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Trial Outcomes & Findings for Trial of Oral Glutamine in Patients With Sickle Cell Anemia (NCT NCT00131508)

NCT ID: NCT00131508

Last Updated: 2017-04-26

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

27 participants

Primary outcome timeframe

Baseline and 12 months

Results posted on

2017-04-26

Participant Flow

27 participants were recruited from July, 2004 through October, 2007.

25 of the 27 participants were randomized to either the Glutamine or Placebo arm.

Participant milestones

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

Reasons for withdrawal

Reasons for withdrawal
Measure
Placebo
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Overall Study
Disease progression
0
2
Overall Study
Non-Compliance
2
4
Overall Study
Withdrawal by Subject
1
2

Baseline Characteristics

Trial of Oral Glutamine in Patients With Sickle Cell Anemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Placebo
n=12 Participants
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
n=13 Participants
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Total
n=25 Participants
Total of all reporting groups
Age, Continuous
9.1 years
STANDARD_DEVIATION 2.9 • n=5 Participants
9.1 years
STANDARD_DEVIATION 3.6 • n=7 Participants
9.1 years
STANDARD_DEVIATION 3.2 • n=5 Participants
Sex: Female, Male
Female
6 Participants
n=5 Participants
9 Participants
n=7 Participants
15 Participants
n=5 Participants
Sex: Female, Male
Male
6 Participants
n=5 Participants
4 Participants
n=7 Participants
10 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure
Placebo
n=12 Participants
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
n=13 Participants
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Change in Resting Energy Expenditure From Baseline to 12 Months
-5.5 REE ratio
Interval -37.6 to 12.2
7.1 REE ratio
Interval -9.0 to 41.3

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure
Placebo
n=12 Participants
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
n=13 Participants
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Change in Body Mass Index From Baseline to 12 Months
0.1 kg/m2
Interval -0.3 to 1.2
0.8 kg/m2
Interval -0.6 to 0.9

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure
Placebo
n=12 Participants
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
n=13 Participants
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Change in Red Blood Cell Glutamine From Baseline to 12 Months
43.5 nmol/mg creatinine
Interval -173.9 to 137.0
100.8 nmol/mg creatinine
Interval 21.1 to 262.4

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure
Placebo
n=12 Participants
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
n=13 Participants
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
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.
12 Months Parent Reported Physical Function
2.5 Units on a scale
Interval 0.3 to 3.3
2.1 Units on a scale
Interval 0.5 to 2.9
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 Parent Reported Physical Function
1.4 Units on a scale
Interval 0.3 to 3.5
2.0 Units on a scale
Interval 0.0 to 2.9
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 Parent Reported Emotional Function
1.0 Units on a scale
Interval 0.0 to 2.0
2.0 Units on a scale
Interval 0.0 to 4.0
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 Parent Reported Social Function
0.6 Units on a scale
Interval 0.0 to 2.6
1.8 Units on a scale
Interval 0.0 to 3.0
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 Parent Reported School Function
1.8 Units on a scale
Interval 0.8 to 2.6
2.0 Units on a scale
Interval 0.0 to 3.6
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.
12 Months Parent Reported Emotional Function
1.4 Units on a scale
Interval 0.0 to 2.0
1.2 Units on a scale
Interval 0.0 to 2.2
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.
12 Months Parent Reported Social Function
2.0 Units on a scale
Interval 0.0 to 2.8
1.2 Units on a scale
Interval 0.4 to 2.4
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.
12 Months Parent Reported School Function
1.8 Units on a scale
Interval 0.4 to 3.2
2.4 Units on a scale
Interval 1.6 to 3.8
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 Participant Reported Physical Function
0.9 Units on a scale
Interval 0.4 to 2.8
1.0 Units on a scale
Interval 0.1 to 2.0
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 Participant Reported Emotional Function
0.8 Units on a scale
Interval 0.0 to 2.6
1.2 Units on a scale
Interval 0.0 to 2.0
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 Participant Reported Social Function
1.0 Units on a scale
Interval 0.0 to 2.0
1.2 Units on a scale
Interval 0.0 to 2.8
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 Participant Reported School Function
1.6 Units on a scale
Interval 0.8 to 2.8
1.3 Units on a scale
Interval 0.0 to 2.4
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.
12 Months Participant Reported Physical Function
1.0 Units on a scale
Interval 0.5 to 2.1
1.0 Units on a scale
Interval 0.6 to 2.9
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.
12 Months Participant Reported Emotional Function
0.8 Units on a scale
Interval 0.0 to 2.0
1.2 Units on a scale
Interval 0.0 to 3.6
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.
12 Months Participant Reported Social Function
1.2 Units on a scale
Interval 0.4 to 2.2
1.0 Units on a scale
Interval 0.0 to 3.6
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.
12 Months Participant Reported School Function
1.6 Units on a scale
Interval 1.0 to 3.2
1.8 Units on a scale
Interval 1.0 to 3.4

SECONDARY outcome

Timeframe: Baseline and 12 months

Population: Analyzed patients had a height measurement at both 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.

Outcome measures

Outcome measures
Measure
Placebo
n=9 Participants
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
n=5 Participants
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Change in Height Z-score From Baseline to 12 Months
-0.1 Z-score
Interval -52.2 to 0.4
0.0 Z-score
Interval -0.2 to 0.3

SECONDARY outcome

Timeframe: Baseline and 12 months

Population: Analyzed patients had a height measurement at both 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.

Outcome measures

Outcome measures
Measure
Placebo
n=9 Participants
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
n=5 Participants
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Change in Height Percentile From Baseline to 12 Months
0.0 Percentile
Interval -12.5 to 20.0
0.0 Percentile
Interval -12.5 to 14.5

SECONDARY outcome

Timeframe: Basline and 12 months

Population: Analyzed patients had a height measurement at both baseline 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.

Outcome measures

Outcome measures
Measure
Placebo
n=9 Participants
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
n=5 Participants
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Change in Weight Percentile From Baseline to 12 Months
0.0 Percentile
Interval -11.0 to 12.5
7.5 Percentile
Interval -12.5 to 12.5

SECONDARY outcome

Timeframe: Baseline and 12 months

Population: Analyzed patients had a height measurement at both 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.

Outcome measures

Outcome measures
Measure
Placebo
n=7 Participants
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
n=3 Participants
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Change in Pulse Rate From Baseline to 12 Months
11 Beats per minute (BPM)
Interval -22.0 to 44.0
17 Beats per minute (BPM)
Interval -16.0 to 34.0

SECONDARY outcome

Timeframe: Baseline and 12 months

Population: Analyzed patients had a height measurement at both 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).

Outcome measures

Outcome measures
Measure
Placebo
n=6 Participants
A mixture of non-essential amino acids containing glycine, alanine, and serine.
Glutamine
n=3 Participants
Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses.
Change in Hand Grip From Baseline to 12 Months.
12 kg
Interval -3.0 to 19.0
5 kg
Interval 3.0 to 12.0

Adverse Events

Placebo

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Glutamine

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Winfred Wang

St Jude Children's Research Hospital

Phone: 1-866-278-5833

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place