Anemia, Sickle CellPhase 2NCT00131508
Trial of Oral Glutamine in Patients With Sickle Cell Anemia
A Phase 2 study testing Glutamine in people with Anemia, Sickle Cell, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 18 years
- Conditions the study is for
- Anemia, Sickle Cell
- What is being tested?
- Glutamine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- May 2004to Apr 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: A Randomized Controlled Trial of Oral Glutamine Supplementation Versus a Placebo Supplement in Children With Sickle Cell Anemia
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.
Read the detailed description
- 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.
- 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.
- 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.
- 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.
- 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.
- This study will evaluate quality of life in children with SCA who have glutamine or placebo for 12 months.
- 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.
- Conditions
- Anemia, Sickle Cell
- Being tested
- Glutamine, Placebo
- Keywords
- Hemoglobin S Disease, Sickle Cell Anemia
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GlutamineDrug
0.6 gm/kg of oral glutamine per day, in two doses for one year.
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| 2 | Experimental | Glutamine Receives: Drug: Glutamine |
| 1 | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Resting Energy Expenditure From Baseline to 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.
Measured: Baseline and 12 months
Secondary outcomes (8)
Change in Body Mass Index From Baseline to 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.
Measured: Baseline and 12 months
Change in Red Blood Cell Glutamine From Baseline to 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.
Measured: Baseline and 12 months
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.
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.
Measured: Baseline and 12 Months
Change in Height Z-score From Baseline to 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.
Measured: Baseline and 12 months
Change in Height Percentile From Baseline to 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.
Measured: Baseline and 12 months
Change in Weight Percentile From Baseline to 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.
Measured: Basline and 12 months
Change in Pulse Rate From Baseline to 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.
Measured: Baseline and 12 months
Change in Hand Grip From Baseline to 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).
Measured: Baseline and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2005 (estimated)
- Study startMay 2004
- Primary completionApr 2009
- Study completionApr 2009
Why it stopped: Due to slow participant accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Anemia, Sickle CellStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Thrasher Research Fund
- Responsible party
- Sponsor
- Investigators
- Ruth Williams, MS, RD, EdD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00131508
- Sponsor's protocol ID
- SCGLU2
- Status last verified
- Mar 2010
- First submitted
- 17 Aug 2005
- Last updated
- 26 Apr 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Controlled Trial of Oral Glutamine Supplementation Versus a Placebo Supplement in Children With Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT00131508
- Acronym / sponsor ID
- SCGLU2
- Conditions
- Anemia, Sickle Cell
- Keywords
- Hemoglobin S Disease, Sickle Cell Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to slow participant accrual
- Enrolment
- 27 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- Thrasher Research Fund
- Study start
- May 2004
- Primary completion
- Apr 2009 (actual)
- Study completion
- Apr 2009 (actual)
- First posted
- 18 Aug 2005 (estimated)
- Results
- Posted 12 May 2010
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
- 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.
- 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.
- 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.
- 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.
- 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.
- This study will evaluate quality of life in children with SCA who have glutamine or placebo for 12 months.
- 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
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 2 | Experimental | Drug: GlutamineGlutamine |
| 1 | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Glutamine0.6 gm/kg of oral glutamine per day, in two doses for one year. | Drug | 2 |
| PlaceboPlacebo | Drug | 1 |
Primary outcome
Change in Resting Energy Expenditure From Baseline to 12 Months
Time frame: 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.
8 secondary outcomes
Change in Body Mass Index From Baseline to 12 Months
Time frame: Baseline and 12 months
Change in Red Blood Cell Glutamine From Baseline to 12 Months
Time frame: Baseline and 12 months
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.
Time frame: Baseline and 12 Months
Change in Height Z-score From Baseline to 12 Months
Time frame: Baseline and 12 months
Change in Height Percentile From Baseline to 12 Months
Time frame: Baseline and 12 months
Change in Weight Percentile From Baseline to 12 Months
Time frame: Basline and 12 months
Change in Pulse Rate From Baseline to 12 Months
Time frame: Baseline and 12 months
Change in Hand Grip From Baseline to 12 Months.
Time frame: Baseline and 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Apr 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- St. Jude Children's Research HospitalLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ruth Williams, MS, RD, EdD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Glutamine in people with Anemia, Sickle Cell, run by St. Jude Children's Research Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 May 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Resting Energy Expenditure From Baseline to 12 Months (time frame: Baseline and 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Apr 2017.