Interrupted Due to Sickle Cell Pain [Weeks 0, 8,16, 24, 32, 40 and 48]
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
- The Effect of Oral L-glutamine on Subjective Quality of Life [Baseline and Week 24 (or at time of discontinuation)]
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.
- Effect of Oral L--glutamine on Height [Baseline, Weeks 4, 24, and 48]
Height will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
- Effect of Oral L--glutamine on Weight [Baseline, Weeks 4, 24 and 48]
Weight will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48
- Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Walk Without Rest [Baseline, Weeks 4, 24, and 48.]
Minutes patient could walk without rest will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
- Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Run Without Rest [Baseline, Weeks 4, 24, and 48.]
Minutes patient could run without rest will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
- Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Walk Without Rest [Baseline, Weeks 4, 24, and 48.]
Distance patient could walk without rest will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
- Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Run Without Rest [Baseline, Weeks 4, 24, and 48.]
Distance patient could run without rest will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.
Eligibility Criteria
Criteria
Ages Eligible for Study:
5 Years
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Kaiser Permanente | Bellflower | California | United States | 90706 |
| 2 | Harbor-UCLA Medical Center | Torrance | California | United States | 90502 |
| 3 | Grady Memorial Hospital | Atlanta | Georgia | United States | 30303 |
| 4 | University of Medicine and Dentistry, New Jersey | New Brunswick | New Jersey | United States | 08903 |
| 5 | Jacobi Medical Center | Bronx | New York | United States | 10461 |
Sponsors and Collaborators
- Emmaus Medical, Inc.
- FDA Office of Orphan Products Development
Investigators
- Principal Investigator: Yutaka Niihara, MD, CEO, Emmaus Medical, Inc
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.Responsible Party:
Emmaus Medical, Inc.
ClinicalTrials.gov Identifier:
NCT00125788
Other Study ID Numbers:
- 10478
- NCT00029887
First Posted:
Aug 2, 2005
Last Update Posted:
Jan 29, 2021
Last Verified:
Dec 1, 2020
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Keywords provided by Emmaus Medical, Inc.
Additional relevant MeSH terms:
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Investigational Product | Placebo |
|---|---|---|
| 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 |
Baseline Characteristics
| Arm/Group Title | Investigational Product | Placebo | Total |
|---|---|---|---|
| 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 Participants | 33 | 29 | 62 |
| Age (Count of 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%
|
| Age (years) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [years] |
30.5
(10.09)
|
26.5
(9.42)
|
28.6
(13.8)
|
| Sex: Female, Male (Count of Participants) | |||
| Female |
22
66.7%
|
10
34.5%
|
32
51.6%
|
| Male |
11
33.3%
|
19
65.5%
|
30
48.4%
|
| Region of Enrollment (participants) [Number] | |||
| United States |
33
100%
|
29
100%
|
62
100%
|
Outcome Measures
| Title | Number of Occurrences of Painful Sickle Cell Crises |
|---|---|
| Description | The mean number of painful sickle crisis through week 48 |
| Time Frame | From Week 0 through Week 48 (cumulative) |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Mean (Standard Deviation) [Crisis] |
4.5
(5.37)
|
10.8
(18.74)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Investigational Product, Placebo |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.076 |
| Comments | ||
| Method | Cochran-Mantel-Haenszel | |
| Comments | ||
| Title | Frequency of Hospitalizations for Sickle Cell Pain |
|---|---|
| Description | The mean number of hospitalizations through week 48 |
| Time Frame | From Week 0 through Week 48 (cumulative) |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Mean (Standard Deviation) [Hosptalizations] |
1.5
(2.46)
|
2.3
(2.42)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Investigational Product, Placebo |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.072 |
| Comments | ||
| Method | Cochran-Mantel-Haenszel | |
| Comments | ||
| Title | Frequency of Emergency Room Visits for Sickle Cell Pain |
|---|---|
| Description | The mean number of emergency room visits through week 48 |
| Time Frame | From Week 0 through Week 48 (cumulative) |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Mean (Standard Deviation) [Emergency room visits] |
3.7
(5.63)
|
9.4
(19.91)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Investigational Product, Placebo |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.129 |
| Comments | ||
| Method | Cochran-Mantel-Haenszel | |
| Comments | ||
| Title | The Effect of Oral L-glutamine on Hematological Parameters - Hemoglobin |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 37 | 33 |
| Baseline |
8.96
(1.477)
|
9.05
(1.219)
|
| Change in Hemoglobin at Week 4 |
0.15
(0.831)
|
-0.13
(0.861)
|
| Change in Hemoglobin at Week 24 |
0.26
(0.641)
|
0.16
(0.607)
|
| Change in Hemoglobin at Week 40 |
0.11
(0.749)
|
-0.07
(0.650)
|
| Title | The Effect of Oral L-glutamine on Hematological Parameters - Hematocrit |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 37 | 33 |
| Baseline |
25.90
(4.493)
|
26.22
(4.125)
|
| Change in Hematocrit at Week 4 |
0.51
(2.784)
|
-0.09
(2.279)
|
| Change in Hematocrit at Week 24 |
1.08
(2.352)
|
0.58
(2.050)
|
| Change in Hematocrit at Week 40 |
0.49
(2.786)
|
0.26
(1.869)
|
| Title | The Effect of Oral L-glutamine on Hematological Parameters - Reticulocyte Count |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 37 | 33 |
| Baseline |
0.266
(0.1125)
|
0.295
(0.0979)
|
| Change in Reticulocyte count at Week 4 |
-0.040
(0.0993)
|
-0.034
(0.0689)
|
| Change in Reticulocyte count at Week 24 |
-0.022
(0.0917)
|
-0.032
(0.1032)
|
| Change in Reticulocyte count at Week 40 |
-0.016
(0.1357)
|
-0.020
(0.0908)
|
| Title | Number of Participants With Narcotic Usage |
|---|---|
| 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Patients with any narcotic use who completed the visit |
| Arm/Group Title | Investigational Product | Placebo |
|---|---|---|
| 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. |
| Measure Participants | 17 | 16 |
| Change to weaker narcotics |
6
18.2%
|
2
6.9%
|
| No change |
10
30.3%
|
9
31%
|
| Change to stronger narcotics |
1
3%
|
5
17.2%
|
| Change to weaker narcotics |
6
18.2%
|
2
6.9%
|
| No change |
10
30.3%
|
9
31%
|
| Change to stronger narcotics |
1
3%
|
5
17.2%
|
| Title | Energy Level (11-point Scale) |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Week 0 |
6.1
(1.92)
|
6.8
(1.99)
|
| Week 8 |
6.9
(1.84)
|
7.1
(1.96)
|
| Week 16 |
6.9
(2.02)
|
7.1
(2.70)
|
| Week 24 |
7.3
(1.65)
|
7.8
(2.02)
|
| Week 32 |
7.3
(1.67)
|
7.4
(1.95)
|
| Week40 |
6.6
(1.54)
|
6.9
(2.29)
|
| Week 48 |
6.8
(1.92)
|
7.3
(1.83)
|
| Title | Patient Appetite (3-point Scale) |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| 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%
|
| Above average |
4
12.1%
|
6
20.7%
|
| Average |
19
57.6%
|
14
48.3%
|
| Below average |
2
6.1%
|
2
6.9%
|
| Not known |
1
3%
|
0
0%
|
| Above average |
3
9.1%
|
9
31%
|
| Average |
18
54.5%
|
11
37.9%
|
| Below average |
7
21.2%
|
2
6.9%
|
| Not known |
0
0%
|
0
0%
|
| Above average |
4
12.1%
|
7
24.1%
|
| Average |
17
51.5%
|
11
37.9%
|
| Below average |
1
3%
|
2
6.9%
|
| Not known |
0
0%
|
0
0%
|
| Above average |
3
9.1%
|
3
10.3%
|
| Average |
15
45.5%
|
12
41.4%
|
| Below average |
1
3%
|
0
0%
|
| Not known |
0
0%
|
0
0%
|
| Above average |
2
6.1%
|
4
13.8%
|
| Average |
13
39.4%
|
7
24.1%
|
| Below average |
3
9.1%
|
2
6.9%
|
| Not known |
0
0%
|
0
0%
|
| Above average |
2
6.1%
|
2
6.9%
|
| Average |
13
39.4%
|
9
31%
|
| Below average |
2
6.1%
|
1
3.4%
|
| Not known |
0
0%
|
0
0%
|
| Title | The Effect of Oral L-glutamine on Vital Signs - Blood Pressure |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 37 | 33 |
| Baseline SBP (mm/Hg) |
115.18
(14.30)
|
119.6
(13.64)
|
| Change in SBP (mm/Hg) at Week 4 |
0.7
(10.98)
|
-0.9
(10.81)
|
| Change in SBP (mm/Hg) at Week 24 |
2.2
(10.48)
|
-1.9
(13.06)
|
| Change in SBP (mm/Hg) at Week 48 |
-1.2
(12.07)
|
-1.5
(12.97)
|
| Baseline DBP (mm/hg) |
63.7
(9.05)
|
64.9
(8.09)
|
| Change in DBP (mm/Hg) at Week 4 |
1.2
(8.09)
|
2.2
(8.73)
|
| Change in DBP (mm/Hg) at Week 24 |
1.2
(9.55)
|
-0.4
(6.88)
|
| Change in DBP (mm/Hg) at Week 48 |
-1.3
(7.98)
|
0.5
(7.91)
|
| Title | The Effect of Oral L-glutamine on Vital Signs - Temperature |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 37 | 33 |
| Baseline |
36.40
(0.686)
|
36.29
(0.613)
|
| Change in Temperature at Week 4 |
-0.05
(0.377)
|
-0.06
(0.269)
|
| Change Temperature at Week 24 |
-0.01
(0.261)
|
-0.07
(0.268)
|
| Change in Temperature at Week 48 |
0.08
(0.362)
|
-0.09
(0.256)
|
| Title | The Effect of Oral L-glutamine on Vital Signs - Respiration |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 37 | 33 |
| Baseline |
17.9
(2.11)
|
17.7
(1.94)
|
| Change in Respiration at Week 4 |
0.3
(1.71)
|
0.6
(1.58)
|
| Change in Respiration at Week 24 |
0.4
(1.73)
|
-0.4
(1.93)
|
| Change in Respiration at Week 48 |
0.4
(2.12)
|
0.6
(2.47)
|
| Title | The Effect of Oral L-glutamine on Vital Signs - Pulse Rate |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 37 | 33 |
| Baseline |
81.6
(11.37)
|
80.5
(11.95)
|
| Change in Pulse Rate at Week 4 |
2.5
(12.88)
|
0.7
(12.11)
|
| Change in Pulse Rate at Week 24 |
1.7
(13.69)
|
-2.7
(12.81)
|
| Change in Pulse Rate at Week 48 |
1.3
(14.53)
|
3.9
(10.07)
|
| Title | Effect of L-glutamine on Alcohol Use |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| NO - Alcohol Use |
30
90.9%
|
27
93.1%
|
| YES - Alcohol Use |
3
9.1%
|
2
6.9%
|
| NO - Alcohol Use |
25
75.8%
|
20
69%
|
| YES - Alcohol Use |
1
3%
|
2
6.9%
|
| NO - Alcohol Use |
27
81.8%
|
20
69%
|
| YES - Alcohol Use |
1
3%
|
2
6.9%
|
| NO - Alcohol Use |
22
66.7%
|
19
65.5%
|
| YES - Alcohol Use |
0
0%
|
1
3.4%
|
| NO - Alcohol Use |
19
57.6%
|
13
44.8%
|
| YES - Alcohol Use |
0
0%
|
2
6.9%
|
| NO - Alcohol Use |
18
54.5%
|
11
37.9%
|
| YES - Alcohol Use |
0
0%
|
2
6.9%
|
| NO - Alcohol Use |
17
51.5%
|
11
37.9%
|
| YES - Alcohol Use |
0
0%
|
1
3.4%
|
| Title | Effect of L-glutamine on Tobacco Use |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| NO - Tobacco use |
28
84.8%
|
27
93.1%
|
| YES - Tobacco use |
5
15.2%
|
2
6.9%
|
| NO - Tobacco use |
23
69.7%
|
21
72.4%
|
| YES - Tobacco use |
3
9.1%
|
1
3.4%
|
| NO - Tobacco use |
24
72.7%
|
22
75.9%
|
| YES - Tobacco use |
4
12.1%
|
0
0%
|
| NO - Tobacco use |
20
60.6%
|
19
65.5%
|
| YES - Tobacco use |
2
6.1%
|
1
3.4%
|
| NO - Tobacco use |
18
54.5%
|
15
51.7%
|
| YES - Tobacco use |
1
3%
|
0
0%
|
| NO - Tobacco use |
17
51.5%
|
13
44.8%
|
| YES - Tobacco use |
1
3%
|
0
0%
|
| NO - Tobacco use |
16
48.5%
|
12
41.4%
|
| YES - Tobacco use |
1
3%
|
0
0%
|
| Title | The Effect of Oral L-glutamine on the Number of Days Patient's Daily Activities Are Interrupted Due to Sickle Cell Pain |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Week 0 |
8.3
(11.19)
|
8.1
(11.94)
|
| Week 8 |
11.9
(21.22)
|
24.2
(29.22)
|
| Week 16 |
23.3
(28.56)
|
15.5
(21.55)
|
| Week 24 |
11.4
(18.34)
|
11.7
(12.75)
|
| Week 32 |
12.5
(17.96)
|
15.7
(17.33)
|
| Week 40 |
13.2
(16.88)
|
28.0
(31.33)
|
| Week 48 |
11.7
(19.17)
|
11.5
(17.58)
|
| Title | The Effect of Oral L-glutamine on Subjective Quality of Life |
|---|---|
| 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) |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Physical Functioning Baseline |
61.7
(25.38)
|
69.9
(26.68)
|
| Physical Functioning Week 24 |
65.5
(23.18)
|
76.0
(17.38)
|
| Physical Health - Baseline |
43.5
(38.17)
|
49.1
(40.72)
|
| Physical Health - Week 24 |
55.6
(41.79)
|
46.7
(40.83)
|
| Emotional Problems - Baseline |
52.7
(41.97)
|
66.7
(41.35)
|
| Emotional Problems - Week 24 |
58.3
(40.20)
|
65.3
(40.24)
|
| Energy/Fatigue - Baseline |
45.7
(12.29)
|
50.0
(17.82)
|
| Energy/Fatigue - Week 24 |
49.8
(19.65)
|
53.4
(21.96)
|
| Emotional Well Being - Baseline |
66.2
(19.37)
|
74.5
(16.02)
|
| Emotional Well Being - Week 24 |
71.9
(17.83)
|
77.1
(21.92)
|
| Social Functioning - Baseline |
61.3
(24.01)
|
62.5
(24.27)
|
| Social Functioning - Week 24 |
72.5
(22.06)
|
65.6
(26.02)
|
| Pain - Baseline |
53.4
(23.41)
|
55.5
(28.07)
|
| Pain - Week 24 |
59.1
(24.60)
|
56.1
(30.76)
|
| General Health - Baseline |
46.0
(17.42)
|
49.7
(21.5)
|
| General Health - Week 24 |
53.9
(16.55)
|
57.0
(18.62)
|
| Title | Effect of Oral L--glutamine on Height |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Baseline |
169.8
(9.49)
|
169.2
(11.87)
|
| Change in Height at Week 4 |
-0.1
(1.18)
|
0.2
(2.87)
|
| Change in Height at Week 24 |
0.1
(1.54)
|
-0.1
(1.68)
|
| Change in Height at Week 48 |
-1.7
(6.58)
|
0.4
(3.35)
|
| Title | Effect of Oral L--glutamine on Weight |
|---|---|
| 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 |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Baseline |
67.1
(13.39)
|
70.7
(18.20)
|
| Change in Weight at Week 4 |
0.8
(1.89)
|
0.1
(3.13)
|
| Change in Weight at Week 24 |
1.2
(2.55)
|
1.4
(5.04)
|
| Change in Weight at Week 48 |
-1.0
(10.79)
|
1.9
(7.39)
|
| Title | Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Walk Without Rest |
|---|---|
| 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. |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Baseline - Minutes patient could walk without rest |
32
(28.25)
|
28.6
(23.94)
|
| Change in Minutes patient could walk without rest at Week 4 |
-2.1
(29.57)
|
-1.8
(20.06)
|
| Change in Minutes patient could walk without rest at Week 24 |
-1.9
(26.30)
|
-3.5
(12.63)
|
| Change in Minutes patient could walk without rest at Week 48 |
-5.8
(24.88)
|
6.7
(28.95)
|
| Title | Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Run Without Rest |
|---|---|
| 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. |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Baseline - Minutes patient could run without rest |
5.1
(8.90)
|
7.4
(10.02)
|
| Change in Minutes patient could run without rest at Week 4 |
-0.7
(4.75)
|
-0.7
(8.64)
|
| Change in Minutes patient could run without rest at Week 24 |
-0.3
(5.17)
|
-1.2
(13.56)
|
| Change in Minutes patient could run without rest at Week 48 |
-0.8
(5.14)
|
-1.2
(12.78)
|
| Title | Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Walk Without Rest |
|---|---|
| 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. |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Baseline - Distance patient could walk without rest |
4097.5
(2739)
|
5680.7
(7695)
|
| Change in Distance patient could walk without rest at Week 4 |
41.9
(1976)
|
302.5
(2022)
|
| Change in Distance patient could walk without rest at Week 24 |
74.5
(2116)
|
-1719
(9569)
|
| Change in Distance patient could walk without rest at Week 48 |
38.8
(2766)
|
-4240
(11782)
|
| Title | Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Run Without Rest |
|---|---|
| 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. |
Outcome Measure Data
| 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 |
|---|---|---|
| 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. |
| Measure Participants | 33 | 29 |
| Baseline - Distance patient could run without rest |
1059.5
(1688)
|
1482.1
(2160)
|
| Change in Distance patient could run without rest at Week 4 |
-31.6
(1054)
|
124.2
(1226)
|
| Change in Distance patient could run without rest at Week 24 |
215.9
(1411)
|
186.5
(2901)
|
| Change in Distance patient could run without rest at Week 48 |
13.5
(1677)
|
-586.7
(2698)
|
Adverse Events
| 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 | ||
| 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 (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 1/37 (2.7%) | 0/33 (0%) | ||
Serious Adverse Events |
||||
| Investigational Product | Placebo | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 24/37 (64.9%) | 21/33 (63.6%) | ||
| Cardiac disorders | ||||
| Palpitations | 0/37 (0%) | 0 | 1/33 (3%) | 1 |
| Congenital, familial and genetic disorders | ||||
| Sickle cell anaemia with crisis | 22/37 (59.5%) | 22 | 17/33 (51.5%) | 17 |
| General disorders | ||||
| pyrexia | 0/37 (0%) | 0 | 1/33 (3%) | 1 |
| Infections and infestations | ||||
| Pneumonia | 3/37 (8.1%) | 3 | 1/33 (3%) | 1 |
| URTI | 0/37 (0%) | 0 | 1/33 (3%) | 1 |
| Investigations | ||||
| Blood potassium increased | 1/37 (2.7%) | 1 | 0/33 (0%) | 0 |
| Metabolism and nutrition disorders | ||||
| Hypoglycaemia | 1/37 (2.7%) | 1 | 0/33 (0%) | 0 |
| Pregnancy, puerperium and perinatal conditions | ||||
| Pregnancy | 2/37 (5.4%) | 2 | 1/33 (3%) | 1 |
| Renal and urinary disorders | ||||
| Renal failure acute | 1/37 (2.7%) | 1 | 0/33 (0%) | 0 |
| Reproductive system and breast disorders | ||||
| Priapism | 0/37 (0%) | 0 | 1/33 (3%) | 1 |
| Respiratory, thoracic and mediastinal disorders | ||||
| Asthma | 0/37 (0%) | 0 | 2/33 (6.1%) | 2 |
| Surgical and medical procedures | ||||
| Hip Arthroplasty | 0/37 (0%) | 0 | 1/33 (3%) | 1 |
Other (Not Including Serious) Adverse Events |
||||
| Investigational Product | Placebo | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 37/37 (100%) | 33/33 (100%) | ||
| Congenital, familial and genetic disorders | ||||
| Sickle cell anemia with crisis | 31/37 (83.8%) | 31 | 26/33 (78.8%) | 26 |
| Gastrointestinal disorders | ||||
| Diarrhea | 3/37 (8.1%) | 3 | 1/33 (3%) | 1 |
| Abdominal pain upper | 2/37 (5.4%) | 2 | 2/33 (6.1%) | 2 |
| Constipation | 2/37 (5.4%) | 2 | 1/33 (3%) | 1 |
| Toothache | 2/37 (5.4%) | 2 | 0/33 (0%) | 0 |
| General disorders | ||||
| Chest pain | 2/37 (5.4%) | 2 | 0/33 (0%) | 0 |
| Influenza-like illness | 0/37 (0%) | 0 | 2/33 (6.1%) | 2 |
| Infections and infestations | ||||
| Pneumonia | 4/37 (10.8%) | 4 | 1/33 (3%) | 1 |
| Nasopharyngitis | 3/37 (8.1%) | 3 | 3/33 (9.1%) | 3 |
| Gastroenteritis | 2/37 (5.4%) | 2 | 5/33 (15.2%) | 5 |
| URTI | 1/37 (2.7%) | 1 | 3/33 (9.1%) | 3 |
| Ear infection | 0/37 (0%) | 0 | 2/33 (6.1%) | 2 |
| Gastroentritis viral | 0/37 (0%) | 0 | 2/33 (6.1%) | 2 |
| Musculoskeletal and connective tissue disorders | ||||
| Arthralgia | 2/37 (5.4%) | 2 | 7/33 (21.2%) | 7 |
| Back pain | 2/37 (5.4%) | 2 | 2/33 (6.1%) | 2 |
| Nervous system disorders | ||||
| Headache | 3/37 (8.1%) | 3 | 3/33 (9.1%) | 3 |
| Convulsion | 2/37 (5.4%) | 2 | 0/33 (0%) | 0 |
| Pregnancy, puerperium and perinatal conditions | ||||
| Pregnancy | 2/37 (5.4%) | 2 | 1/33 (3%) | 1 |
| Respiratory, thoracic and mediastinal disorders | ||||
| Cough | 2/37 (5.4%) | 2 | 0/33 (0%) | 0 |
| Asthma | 0/37 (0%) | 0 | 2/33 (6.1%) | 2 |
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
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
| Name/Title | Yutaka Niihara, MD, MPH |
|---|---|
| Organization | Emmaus Medical, Inc. |
| Phone | 310-214-0065 |
| yniihara@emmausmedical.com |
Responsible Party:
Emmaus Medical, Inc.
ClinicalTrials.gov Identifier:
NCT00125788
Other Study ID Numbers:
- 10478
- NCT00029887
First Posted:
Aug 2, 2005
Last Update Posted:
Jan 29, 2021
Last Verified:
Dec 1, 2020