Trial of Oral Glutamine in Patients With Sickle Cell Anemia

• Sponsor: St. Jude Children's Research Hospital (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00131508
• Collaborator: Thrasher Research Fund (Other)
• Enrollment: 27
• Location: 1
• Arms: 2
• Duration (Months): 59
• Patients Per Site Per Month: 0.5

Study Details

Study Description

Brief Summary

Children with sickle cell anemia (SCA) seem to have higher energy needs than children who do not have the disease. This may be the reason why children and teenagers with sickle cell anemia tend to be smaller, weigh less, and have less fat and muscle than children and teens that do not have the disease.

This study is being done to find out if giving a supplement called glutamine will help children with sickle cell anemia by lowering their energy needs and improving their growth and strength. Children will be randomly assigned (like a flip of a coin) to one of two groups. One group will take glutamine and one group will take a placebo (a protein mixture that looks like glutamine but may not have the same effect in the body). No one will know which group is taking which supplement until the study has been completed. Children will be in the study for 12 months.

Condition or Disease	Intervention/Treatment	Phase
Anemia, Sickle Cell	Drug: Glutamine; Drug: Placebo	Phase 2

Detailed Description

1. The study will compare the effect of glutamine and placebo on resting energy expenditure (REE) in children with sickle cell anemia (SCA) by comparing the change in REE ratio between baseline and 12 months.

2. The study investigates the effect of oral glutamine and placebo on body composition in children with SCA by comparing the difference in body mass indexes (BMIs) and percent of body fat (DEXA Scan) between baseline and 12 months of treatment in the two groups.

3. This Study will investigate the effect of oral glutamine and placebo on growth in children with SCA by comparing the Z scores for one year before baseline to 1 year while on study.

4. This study will investigate the effect of oral glutamine and placebo in children with SCA by comparing the difference in the levels of plasma and red blood cell glutamine between baseline and 12 months of treatment in the two groups.

5. This study will investigate the clinical effects (strength and exercise endurance) of oral glutamine and placebo in children with SCA by comparing the difference between baseline and 12 months of treatment in the two groups.

6. This study will evaluate quality of life in children with SCA who have glutamine or placebo for 12 months.

7. This study will evaluate the changes in REE over time in a small group of patients that will have REE measurement at months 3, 6, and 9. This objective will be offered to all patients, but will be "additional studies" that are not required to participate in the protocol.

Study Design

• Study Type: Interventional
• Actual Enrollment: 27 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Care Provider, Investigator)
• Primary Purpose: Prevention
• Official Title: A Randomized Controlled Trial of Oral Glutamine Supplementation Versus a Placebo Supplement in Children With Sickle Cell Anemia
• Study Start Date: May 1, 2004
• Actual Primary Completion Date: Apr 1, 2009
• Actual Study Completion Date: Apr 1, 2009

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 2 Glutamine	Drug: Glutamine; 0.6 gm/kg of oral glutamine per day, in two doses for one year.
Placebo Comparator: 1	Drug: Placebo; Placebo

Outcome Measures

Primary Outcome Measures

1. Change in Resting Energy Expenditure From Baseline to 12 Months [Baseline and 12 months]

   To compare the effect of glutamine and placebo on resting energy expenditure (REE) in children with sickle cell anemia (SCA) by comparing the change in REE ratio between baseline and 12 months. REE was measured by indirect calorimetry, using a metabolic cart.REE Ratio =(REE Measured/REE Predicted)x 100).Change was defined as 12 Month REE Ratio minus Baseline REE Ratio.The REE Ratio was evaluated at baseline and 12 months.The REE Ratio is calculated as (REE Measured / REE Predicted) x 100).REE units are measured as (Kcal / day).Change was defined as 12 Month REE Ratio minus Baseline REE Ratio.

Secondary Outcome Measures

1. Change in Body Mass Index From Baseline to 12 Months [Baseline and 12 months]

   To investigate the effect of oral glutamine and placebo on body composition in children with SCA by comparing the difference in body mass indexes (BMI) between baseline and 12 months of treatment in the two groups.

2. Change in Red Blood Cell Glutamine From Baseline to 12 Months [Baseline and 12 months]

   To investigate the effect of oral glutamine and placebo in children with Sickle Cell Anemia (SCA) by comparing the difference in the levels of red blood cell glutamine between baseline and 12 months of treatment in the two groups.

3. Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. [Baseline and 12 Months]

   Evaluation of quality of life at baseline and 12 months in the glutamine versus placebo group using the PedsQL Version 4.0 inventory. This instrument measures individual well being across physical, emotional, social, and school function categories using patient self-reports and/or parent reports. The tool contains a 15-question, age-specific, self-report inventory (for children age 5-7 years, 8-12 years, and 13-18 years) and a corresponding parent inventory. Lower scores indicate a better quality of life.

4. Change in Height Z-score From Baseline to 12 Months [Baseline and 12 months]

   To investigate the effect of oral glutamine and placebo on height Z-score in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment.

5. Change in Height Percentile From Baseline to 12 Months [Baseline and 12 months]

   To investigate the effect of oral glutamine and placebo on height percentile in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment.

6. Change in Weight Percentile From Baseline to 12 Months [Basline and 12 months]

   To investigate the effect of oral glutamine and placebo on weight in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment.

7. Change in Pulse Rate From Baseline to 12 Months [Baseline and 12 months]

   To investigate the clinical effects of oral glutamine and placebo on pulse rate in children with Sickle Cell Anemia (SCA) by comparing the difference between baseline and 12 months of treatment between the two groups.

8. Change in Hand Grip From Baseline to 12 Months. [Baseline and 12 months]

   To investigate the clinical effects of oral glutamine and placebo on hand grip in children with Sickle Cell Anemia (SCA) by comparing the difference between baseline and 12 months of treatment between the two groups. Hand grip strength is a measure of muscle strength.Units are measured in Kg.Muscle strength is measured using a hydraulic hand-held dynamometer.Change was defined as 12 Month measure minus baseline.Muscle strength is measured using the hand grip strength via a hydraulic hand-held dynamometer (Kg).

Eligibility Criteria

Criteria

• Ages Eligible for Study: 5 Years to 18 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

Active patient (presently receiving medical care for SCA) at the St. Jude Comprehensive Sickle Cell Center (SJCSCC) or an affiliate or alliance of St Jude. Patient must not be in the high risk category as defined by the SJCSCC. High risk is defined as follows:

• 3 or more admissions and/or emergency department visits for pain within the past 12 months, or;

• 2 or more episodes of acute chest syndrome within the past 24 months, or;

• A combination of pain and ACS events > 3 within the past 12 months.

• 5-18 years of age

• Diagnosis of Hb SS or HB0Thal

• Weight >15 kg

• <50th percentile for height/age or weight/age or weight/ height, or <90% ideal body weight, or <90% of BMI for age/ gender.

• Is willing to sign informed consent

Exclusion Criteria

• Patients receiving hydroxyurea or any other anti-sickling agent, chronic transfusion, or nutrition supplements.A nutrition supplement is any high calorie or high protein food additive or oral supplement being used for the purpose of weight gain.

• History of poor compliance (Missing two or more clinic appointments in the past year).

• Renal or liver dysfunction

• Renal dysfunction as defined by serum creatinine >1.5 times normal for age based on testing lab.

• Hepatic dysfunction as defined by alanine aminotransferase (ALT) >2 times the upper limit of normal for age based on testing lab.

• Breastfeeding

• Pregnancy.Females of childbearing potential must have negative serum or urine pregnancy test (record date of test).

• Patients enrolled on previous glutamine protocol (SCDGLU).

• Patients who are considered high risk

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	St. Jude Children's Research Hospital	Memphis	Tennessee	United States	38105

Sponsors and Collaborators

• St. Jude Children's Research Hospital
• Thrasher Research Fund

Investigators

• Principal Investigator: Ruth Williams, MS, RD, EdD, St. Jude Children's Research Hospital

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• St. Jude Children's Research Hospital

Publications

None provided.

• Responsible Party: St. Jude Children's Research Hospital
• ClinicalTrials.gov Identifier: NCT00131508
• Other Study ID Numbers: SCGLU2
• First Posted: Aug 18, 2005
• Last Update Posted: Apr 26, 2017
• Last Verified: Mar 1, 2010

• Keywords provided by St. Jude Children's Research Hospital: Hemoglobin S Disease; Sickle Cell Anemia
• Additional relevant MeSH terms: Anemia; Anemia, Sickle Cell

Study Results

Participant Flow

Recruitment Details	27 participants were recruited from July, 2004 through October, 2007.
Pre-assignment Detail	25 of the 27 participants were randomized to either the Glutamine or Placebo arm.

Arm/Group Title	Placebo	Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Period Title: Overall Study
STARTED	12	13
COMPLETED	9	5
NOT COMPLETED	3	8

Baseline Characteristics

Arm/Group Title	Placebo	Glutamine	Total
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.	Total of all reporting groups
Overall Participants	12	13	25
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	9.1 (2.9)	9.1 (3.6)	9.1 (3.2)
Sex: Female, Male (Count of Participants)
Female	6 50%	9 69.2%	15 60%
Male	6 50%	4 30.8%	10 40%

Outcome Measures

1. Primary Outcome

Title	Change in Resting Energy Expenditure From Baseline to 12 Months
Description	To compare the effect of glutamine and placebo on resting energy expenditure (REE) in children with sickle cell anemia (SCA) by comparing the change in REE ratio between baseline and 12 months. REE was measured by indirect calorimetry, using a metabolic cart.REE Ratio =(REE Measured/REE Predicted)x 100).Change was defined as 12 Month REE Ratio minus Baseline REE Ratio.The REE Ratio was evaluated at baseline and 12 months.The REE Ratio is calculated as (REE Measured / REE Predicted) x 100).REE units are measured as (Kcal / day).Change was defined as 12 Month REE Ratio minus Baseline REE Ratio.
Time Frame	Baseline and 12 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Measure Participants	12	13
Median (Full Range) [REE ratio]	-5.5	7.1

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change in REE ratio between the placebo and glutamine groups. The study was designed to provide 80% power at an alpha level of 0.05 for this objective. Due to slow accrual, the sample size of 46 participants required to obtain the designed power of the study was not realized.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.17
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

2. Secondary Outcome

Title	Change in Body Mass Index From Baseline to 12 Months
Description	To investigate the effect of oral glutamine and placebo on body composition in children with SCA by comparing the difference in body mass indexes (BMI) between baseline and 12 months of treatment in the two groups.
Time Frame	Baseline and 12 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Measure Participants	12	13
Median (Full Range) [kg/m2]	0.1	0.8

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change of body mass index between the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.53
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

3. Secondary Outcome

Title	Change in Red Blood Cell Glutamine From Baseline to 12 Months
Description	To investigate the effect of oral glutamine and placebo in children with Sickle Cell Anemia (SCA) by comparing the difference in the levels of red blood cell glutamine between baseline and 12 months of treatment in the two groups.
Time Frame	Baseline and 12 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Measure Participants	12	13
Median (Full Range) [nmol/mg creatinine]	43.5	100.8

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change in red blood cell glutamine between the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.24
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

4. Secondary Outcome

Title	Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports.
Description	Evaluation of quality of life at baseline and 12 months in the glutamine versus placebo group using the PedsQL Version 4.0 inventory. This instrument measures individual well being across physical, emotional, social, and school function categories using patient self-reports and/or parent reports. The tool contains a 15-question, age-specific, self-report inventory (for children age 5-7 years, 8-12 years, and 13-18 years) and a corresponding parent inventory. Lower scores indicate a better quality of life.
Time Frame	Baseline and 12 Months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Measure Participants	12	13
Baseline Parent Reported Physical Function	1.4	2.0
Baseline Parent Reported Emotional Function	1.0	2.0
Baseline Parent Reported Social Function	0.6	1.8
Baseline Parent Reported School Function	1.8	2.0
12 Months Parent Reported Physical Function	2.5	2.1
12 Months Parent Reported Emotional Function	1.4	1.2
12 Months Parent Reported Social Function	2.0	1.2
12 Months Parent Reported School Function	1.8	2.4
Baseline Participant Reported Physical Function	0.9	1.0
Baseline Participant Reported Emotional Function	0.8	1.2
Baseline Participant Reported Social Function	1.0	1.2
Baseline Participant Reported School Function	1.6	1.3
12 Months Participant Reported Physical Function	1.0	1.0
12 Months Participant Reported Emotional Function	0.8	1.2
12 Months Participant Reported Social Function	1.2	1.0
12 Months Participant Reported School Function	1.6	1.8

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between baseline parent reported physical function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.62
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between baseline parent reported emotional function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.14
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between baseline parent reported social function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.20
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 4

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between baseline parent reported school function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.62
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 5

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between 12 month parent reported physical function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.61
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 6

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between 12 month parent reported emotional function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.65
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 7

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between 12 month parent reported social function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.55
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 8

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between 12 month parent reported school function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.69
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 9

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between baseline patient reported physical function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.82
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 10

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between baseline patient reported emotional function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.99
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 11

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between baseline patient reported social function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.30
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 12

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between baseline patient reported school function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.24
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 13

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between 12 month patient reported physical function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.50
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 14

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between 12 month patient reported emotional function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.45
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 15

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between 12 month patient reported social function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.46
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 16

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change between 12 month patient reported school function in the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.84
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

5. Secondary Outcome

Title	Change in Height Z-score From Baseline to 12 Months
Description	To investigate the effect of oral glutamine and placebo on height Z-score in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment.
Time Frame	Baseline and 12 months

Outcome Measure Data

Analysis Population Description

Analyzed patients had a height measurement at both baseline and 12 months.

Arm/Group Title	Placebo	Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Measure Participants	9	5
Median (Full Range) [Z-score]	-0.1	0.0

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo
	Comments	We tested the null hypothesis that the median difference in height Z-Score betweeen baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.20
	Comments
	Method	Wilcoxon Signed Rank Test
	Comments

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Glutamine
	Comments	We tested the null hypothesis that the median difference in height Z-Score betweeen baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.81
	Comments
	Method	Wilcoxon Signed Rank Test
	Comments

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested the null hypothesis that the median difference in height Z-Score betweeen baseline and 12 months differs between the Glutamine and Placebo groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.70
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

6. Secondary Outcome

Title	Change in Height Percentile From Baseline to 12 Months
Description	To investigate the effect of oral glutamine and placebo on height percentile in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment.
Time Frame	Baseline and 12 months

Outcome Measure Data

Analysis Population Description

Analyzed patients had a height measurement at both baseline and 12 months.

Arm/Group Title	Placebo	Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Measure Participants	9	5
Median (Full Range) [Percentile]	0.0	0.0

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo
	Comments	We tested the null hypothesis that the median difference in height percentile between baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.69
	Comments
	Method	Wilcoxon Signed Rank Test
	Comments

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Glutamine
	Comments	We tested the null hypothesis that the median difference in height percentile between baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.75
	Comments
	Method	Wilcoxon Signed Rank Test
	Comments

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We test the null hypothesis that the median difference in height percentile between baseline and 12 months differs between the Glutamine and Placebo groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.93
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

7. Secondary Outcome

Title	Change in Weight Percentile From Baseline to 12 Months
Description	To investigate the effect of oral glutamine and placebo on weight in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment.
Time Frame	Basline and 12 months

Outcome Measure Data

Analysis Population Description

Analyzed patients had a height measurement at both baseline and 12 months.

Arm/Group Title	Placebo	Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Measure Participants	9	5
Median (Full Range) [Percentile]	0.0	7.5

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo
	Comments	We tested the null hypothesis that the median difference in weight percentile between baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.56
	Comments
	Method	Wilcoxon Signed Rank Test
	Comments

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Glutamine
	Comments	We tested the null hypothesis that the median difference in weight percentile between baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	1.0
	Comments
	Method	Wilcoxon Signed Rank Test
	Comments

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We test the null hypothesis that the median difference in weight percentile between baseline and 12 months differs between the Glutamine and Placebo groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.61
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

8. Secondary Outcome

Title	Change in Pulse Rate From Baseline to 12 Months
Description	To investigate the clinical effects of oral glutamine and placebo on pulse rate in children with Sickle Cell Anemia (SCA) by comparing the difference between baseline and 12 months of treatment between the two groups.
Time Frame	Baseline and 12 months

Outcome Measure Data

Analysis Population Description

Analyzed patients had a height measurement at both baseline and 12 months.

Arm/Group Title	Placebo	Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Measure Participants	7	3
Median (Full Range) [Beats per minute (BPM)]	11	17

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change in pulse rate between the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.83
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

9. Secondary Outcome

Title	Change in Hand Grip From Baseline to 12 Months.
Description	To investigate the clinical effects of oral glutamine and placebo on hand grip in children with Sickle Cell Anemia (SCA) by comparing the difference between baseline and 12 months of treatment between the two groups. Hand grip strength is a measure of muscle strength.Units are measured in Kg.Muscle strength is measured using a hydraulic hand-held dynamometer.Change was defined as 12 Month measure minus baseline.Muscle strength is measured using the hand grip strength via a hydraulic hand-held dynamometer (Kg).
Time Frame	Baseline and 12 months

Outcome Measure Data

Analysis Population Description

Analyzed patients had a height measurement at both baseline and 12 months.

Arm/Group Title	Placebo	Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.	Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Measure Participants	6	3
Median (Full Range) [kg]	12	5

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Glutamine
	Comments	We tested for a significant difference in the median change in hand grip between the placebo and glutamine groups.
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.40
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

Adverse Events

Time Frame	Baseline through 12 months for an average of one year.
Adverse Event Reporting Description	Patients were monitored for adverse events throughout the entire course of the study from baseline through 12 months for an average of one year.
Arm/Group Title	Placebo		Glutamine
Arm/Group Description	A mixture of non-essential amino acids containing glycine, alanine, and serine.		Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
All Cause Mortality
	Placebo		Glutamine
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	Placebo		Glutamine
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/12 (0%)		0/13 (0%)
Other (Not Including Serious) Adverse Events
	Placebo		Glutamine
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/12 (0%)		0/13 (0%)

Limitations/Caveats

The study was designed to enroll 46 participants (23 per arm). Due to slow accrual the study was terminated after enrolling 27 participants. Of the 27 participants enrolled, 2 dropped out of the study prior to randomization.

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Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT 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.