Sickle Cell Anemia, Sickle ß0-Thalassemia
Conditions
Keywords
Sickle Cell Anemia, Sickle Cell Pain Crises, Painful Crises, Sickle Cell Pain, Vaso-occlusive Crises
Brief summary
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for Sickle Cell Anemia or Sickle ß0 Thalassemia as evaluated by the number of occurrences of sickle cell crises.
Detailed description
Primary objective: To evaluate the efficacy of oral L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia as evaluated by the number of occurrences of sickle cell crises. Secondary objectives: To assess the effect of oral L-glutamine on: (a) frequency of hospitalizations for sickle cell pain; (b) frequency of emergency room/medical facility visits for sickle cell pain; and (c) hematological parameters (hemoglobin, hematocrit, and reticulocyte count); and to assess the safety of L-glutamine as a therapy for sickle cell anemia as evaluated by adverse events, laboratory parameters, and vital signs. Methodology: This was a 2:1 randomized, double-blind, placebo-controlled, parallel-group, multicenter study in patients with sickle cell anemia and sickle ß0-thalassemia who were at least 5 years old. Informed consent was obtained up to four weeks prior to Week 0 (Baseline). Screening procedures were performed anytime between the date of consent and Week 0, as long as all eligibility criteria had been confirmed prior to Week 0. At Week 0, patients were randomized (to L-glutamine or placebo) and underwent 48 weeks of treatment (orally BID), with dose calculated according to patient weight. Patient clinic visits occurred every 4 weeks, and phone calls took place between visits to monitor compliance. After 48 weeks of treatment, the dose was tapered to 0 within 3 weeks. A final evaluation visit occurred 2 weeks after last dose for a total of 53 weeks on study.
Interventions
DRUGL-glutamine
0.3 g/kg of L-glutamine will be administered twice a day orally to each patient for 48 weeks. The dosage will be in increments of 5 grams based on weight. The upper limit for daily dose of study medication will be set at 30 grams. Patients will be given verbal and written instructions for self-administration of the study medication at the Baseline visit. The powder can be mixed with water or most non-heated beverages other than alcohol, or can be mixed with most non-heated foods such as yogurt, applesauce, or cereal for administration. Mixing L-glutamine with soda or highly acidic juices (such as grapefruit juice or lemonade) is not recommended.
DRUGPlacebo
0.3 g/kg of placebo (100% maltodextrin) will be administered twice a day orally to each patient for 48 weeks. The dosage will be in increments of 5 grams based on weight. The upper limit for daily dose of study medication will be set at 30 grams. Patients will be given verbal and written instructions for self-administration of the study medication at the Baseline visit. The powder can be mixed with water or most non-heated beverages other than alcohol, or can be mixed with most non-heated foods such as yogurt, applesauce, or cereal for administration.
Sponsors
Emmaus Medical, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
5 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patient is at least five years of age. * Patient has been diagnosed with sickle cell anemia or sickle ß°-thalassemia (documented by hemoglobin electrophoresis). * Patient has had at least two documented episodes of sickle cell 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 duration of the study. * 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 avoid pregnancy during the study and is willing and agrees to practice a recognized form of birth control during the course of the study (e.g. barrier, birth control pills, abstinence).
Exclusion criteria
* Patient has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit. * Patient has prothrombin time 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 uncontrolled liver disease or renal insufficiency. * Patient is pregnant or lactating or has the intention of becoming pregnant during the study (if female and of child-bearing potential). * Patient is currently taking or has been treated with any form of glutamine supplement within 30 days of the screening visit. * Patient has been treated with an experimental anti-sickling medication/ treatment within 30 days of the screening visit (with the exception of hydroxyurea in pediatric patients). * Patient is currently taking or has been treated with an investigational drug within 30 days of the screening visit (with the exception of hydroxyurea in pediatric patients). * Patient is currently enrolled in an investigational drug or device study and/or has participated in such a study 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Number of Occurrences of Sickle Cell Crises | 48 weeks | The number of occurrences of protocol-defined sickle cell crises that occur from Week 0 to Week 48 will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The Number of Emergency Room/Medical Facility Visits for Sickle Cell Pain | 48 weeks | The number of emergency room visits or medical facility visits that occur from Week 0 to Week 48, will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia. |
| The Effect of Oral -L-glutamine on Hematological Parameters | Baseline, Week 4, 24 and 48 | To assess the effect of oral L-glutamine on hematological parameters (hemoglobin), Change from Baseline will be reported at Weeks 4, 24 and 48. |
| The Number of Hospitalizations for Sickle Cell Pain | 48 weeks | The number of hospitalizations that occur from Week 0 to Week 48, will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia. |
| The Effect of Oral L-glutamine on Hematological Parameters | Baseline, Week 4, 24 and 48 | To assess the effect of oral L-glutamine on hematological parameters (hematocrit), Change from Baseline will be reported at Weeks 4, 24 and 48. |
| Effect of Oral L-glutamine on Vital Signs | Baseline, Week 4, Week 24 and Week 48 | To assess the effect of oral L-glutamine on Vital signs (temperature). Change from Baseline will be reported at Weeks 4, 24, and 48. |
| The Effect of Oral L-glutamine on Vital Signs | Baseline, Week 4, 24, and 48 | To assess the effect of oral L-glutamine on Vital signs (systolic and diastolic blood pressure). Change from Baseline will be reported at Weeks 4, 24, and 48. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| L-glutamine Patients will be randomized to receive investigational product, L-Glutamine.
L-glutamine: 0.3 g/kg of L-glutamine will be administered twice a day orally to each patient for 48 weeks. The dosage will be in increments of 5 grams based on weight. The upper limit for daily dose of study medication will be set at 30 grams. Patients will be given verbal and written instructions for self-administration of the study medication at the Baseline visit. The powder can be mixed with water or most non-heated beverages other than alcohol, or can be mixed with most non-heated foods such as yogurt, applesauce, or cereal for administration. Mixing L-glutamine with soda or highly acidic juices (such as grapefruit juice or lemonade) is not recommended. | 152 |
| Placebo Patients will be randomized to receive Placebo.
Placebo: 0.3 g/kg of placebo (100% maltodextrin) will be administered twice a day orally to each patient for 48 weeks. The dosage will be in increments of 5 grams based on weight. The upper limit for daily dose of study medication will be set at 30 grams. Patients will be given verbal and written instructions for self-administration of the study medication at the Baseline visit. The powder can be mixed with water or most non-heated beverages other than alcohol, or can be mixed with most non-heated foods such as yogurt, applesauce, or cereal for administration. | 78 |
| Total | 230 |
Baseline characteristics
| Characteristic | L-glutamine | Placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 75 Participants | 43 Participants | 118 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 77 Participants | 35 Participants | 112 Participants |
| Age, Continuous | 22.4 years STANDARD_DEVIATION 12.32 | 21.4 years STANDARD_DEVIATION 12.42 | 22.0 years STANDARD_DEVIATION 12.33 |
| Diagnosis Sickle Beta plus Thalassemia | 2 Participants | 0 Participants | 2 Participants |
| Diagnosis Sickle Beta zero Thalassemia | 14 Participants | 7 Participants | 21 Participants |
| Diagnosis Sickle Cell Anemia | 136 Participants | 71 Participants | 207 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 144 Participants | 73 Participants | 217 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 8 Participants | 5 Participants | 13 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Female | 79 Participants | 45 Participants | 124 Participants |
| Sex: Female, Male Male | 73 Participants | 33 Participants | 106 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 148 / 151 | 78 / 78 |
| serious Total, serious adverse events | 118 / 151 | 68 / 78 |
Outcome results
Primary
The Number of Occurrences of Sickle Cell Crises
The number of occurrences of protocol-defined sickle cell crises that occur from Week 0 to Week 48 will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia.
Time frame: 48 weeks
Population: Intent-to-Treat Population - Included all patients who were randomized and dispensed study medication.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| L-glutamine | The Number of Occurrences of Sickle Cell Crises | 3 Number of crises |
| 100% Maltodextrin | The Number of Occurrences of Sickle Cell Crises | 4 Number of crises |
p-value: 0.0052Cochran-Mantel-Haenszel
Secondary
Effect of Oral L-glutamine on Vital Signs
To assess the effect of oral L-glutamine on Vital signs (temperature). Change from Baseline will be reported at Weeks 4, 24, and 48.
Time frame: Baseline, Week 4, Week 24 and Week 48
Population: Safety Population which includes all patients that took at least one dose of study medication.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| L-glutamine | Effect of Oral L-glutamine on Vital Signs | Temperature at Baseline | 36.85 degree C | Standard Deviation 0.387 |
| L-glutamine | Effect of Oral L-glutamine on Vital Signs | Change in Temperature at Week 4 | -0.06 degree C | Standard Deviation 0.436 |
| L-glutamine | Effect of Oral L-glutamine on Vital Signs | Change in Temperature at Week 24 | -0.05 degree C | Standard Deviation 0.469 |
| L-glutamine | Effect of Oral L-glutamine on Vital Signs | Change in Temperature at Week 48 | -0.09 degree C | Standard Deviation 0.442 |
| 100% Maltodextrin | Effect of Oral L-glutamine on Vital Signs | Change in Temperature at Week 48 | 0.05 degree C | Standard Deviation 0.521 |
| 100% Maltodextrin | Effect of Oral L-glutamine on Vital Signs | Temperature at Baseline | 36.83 degree C | Standard Deviation 0.403 |
| 100% Maltodextrin | Effect of Oral L-glutamine on Vital Signs | Change in Temperature at Week 24 | 0.03 degree C | Standard Deviation 0.51 |
| 100% Maltodextrin | Effect of Oral L-glutamine on Vital Signs | Change in Temperature at Week 4 | -0.02 degree C | Standard Deviation 0.54 |
Secondary
The Effect of Oral -L-glutamine on Hematological Parameters
To assess the effect of oral L-glutamine on hematological parameters (hemoglobin), Change from Baseline will be reported at Weeks 4, 24 and 48.
Time frame: Baseline, Week 4, 24 and 48
Population: Safety population - The safety population included all patients who received at least 1 dose of study medication.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| L-glutamine | The Effect of Oral -L-glutamine on Hematological Parameters | Hemoglobin at Baseline | 8.82 g/dL | Standard Deviation 1.43 |
| L-glutamine | The Effect of Oral -L-glutamine on Hematological Parameters | Change in Hemoglobin at week 4 | 0.04 g/dL | Standard Deviation 0.84 |
| L-glutamine | The Effect of Oral -L-glutamine on Hematological Parameters | Change in Hemoglobin at Week 24 | -0.17 g/dL | Standard Deviation 1.01 |
| L-glutamine | The Effect of Oral -L-glutamine on Hematological Parameters | Change in Hemoglobin at Week 48 | -0.12 g/dL | Standard Deviation 0.95 |
| 100% Maltodextrin | The Effect of Oral -L-glutamine on Hematological Parameters | Change in Hemoglobin at Week 48 | -0.12 g/dL | Standard Deviation 0.96 |
| 100% Maltodextrin | The Effect of Oral -L-glutamine on Hematological Parameters | Hemoglobin at Baseline | 8.71 g/dL | Standard Deviation 1.17 |
| 100% Maltodextrin | The Effect of Oral -L-glutamine on Hematological Parameters | Change in Hemoglobin at Week 24 | -0.12 g/dL | Standard Deviation 1.24 |
| 100% Maltodextrin | The Effect of Oral -L-glutamine on Hematological Parameters | Change in Hemoglobin at week 4 | 0.23 g/dL | Standard Deviation 0.71 |
Secondary
The Effect of Oral L-glutamine on Hematological Parameters
To assess the effect of oral L-glutamine on hematological parameters (hematocrit), Change from Baseline will be reported at Weeks 4, 24 and 48.
Time frame: Baseline, Week 4, 24 and 48
Population: Safety population - The safety population included all patients who received at least 1 dose of study medication.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| L-glutamine | The Effect of Oral L-glutamine on Hematological Parameters | Hematocrit at Baseline | 27.67 % of red blood cells | Standard Deviation 4.4 |
| L-glutamine | The Effect of Oral L-glutamine on Hematological Parameters | Change in Hematocrit at Week 4 | 0.16 % of red blood cells | Standard Deviation 2.77 |
| L-glutamine | The Effect of Oral L-glutamine on Hematological Parameters | Change in Hematocrit Week 24 | -0.26 % of red blood cells | Standard Deviation 3.42 |
| L-glutamine | The Effect of Oral L-glutamine on Hematological Parameters | Change in Hematocrit at Week 48 | 0.16 % of red blood cells | Standard Deviation 3.27 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Hematological Parameters | Change in Hematocrit at Week 48 | 0.11 % of red blood cells | Standard Deviation 3.19 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Hematological Parameters | Hematocrit at Baseline | 27.53 % of red blood cells | Standard Deviation 3.61 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Hematological Parameters | Change in Hematocrit Week 24 | -0.15 % of red blood cells | Standard Deviation 4.13 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Hematological Parameters | Change in Hematocrit at Week 4 | 0.75 % of red blood cells | Standard Deviation 2.52 |
Secondary
The Effect of Oral L-glutamine on Hematological Parameters
To assess the effect of oral L-glutamine on hematological parameters (reticulocyte count), Change from Baseline will be reported at Weeks 4, 24 and 48.
Time frame: Baseline, Week 4, 24 and 48
Population: Safety population - The safety population included all patients who received at least 1 dose of study medication.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| L-glutamine | The Effect of Oral L-glutamine on Hematological Parameters | Reticulocyte (Abs) | 283.62 1000 cells/uL | Standard Deviation 129.49 |
| L-glutamine | The Effect of Oral L-glutamine on Hematological Parameters | Change in Reticulocyte (Abs) at Week 4 | -9.28 1000 cells/uL | Standard Deviation 104.88 |
| L-glutamine | The Effect of Oral L-glutamine on Hematological Parameters | Change in Reticulocyte (Abs) at Week 24 | 7.94 1000 cells/uL | Standard Deviation 111.85 |
| L-glutamine | The Effect of Oral L-glutamine on Hematological Parameters | Change in Reticulocyte (Abs) at Week 48 | 50.89 1000 cells/uL | Standard Deviation 112.93 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Hematological Parameters | Change in Reticulocyte (Abs) at Week 48 | 26.27 1000 cells/uL | Standard Deviation 140.65 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Hematological Parameters | Reticulocyte (Abs) | 295.03 1000 cells/uL | Standard Deviation 140.1 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Hematological Parameters | Change in Reticulocyte (Abs) at Week 24 | -1.93 1000 cells/uL | Standard Deviation 137.65 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Hematological Parameters | Change in Reticulocyte (Abs) at Week 4 | -23.09 1000 cells/uL | Standard Deviation 160.41 |
Secondary
The Effect of Oral L-glutamine on Vital Signs
To assess the effect of oral L-glutamine on Vital signs (respiration). Change from Baseline will be reported at Weeks 4, 24, and 48.
Time frame: Baseline, Week 4, Week 24 and Week 48
Population: Safety Population which includes all patients that took at least one dose of study medication.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Respiration at Baseline | 18.9 breaths/min | Standard Deviation 3 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Respiration at Week 24 | -0.7 breaths/min | Standard Deviation 3.03 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Respiration at Week 48 | -0.7 breaths/min | Standard Deviation 3.25 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Respiration at Week 4 | -0.2 breaths/min | Standard Deviation 3.01 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Respiration at Week 48 | -0.6 breaths/min | Standard Deviation 2.94 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Respiration at Baseline | 19.1 breaths/min | Standard Deviation 2.34 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Respiration at Week 4 | -0.2 breaths/min | Standard Deviation 2.55 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Respiration at Week 24 | -0.6 breaths/min | Standard Deviation 3.36 |
Secondary
The Effect of Oral L-glutamine on Vital Signs
To assess the effect of oral L-glutamine on Vital signs (systolic and diastolic blood pressure). Change from Baseline will be reported at Weeks 4, 24, and 48.
Time frame: Baseline, Week 4, 24, and 48
Population: Safety Population which includes all patients that took at least one dose of study medication.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Systolic blood pressure at Baseline | 111.3 mm Hg | Standard Deviation 11.2 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change Systolic blood pressure at Week 4 | 0.5 mm Hg | Standard Deviation 11.19 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Systolic blood pressure at Week 24 | 1.1 mm Hg | Standard Deviation 10.78 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Systolic blood pressure at Week 48 | 2.2 mm Hg | Standard Deviation 10.78 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Diastolic blood pressure at Baseline | 64.8 mm Hg | Standard Deviation 8.61 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Diastolic blood pressure at Week 4 | -0.7 mm Hg | Standard Deviation 9.08 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Diastolic blood pressure at Week 24 | -0.7 mm Hg | Standard Deviation 8.4 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Diastolic blood pressure at Week 48 | 0.4 mm Hg | Standard Deviation 8.71 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Diastolic blood pressure at Week 48 | 2.0 mm Hg | Standard Deviation 9.96 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Systolic blood pressure at Baseline | 114.6 mm Hg | Standard Deviation 14.16 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Diastolic blood pressure at Baseline | 66.2 mm Hg | Standard Deviation 9.75 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change Systolic blood pressure at Week 4 | -0.2 mm Hg | Standard Deviation 10.68 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Diastolic blood pressure at Week 24 | 0.6 mm Hg | Standard Deviation 12.44 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Systolic blood pressure at Week 24 | 0.5 mm Hg | Standard Deviation 14.23 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Diastolic blood pressure at Week 4 | 0.3 mm Hg | Standard Deviation 10.29 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Systolic blood pressure at Week 48 | 2.6 mm Hg | Standard Deviation 15.7 |
Secondary
The Effect of Oral L-glutamine on Vital Signs
To assess the effect of oral L-glutamine on Vital signs (pulse rate). Change from Baseline will be reported at Weeks 4, 24, and 48.
Time frame: Baseline, Week 4, Week 24 and Week 48
Population: Safety Population which includes all patients that took at least one dose of study medication.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Pulse Rate (bpm) at Baseline | 85.6 bpm | Standard Deviation 13.15 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Pulse Rate (bpm) at Week 4 | -0.1 bpm | Standard Deviation 12.07 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Pulse Rate (bpm) at Week 24 | 3.0 bpm | Standard Deviation 14.58 |
| L-glutamine | The Effect of Oral L-glutamine on Vital Signs | Change in Pulse Rate (bpm) at 48 | 1.1 bpm | Standard Deviation 14.74 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Pulse Rate (bpm) at 48 | 0.2 bpm | Standard Deviation 15.33 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Pulse Rate (bpm) at Baseline | 88.5 bpm | Standard Deviation 14.07 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Pulse Rate (bpm) at Week 24 | -1.5 bpm | Standard Deviation 15.05 |
| 100% Maltodextrin | The Effect of Oral L-glutamine on Vital Signs | Change in Pulse Rate (bpm) at Week 4 | -0.4 bpm | Standard Deviation 14.96 |
Secondary
The Number of Emergency Room/Medical Facility Visits for Sickle Cell Pain
The number of emergency room visits or medical facility visits that occur from Week 0 to Week 48, will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia.
Time frame: 48 weeks
Population: Intent-to-Treat Population - Included all patients who were randomized and dispensed study medication.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| L-glutamine | The Number of Emergency Room/Medical Facility Visits for Sickle Cell Pain | 1 Number of ER visits |
| 100% Maltodextrin | The Number of Emergency Room/Medical Facility Visits for Sickle Cell Pain | 1 Number of ER visits |
p-value: 0.0888Cochran-Mantel-Haenszel
Secondary
The Number of Hospitalizations for Sickle Cell Pain
The number of hospitalizations that occur from Week 0 to Week 48, will be used to evaluate the efficacy of oral L-glutamine as a treatment for sickle cell anemia and beta-0 thalassemia.
Time frame: 48 weeks
Population: Intent-to-Treat Population - Included all patients who were randomized and dispensed study medication.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| L-glutamine | The Number of Hospitalizations for Sickle Cell Pain | 2 Number of hospitalizations |
| 100% Maltodextrin | The Number of Hospitalizations for Sickle Cell Pain | 3 Number of hospitalizations |
p-value: 0.0045Cochran-Mantel-Haenszel