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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| L-glutamine | Experimental | Patients will be randomized to receive investigational product, L-Glutamine. |
|
| 100% maltodextrin | Placebo Comparator | Patients will be randomized to receive Placebo. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| L-glutamine | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 48 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 48 weeks |
| The Number of Emergency Room/Medical Facility Visits for Sickle Cell Pain |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Yutaka Niihara, MD, MPH | Chairman and CEO | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of South Alabama Medical Center | Mobile | Alabama | 36617 | United States | ||
| Phoenix Children's Hospital Center for Cancer and Blood Disorders |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38775255 | Derived | Bolarinwa AB, Oduwole O, Okebe J, Ogbenna AA, Otokiti OE, Olatinwo AT. Antioxidant supplementation for sickle cell disease. Cochrane Database Syst Rev. 2024 May 22;5(5):CD013590. doi: 10.1002/14651858.CD013590.pub2. | |
| 30021096 | Derived | Niihara Y, Miller ST, Kanter J, Lanzkron S, Smith WR, Hsu LL, Gordeuk VR, Viswanathan K, Sarnaik S, Osunkwo I, Guillaume E, Sadanandan S, Sieger L, Lasky JL, Panosyan EH, Blake OA, New TN, Bellevue R, Tran LT, Razon RL, Stark CW, Neumayr LD, Vichinsky EP; Investigators of the Phase 3 Trial of l-Glutamine in Sickle Cell Disease. A Phase 3 Trial of l-Glutamine in Sickle Cell Disease. N Engl J Med. 2018 Jul 19;379(3):226-235. doi: 10.1056/NEJMoa1715971. |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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|
|
| Placebo | Drug | 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. |
|
|
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. |
| 48 weeks |
| 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. | Baseline, Week 4, 24 and 48 |
| 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. | Baseline, Week 4, 24, and 48 |
| 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. | Baseline, Week 4, 24 and 48 |
| 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. | Baseline, Week 4, 24 and 48 |
| 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. | Baseline, Week 4, Week 24 and Week 48 |
| 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. | Baseline, Week 4, Week 24 and Week 48 |
| 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. | Baseline, Week 4, Week 24 and Week 48 |
| Phoenix |
| Arizona |
| 85016 |
| United States |
| Kaiser Permanente | Inglewood | California | 90301 | United States |
| Children's Hospital & Research Center at Oakland | Oakland | California | 94609 | United States |
| Children's Hospital of Orange County | Orange | California | 92868 | United States |
| Harbor-UCLA Medical Center | Torrance | California | 90509 | United States |
| University of Denver School of Medicine Sickle Cell Treatment & Research Center | Aurora | Colorado | 80045 | United States |
| Howard University Hospital & Howard University | Washington D.C. | District of Columbia | 20060 | United States |
| University of Florida | Gainesville | Florida | 32610-0296 | United States |
| All Children's Hospital | St. Petersburg | Florida | 33701 | United States |
| Children's Healthcare of Atlanta at Egleston/Emory University | Atlanta | Georgia | 30322 | United States |
| University of Illinois at Chicago | Chicago | Illinois | 60612 | United States |
| University of Louisville School of Medicine | Louisville | Kentucky | 40202 | United States |
| Sickle Cell Center of S. Louisiana, Tulane University School of Medicine | New Orleans | Louisiana | 70112 | United States |
| Johns Hopkins University | Baltimore | Maryland | 21205 | United States |
| Boston University Medical Center | Boston | Massachusetts | 02118 | United States |
| Children's Hospital of Michigan | Detroit | Michigan | 48201 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| Children's Mercy Hospitals and Clinics | Kansas City | Missouri | 64108 | United States |
| Children's Specialty Center of Nevada | Las Vegas | Nevada | 89109 | United States |
| Comprehensive Cancer Center of Nevada | Las Vegas | Nevada | 89109 | United States |
| Cooper University Hospital | Camden | New Jersey | 08103 | United States |
| The Brooklyn Hospital Center | Brooklyn | New York | 11201 | United States |
| SUNY - Downstate Medical Center | Brooklyn | New York | 11203 | United States |
| Brookdale University Hospital and Medical Center | Brooklyn | New York | 11212 | United States |
| New York Methodist Hospital - SC/Thalassemia Program | Brooklyn | New York | 11215 | United States |
| Interfaith Medical Center | Brooklyn | New York | 11238 | United States |
| Bronx Lebanon Hospital | The Bronx | New York | 11203 | United States |
| Presbyterian Blume Pediatric Hematology-Oncology Clinic | Charlotte | North Carolina | 28204 | United States |
| University of Tennessee Cancer Institute | Memphis | Tennessee | 38104 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298-0306 | United States |
| FG001 | Placebo | Patients will be randomized to receive Placebo. Placebo (100% maltodextrin): 0.3 g/kg of placebo 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. |
| COMPLETED |
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| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 | 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. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Diagnosis | Count of Participants | Participants |
|
| 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 | 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. | Intent-to-Treat Population - Included all patients who were randomized and dispensed study medication. | Posted | Median | Full Range | Number of crises | 48 weeks |
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| 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. | Intent-to-Treat Population - Included all patients who were randomized and dispensed study medication. | Posted | Median | Full Range | Number of hospitalizations | 48 weeks |
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| 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. | Intent-to-Treat Population - Included all patients who were randomized and dispensed study medication. | Posted | Median | Full Range | Number of ER visits | 48 weeks |
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| 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. | Safety population - The safety population included all patients who received at least 1 dose of study medication. | Posted | Mean | Standard Deviation | g/dL | Baseline, Week 4, 24 and 48 |
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| 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. | Safety Population which includes all patients that took at least one dose of study medication. | Posted | Mean | Standard Deviation | mm Hg | Baseline, Week 4, 24, and 48 |
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| 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. | Safety population - The safety population included all patients who received at least 1 dose of study medication. | Posted | Mean | Standard Deviation | % of red blood cells | Baseline, Week 4, 24 and 48 |
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| 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. | Safety population - The safety population included all patients who received at least 1 dose of study medication. | Posted | Mean | Standard Deviation | 1000 cells/uL | Baseline, Week 4, 24 and 48 |
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| 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. | Safety Population which includes all patients that took at least one dose of study medication. | Posted | Mean | Standard Deviation | bpm | Baseline, Week 4, Week 24 and Week 48 |
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| 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. | Safety Population which includes all patients that took at least one dose of study medication. | Posted | Mean | Standard Deviation | degree C | Baseline, Week 4, Week 24 and Week 48 |
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| 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. | Safety Population which includes all patients that took at least one dose of study medication. | Posted | Mean | Standard Deviation | breaths/min | Baseline, Week 4, Week 24 and Week 48 |
|
Adverse events data were collected throughout the course of the study (53 weeks or about 1 year).
The Safety population will include all patients who received at least one dose of study medication.
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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 | 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. | 118 | 151 | 148 | 151 | ||
| EG001 | 100% Maltodextrin | Patients will be randomized to receive Placebo. 100% maltodextrin: 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. | 68 | 78 | 78 | 78 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute Chest Syndrome | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Aplastic anaemia | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypersplenism | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Leucocytosis | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cardiac Arrest | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Sickle cell anaemia with crisis | Congenital, familial and genetic disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pancreatic acute | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Death | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Drug withdrawal syndrome | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Necrosis | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cholelithiasis | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hyperbilirubinaemia | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Gastrointestinal viral infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Lobar pneumonia | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Osteomyelitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Osteomyelitis acute | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Pharnygitis streptococcal | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Sinustitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Tonsilitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Tooth infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Complication of device removal | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Device leakage | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Device malfunction | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Device occlusion | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Haemolytic transfusion reaction | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Ligament rupture | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Post-traumatic reaction | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Traumatic arthropathy | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hyperkalaemia | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Arthropathy | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Bunion | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Bone infarction | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Joint effusion | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pregnancy | Pregnancy, puerperium and perinatal conditions | MedDRA (12.0) | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Renal failure acute | Renal and urinary disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Priapism | Reproductive system and breast disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Bronchial hyperreactivity | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Haemothorax | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Respiratory depression | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
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| Hip arthroplasty | Surgical and medical procedures | MedDRA (12.0) | Systematic Assessment |
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| Strabismus correction | Surgical and medical procedures | MedDRA (12.0) | Systematic Assessment |
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| Tonsillectomy | Surgical and medical procedures | MedDRA (12.0) | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Thrombophlebits superficial | Vascular disorders | MedDRA (12.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute chest syndrome | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
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| Leukocytosis | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
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| Anaemia | Blood and lymphatic system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Sickle cell anaemia with crisis | Congenital, familial and genetic disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Ocular icterus | Eye disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Chest pain | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypomanesaemia | Investigations | MedDRA (12.0) | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Oropharnyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
|
PI agrees that any INFORMATION submitted to it by EMMAUS shall be maintained in secrecy for a period of seven (7) years from each disclosure of INFORMATION. PI will use the up most due diligence to prevent disclosure by it except to its employees, agents, and contractors necessary for evaluation, all of whom shall be bound by similar written obligations of confidentiality, and who agree not to use the INFORMATION for any purpose other than for evaluation purposes.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Yutaka Niihara, MD, MPH | Emmaus Medical, Inc | 310-214-0065 | yniihara@emmausmedical.com |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D000098644 | Vaso-Occlusive Crises |
| ID | Term |
|---|---|
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| D005973 | Glutamine |
| C008315 | maltodextrin |
| ID | Term |
|---|---|
| D024361 | Amino Acids, Basic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000599 | Amino Acids, Diamino |
| D021542 | Amino Acids, Neutral |
Not provided
Not provided
| >=65 years |
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| Male |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Sickle Beta plus Thalassemia |
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| Sickle Beta zero Thalassemia |
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