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| ID | Type | Description | Link |
|---|---|---|---|
| IRB 2008-059 | Other Identifier | Institutional Review Board | |
| 1R01FD003531-01 | U.S. FDA Grant/Contract | View source |
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| Name | Class |
|---|---|
| Food and Drug Administration (FDA) | FED |
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The primary hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease (SCD) and thalassemia (Thal) patients with PH. PH is defined as a tricuspid regurgitant jet velocity (TRV) on Doppler echocardiography > 2.5 m/s. We also predict that glutamine therapy will increase arginine bioavailability and subsequently alter sickle red cell endothelial interaction that can be identified using endo-PAT technology through nitric oxide (NO) generation, leading to changes in biological markers, and clinical outcome. Specifically our second hypothesis is that oral glutamine will decrease biomarkers of hemolysis and adhesion molecules, and improve the imbalanced arginine-to-ornithine ratio that occurs in hemolytic anemias, leading to improved arginine bioavailability and clinical endpoints of endothelial dysfunction and PH in patients with SCD and Thal.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment: L-glutamine | Experimental | Patients will receive an 8-week course of oral L-glutamine 10 grams TID |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| L-Glutamine | Drug | Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children < 15 years of age. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Erythrocyte Glutamine/Glutamate Ratio at 8 Weeks | Erythrocyte Glutamine/Glutamate Ratio: a novel biomarker of oxidative stress | 8 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma Glutamine | 8 weeks | |
| Tricuspid Regurgitant Jet Velocity on Doppler Echocardiography | Tricuspid Regurgitant Jet Velocity was measured using Doppler Echocardiography in meters per second. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Claudia Morris, MD | Emory University | Principal Investigator |
| Augusta Saulys, MD | Children's Hosptial & Research Center Oakland | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital & Research Center Oakland | Oakland | California | 94608 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment: L-glutamine | Patients will receive an 8-week course of oral L-glutamine 10 grams TID L-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children < 15 years of age. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Treatment: L-glutamine | Patients will receive an 8-week course of oral L-glutamine 10 grams TID L-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children < 15 years of age. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Erythrocyte Glutamine/Glutamate Ratio at 8 Weeks | Erythrocyte Glutamine/Glutamate Ratio: a novel biomarker of oxidative stress | Blood work only available for N=6 participants, as reported here. | Posted | Mean | Standard Deviation | Ratio | 8 weeks |
|
|
8 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Treatment: L-glutamine | Patients will receive an 8-week course of oral L-glutamine 10 grams TID L-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children < 15 years of age. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pain | General disorders |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sexually Transmitted Disease Related Pain | Reproductive system and breast disorders |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Claudia R. Morris MD, FAAP | Emory University School of Medicine | 404-727-5500 | claudia.r.morris@emory.edu |
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| ID | Term |
|---|---|
| D006976 | Hypertension, Pulmonary |
| D000755 | Anemia, Sickle Cell |
| D013789 | Thalassemia |
| ID | Term |
|---|---|
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D006973 | Hypertension |
| D014652 | Vascular Diseases |
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| ID | Term |
|---|---|
| D005973 | Glutamine |
| ID | Term |
|---|---|
| D024361 | Amino Acids, Basic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000599 | Amino Acids, Diamino |
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| 8 week |
| 6 Minute Walk Distance | The six-minute walk test (6MWT) measures the distance in meters an individual is able to walk over a total of six minutes on a hard, flat surface. | 8 weeks |
| Liver Function Tests | Alanine aminotransferase (ALT) Aspartate aminotransferase (AST) | 8 weeks |
| Renal Function Tests | Creatinine Blood urea nitrogen (BUN) | 8 weeks |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Erythrocyte Glutamine/Glutamate Ratio | Blood work only available for N=6 participants, as reported here. | Mean | Standard Deviation | Ratio |
|
| Plasma Glutamine | Blood work only available for N=6 participants, as reported here. | Mean | Standard Deviation | µmol/L |
|
| Tricuspid Regurgitant Jet Velocity on Doppler Echocardiography | Tricuspid Regurgitant Jet Velocity was measured using Doppler Echocardiography in meters per second. | Mean | Standard Deviation | Meters per second |
|
| 6 Minute Walk Distance | The six-minute walk test (6MWT) measures the distance in meters an individual is able to walk over a total of six minutes on a hard, flat surface. | Mean | Standard Deviation | Meters |
|
| Liver Function Tests | Mean | Standard Deviation | U/L |
|
| Renal Function Tests | Mean | Standard Deviation | mg/dL |
|
|
| Secondary | Plasma Glutamine | Posted | Mean | Standard Deviation | µmol/L | 8 weeks |
|
|
|
| Secondary | Tricuspid Regurgitant Jet Velocity on Doppler Echocardiography | Tricuspid Regurgitant Jet Velocity was measured using Doppler Echocardiography in meters per second. | Data available for 11 participants only because one patient missed their appointment for their week 8 echocardiogram and was lost to follow up, and another patient died of sepsis before week 8 visit. | Posted | Mean | Standard Deviation | Meters per second | 8 week |
|
|
|
| Secondary | 6 Minute Walk Distance | The six-minute walk test (6MWT) measures the distance in meters an individual is able to walk over a total of six minutes on a hard, flat surface. | Posted | Mean | Standard Deviation | Meters | 8 weeks |
|
|
|
| Secondary | Liver Function Tests | Alanine aminotransferase (ALT) Aspartate aminotransferase (AST) | Posted | Mean | Standard Deviation | U/L | 8 weeks |
|
|
|
| Secondary | Renal Function Tests | Creatinine Blood urea nitrogen (BUN) | Posted | Mean | Standard Deviation | mg/dL | 8 weeks |
|
|
|
| 1 |
| 13 |
| 4 |
| 13 |
| 1 |
| 13 |
| Acute Chest Syndrome | General disorders |
|
| Sepsis | Infections and infestations |
|
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| D002318 |
| Cardiovascular Diseases |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D021542 | Amino Acids, Neutral |