Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| R44HL093893 | U.S. NIH Grant/Contract | View source | |
| R01FD004805 | U.S. FDA Grant/Contract | View source |
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Not provided
Not provided
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Not provided
| Name | Class |
|---|---|
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
| Food and Drug Administration (FDA) | FED |
Not provided
Not provided
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Not provided
The purpose of this study was to determine whether the investigational drug SelG1 when given to sickle cell disease patients either taking or not taking hydroxyurea was effective in preventing or reducing the occurrence of pain crises. SelG1 prevents various cells in the bloodstream from sticking together. By stopping these cell-cell interactions, SelG1 may prevent small blood vessels from becoming blocked and therefore reduce the occurrence and severity of pain crises. Other effects of SelG1 was evaluated, as well as the safety of the drug and how long it stayed in the blood stream.
Funding Source - FDA Office of Orphan Products Development (OOPD)
Not provided
Not provided
Not provided
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| High-dose SelG1 (Selg1 5.0 mg/kg) | Experimental | IV Infusion, once every 4 weeks through Week 50 |
|
| Low-dose SelG1 (Selg1 2.5 mg/kg) | Experimental | IV Infusion, once every 4 weeks through Week 50 |
|
| Placebo | Placebo Comparator | IV Infusion, once every 4 weeks through Week 50 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SelG1 | Drug |
| ||
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median | An SCPC is defined as an acute episode of pain with no other medically determined cause than a vasoocclusive event that requires a medical facility visit and treatment with oral or parenteral narcotics, or parenteral non-steroidal anti-inflammatory drugs. The annual rate of SCPC is defined as the total number of pain crises for a patient occurring from the date of randomization to the end date multiplied by 365 divided by the number of days during that same time period. End date is defined as the last dose date plus 14 days. For participants never dosed, the end date was the end of study date. This calculation accounts for early dropouts or lost to follow-up by extrapolating the SCPC rate of every participant to one year. | One year |
| Annual Rate of Sickle Cell-related Pain Crises (SCPC) - Per Standard Median | An SCPC is defined as an acute episode of pain with no other medically determined cause than a vasoocclusive event that requires a medical facility visit and treatment with oral or parenteral narcotics, or parenteral non-steroidal anti-inflammatory drugs. The annual rate of SCPC is defined as the total number of pain crises for a patient occurring from the date of randomization to the end date multiplied by 365 divided by the number of days during that same time period. End date is defined as the last dose date plus 14 days. For participants never dosed, the end date was the end of study date. This calculation accounts for early dropouts or lost to follow-up by extrapolating the SCPC rate of every participant to one year. | One year |
| Measure | Description | Time Frame |
|---|---|---|
| Annual Rate of Days Hospitalized (Key Secondary Endpoint) Per Hodges-Lehmann Median | The annual rate of days hospitalized was calculated as the number of days hospitalized multiplied by 365 divided by the end date minus the date of randomization plus one where the end date is defined as the last dose date plus 14 days (for subjects never dosed, the end date equaled the end of study date, which was the last site contact for these patients). |
Not provided
Key Inclusion Criteria:
Key Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36529836 | Derived | Sy SKB, Tanaka C, Grosch K. Population Pharmacokinetics and Pharmacodynamics of Crizanlizumab in Healthy Subjects and Patients with Sickle Cell Disease. Clin Pharmacokinet. 2023 Feb;62(2):249-266. doi: 10.1007/s40262-022-01193-4. Epub 2022 Dec 18. | |
| 36355805 | Derived | Kanter J, Brown RC, Norris C, Nair SM, Kutlar A, Manwani D, Shah N, Tanaka C, Bodla S, Sanchez-Olle G, Albers U, Liles D. Pharmacokinetics, pharmacodynamics, safety, and efficacy of crizanlizumab in patients with sickle cell disease. Blood Adv. 2023 Mar 28;7(6):943-952. doi: 10.1182/bloodadvances.2022008209. |
Not provided
Not provided
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.
Not provided
Not provided
Not provided
The study included a Screening Phase, Treatment Phase, and Follow-up Evaluation Phase. During the Screening Phase, potential study participants were to be fully screened for both inclusion and exclusion criteria before and undergo clinical and laboratory evaluations within 30 days prior to randomization into the study.
Approx. 174 patients were planned. A total of 198 patients were randomized. 192 patients received at least 1 dose of study drug & were analyzed for safety; 198 patients were analyzed for efficacy, 125 patients contributed data to the analysis of SelG1 PK data, & 176 patients contributed data to the analysis of PD data (% P-selectin inhibition).
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | High-dose SelG1 (Selg1 5.0 mg/kg) | IV Infusion, once every 4 weeks through Week 50 SelG1 |
| FG001 | Low-dose SelG1 (Selg1 2.5 mg/kg) | IV Infusion, once every 4 weeks through Week 50 SelG1 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
Not provided
Not provided
| Drug |
|
| One year |
| Time to First Sickle Cell-related Pain Crisis | Time to first SCPC is defined as months from randomization to first SCPC. A participant without SCPC before withdrawal or completion of the study is considered censored at the time of the end date. End date is defined as the last dose plus 14 days. For participants never dosed, the end date is the end of study date. | Up to one year |
| Time to Second Sickle Cell-related Pain Crisis | Time to second SCPC is defined as months from randomization to second SCPC. A patient with less than two SCPC before withdrawal or completion of the study is considered censored at the time of the end date. End date is defined as the last dose plus 14 days. For patients never dosed, the end date is the end of study date. | Up to one year |
| Annual Rate of Uncomplicated Sickle Cell-related Pain Crisis Per Hodges-Lehmann Median | Uncomplicated SCPC is defined as an acute episode of pain with no known cause for pain other than a vasoocclusive event; requiring a visit to a medical facility; and requiring treatment with a parenteral or oral narcotic (including opiates), or parenteral NSAIDs; but is NOT classified as an acute chest syndrome, hepatic sequestration, splenic sequestration or priapism. | Up to one year |
| Annual Rate of Acute Chest Syndrome Per Hodges-Lehmann Median | Acute Chest Syndrome (ACS) is defined on the basis of the finding of a new pulmonary infiltrate involving at least one complete lung segment that was consistent with alveolar consolidation, but excluding atelectasis (as indicated by chest X-ray). At least one of the following additional signs or symptoms needs to be present as well: a participant had to have reported chest pain, a temperature of more than 38.5oC, tachypnea, wheezing or cough. | One year |
| Patient Reported Outcome: Change From Baseline in Pain Severity/Pain Interference Domain From Brief Pain Inventory (BPI) Questionnaire | The BPI instrument was completed by the patients at pre-specified study visits prior to & during the Treatment & Follow-Up Evaluation Phases. Patients completed the brief pain inventory long-form, 1-week recall at the indicated pre-specified study visits. The BPI is a standardized self-reported questionnaire developed to provide information on the intensity of pain (the sensory dimension) as well as the degree to which pain interferes with function (the reactive dimension). The BPI also asks questions about pain relief, pain quality, & the patient's perception of the cause of pain. Since pain can be quite variable over a day, the BPI asks patients to rate their pain at the time of responding to the questionnaire (pain now), & also at its worst, least, & average over the previous week. The scorings for pain & interference have a range from 0 (no pain/no interference) to 10 (worst pain/complete interference). The BPI scoring manual was used to calculate scores for each domain. | Baseline, Day 15, Week 14, Week 26, Week 38, Week 52, and Week 58, up to 58 weeks |
| Mobile |
| Alabama |
| United States |
| Little Rock | Arkansas | United States |
| Los Angeles | California | United States |
| Oakland | California | United States |
| Orange | California | United States |
| Sacramento | California | United States |
| Aurora | Colorado | United States |
| New Haven | Connecticut | United States |
| Washington D.C. | District of Columbia | United States |
| Daytona Beach | Florida | United States |
| Jacksonville | Florida | United States |
| Miami | Florida | United States |
| Orlando | Florida | United States |
| Tampa | Florida | United States |
| Atlanta | Georgia | United States |
| Augusta | Georgia | United States |
| Chicago | Illinois | United States |
| Peoria | Illinois | United States |
| Indianapolis | Indiana | United States |
| Louisville | Kentucky | United States |
| Baton Rouge | Louisiana | United States |
| New Orleans | Louisiana | United States |
| Baltimore | Maryland | United States |
| Bethesda | Maryland | United States |
| Boston | Massachusetts | United States |
| Detroit | Michigan | United States |
| Jackson | Mississippi | United States |
| Kansas City | Missouri | United States |
| Las Vegas | Nevada | United States |
| New Brunswick | New Jersey | United States |
| Newark | New Jersey | United States |
| Brooklyn | New York | United States |
| New Hyde Park | New York | United States |
| The Bronx | New York | United States |
| Chapel Hill | North Carolina | United States |
| Durham | North Carolina | United States |
| Greenville | North Carolina | United States |
| Cleveland | Ohio | United States |
| Columbus | Ohio | United States |
| Oklahoma City | Oklahoma | United States |
| Philadelphia | Pennsylvania | United States |
| Pittsburgh | Pennsylvania | United States |
| Charleston | South Carolina | United States |
| Sumter | South Carolina | United States |
| Fort Worth | Texas | United States |
| Houston | Texas | United States |
| Norfolk | Virginia | United States |
| Richmond | Virginia | United States |
| Salvador | Estado de Bahia | Brazil |
| Porto Alegre | Rio Grande do Sul | Brazil |
| Campinas | São Paulo | Brazil |
| São José Do Rio Preto | São Paulo | Brazil |
| Rio de Janeiro | Brazil |
| São Paulo | Brazil |
| Kingston | Jamaica |
| 27959701 | Derived | Ataga KI, Kutlar A, Kanter J, Liles D, Cancado R, Friedrisch J, Guthrie TH, Knight-Madden J, Alvarez OA, Gordeuk VR, Gualandro S, Colella MP, Smith WR, Rollins SA, Stocker JW, Rother RP. Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease. N Engl J Med. 2017 Feb 2;376(5):429-439. doi: 10.1056/NEJMoa1611770. Epub 2016 Dec 3. |
| FG002 | Placebo | IV Infusion, once every 4 weeks through Week 50 Placebo |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Intent-to-Treat (ITT): The ITT population includes all randomized patients.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | High-dose SelG1 (Selg1 5.0 mg/kg) | IV Infusion, once every 4 weeks through Week 50 SelG1 |
| BG001 | Low-dose SelG1 (Selg1 2.5 mg/kg) | IV Infusion, once every 4 weeks through Week 50 SelG1 |
| BG002 | Placebo | IV Infusion, once every 4 weeks through Week 50 Placebo |
| BG003 | 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, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Number | participants | No |
|
| 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 | Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median | An SCPC is defined as an acute episode of pain with no other medically determined cause than a vasoocclusive event that requires a medical facility visit and treatment with oral or parenteral narcotics, or parenteral non-steroidal anti-inflammatory drugs. The annual rate of SCPC is defined as the total number of pain crises for a patient occurring from the date of randomization to the end date multiplied by 365 divided by the number of days during that same time period. End date is defined as the last dose date plus 14 days. For participants never dosed, the end date was the end of study date. This calculation accounts for early dropouts or lost to follow-up by extrapolating the SCPC rate of every participant to one year. | Intent-to-Treat (ITT) population: The ITT population includes all randomized participants. | Posted | Median | Full Range | SCPC per year | One year |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | Annual Rate of Sickle Cell-related Pain Crises (SCPC) - Per Standard Median | An SCPC is defined as an acute episode of pain with no other medically determined cause than a vasoocclusive event that requires a medical facility visit and treatment with oral or parenteral narcotics, or parenteral non-steroidal anti-inflammatory drugs. The annual rate of SCPC is defined as the total number of pain crises for a patient occurring from the date of randomization to the end date multiplied by 365 divided by the number of days during that same time period. End date is defined as the last dose date plus 14 days. For participants never dosed, the end date was the end of study date. This calculation accounts for early dropouts or lost to follow-up by extrapolating the SCPC rate of every participant to one year. | Intent-to-Treat (ITT) population: The ITT population includes all randomized participants. | Posted | Median | Full Range | SCPC per year | One year |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Annual Rate of Days Hospitalized (Key Secondary Endpoint) Per Hodges-Lehmann Median | The annual rate of days hospitalized was calculated as the number of days hospitalized multiplied by 365 divided by the end date minus the date of randomization plus one where the end date is defined as the last dose date plus 14 days (for subjects never dosed, the end date equaled the end of study date, which was the last site contact for these patients). | ITT: The ITT population includes all randomized patients. | Posted | Median | Full Range | Days hospitalized per year | One year |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Time to First Sickle Cell-related Pain Crisis | Time to first SCPC is defined as months from randomization to first SCPC. A participant without SCPC before withdrawal or completion of the study is considered censored at the time of the end date. End date is defined as the last dose plus 14 days. For participants never dosed, the end date is the end of study date. | ITT population: The ITT population includes all randomized participants. | Posted | Median | Inter-Quartile Range | months | Up to one year |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Time to Second Sickle Cell-related Pain Crisis | Time to second SCPC is defined as months from randomization to second SCPC. A patient with less than two SCPC before withdrawal or completion of the study is considered censored at the time of the end date. End date is defined as the last dose plus 14 days. For patients never dosed, the end date is the end of study date. | ITT population: The ITT population includes all randomized participants. | Posted | Median | Inter-Quartile Range | months | Up to one year |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Annual Rate of Uncomplicated Sickle Cell-related Pain Crisis Per Hodges-Lehmann Median | Uncomplicated SCPC is defined as an acute episode of pain with no known cause for pain other than a vasoocclusive event; requiring a visit to a medical facility; and requiring treatment with a parenteral or oral narcotic (including opiates), or parenteral NSAIDs; but is NOT classified as an acute chest syndrome, hepatic sequestration, splenic sequestration or priapism. | ITT population: The ITT population includes all randomized participants. | Posted | Median | Full Range | Uncomplicated SCPC per year | Up to one year |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Annual Rate of Acute Chest Syndrome Per Hodges-Lehmann Median | Acute Chest Syndrome (ACS) is defined on the basis of the finding of a new pulmonary infiltrate involving at least one complete lung segment that was consistent with alveolar consolidation, but excluding atelectasis (as indicated by chest X-ray). At least one of the following additional signs or symptoms needs to be present as well: a participant had to have reported chest pain, a temperature of more than 38.5oC, tachypnea, wheezing or cough. | ITT population: The ITT population includes all randomized participants. | Posted | Median | Full Range | accute chest syndrome per year | One year |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Patient Reported Outcome: Change From Baseline in Pain Severity/Pain Interference Domain From Brief Pain Inventory (BPI) Questionnaire | The BPI instrument was completed by the patients at pre-specified study visits prior to & during the Treatment & Follow-Up Evaluation Phases. Patients completed the brief pain inventory long-form, 1-week recall at the indicated pre-specified study visits. The BPI is a standardized self-reported questionnaire developed to provide information on the intensity of pain (the sensory dimension) as well as the degree to which pain interferes with function (the reactive dimension). The BPI also asks questions about pain relief, pain quality, & the patient's perception of the cause of pain. Since pain can be quite variable over a day, the BPI asks patients to rate their pain at the time of responding to the questionnaire (pain now), & also at its worst, least, & average over the previous week. The scorings for pain & interference have a range from 0 (no pain/no interference) to 10 (worst pain/complete interference). The BPI scoring manual was used to calculate scores for each domain. | ITT population: The ITT population includes all randomized participants. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Day 15, Week 14, Week 26, Week 38, Week 52, and Week 58, up to 58 weeks |
|
Adverse Event (AE) timeframe: Adverse events were collected from first dose of study treatment until end of study treatment, up to a maximum duration of 58 weeks.
Adverse Event (AE): Any sign or symptom that occurs during the study treatment. Of the 198 patients randomized, 192 received at least one dose of study treatment and were included in the Safety set.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | High-dose SelG1 (5.0 mg/kg SelG1) | IV Infusion, once every 4 weeks through Week 50 | 2 | 66 | 17 | 66 | 46 | 66 |
| EG001 | Low-dose SelG1 (2.5 mg/kg SelG10 | IV Infusion, once every 4 weeks through Week 50 | 1 | 64 | 21 | 64 | 47 | 64 |
| EG002 | Placebo | IV Infusion, once every 4 weeks through Week 50 | 2 | 62 | 17 | 62 | 42 | 62 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ANAEMIA | Blood and lymphatic system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| LEUKOCYTOSIS | Blood and lymphatic system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| BRADYCARDIA | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| PALPITATIONS | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| RIGHT VENTRICULAR FAILURE | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| SICKLE CELL ANAEMIA WITH CRISIS | Congenital, familial and genetic disorders | MedDRA (21.0) | Systematic Assessment |
| |
| HYPERPARATHYROIDISM PRIMARY | Endocrine disorders | MedDRA (21.0) | Systematic Assessment |
| |
| DIARRHOEA | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| NON-CARDIAC CHEST PAIN | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| SYSTEMIC INFLAMMATORY RESPONSE SYNDROME | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| BILE DUCT STONE | Hepatobiliary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| CHOLANGITIS | Hepatobiliary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| CHOLECYSTITIS ACUTE | Hepatobiliary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| APPENDICITIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| ARTHRITIS INFECTIVE | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| ATYPICAL PNEUMONIA | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| BACTERIAL SEPSIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| CELLULITIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| CLOSTRIDIUM DIFFICILE COLITIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| ENDOCARDITIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| ESCHERICHIA SEPSIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| GASTROENTERITIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| GASTROENTERITIS VIRAL | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| INFLUENZA | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| LOWER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| OSTEOMYELITIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| PNEUMONIA | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| POST PROCEDURAL INFECTION | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| PYELONEPHRITIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| PYELONEPHRITIS ACUTE | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| SEPSIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| SINUSITIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| TOOTH INFECTION | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| URINARY TRACT INFECTION | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| VIRAL INFECTION | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| ACUTE HAEMOLYTIC TRANSFUSION REACTION | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| INFUSION RELATED REACTION | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| NEUTROPHIL COUNT DECREASED | Investigations | MedDRA (21.0) | Systematic Assessment |
| |
| BACK PAIN | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| OSTEONECROSIS | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| COMA | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| DIZZINESS | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| HAEMORRHAGE INTRACRANIAL | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| ISCHAEMIC STROKE | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| SYNCOPE | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| PRE-ECLAMPSIA | Pregnancy, puerperium and perinatal conditions | MedDRA (21.0) | Systematic Assessment |
| |
| MAJOR DEPRESSION | Psychiatric disorders | MedDRA (21.0) | Systematic Assessment |
| |
| SUICIDE ATTEMPT | Psychiatric disorders | MedDRA (21.0) | Systematic Assessment |
| |
| RENAL PAPILLARY NECROSIS | Renal and urinary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| URINARY RETENTION | Renal and urinary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| ACUTE CHEST SYNDROME | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| ASPIRATION | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| STATUS ASTHMATICUS | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| SKIN ULCER | Skin and subcutaneous tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| ABORTION INDUCED | Surgical and medical procedures | MedDRA (21.0) | Systematic Assessment |
| |
| CENTRAL VENOUS CATHETERISATION | Surgical and medical procedures | MedDRA (21.0) | Systematic Assessment |
| |
| HYSTERECTOMY | Surgical and medical procedures | MedDRA (21.0) | Systematic Assessment |
| |
| ANGIOPATHY | Vascular disorders | MedDRA (21.0) | Systematic Assessment |
| |
| DEEP VEIN THROMBOSIS | Vascular disorders | MedDRA (21.0) | Systematic Assessment |
| |
| HYPOTENSION | Vascular disorders | MedDRA (21.0) | Systematic Assessment |
| |
| VENOUS THROMBOSIS LIMB | Vascular disorders | MedDRA (21.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| NEUTROPENIA | Blood and lymphatic system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| ABDOMINAL PAIN | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| ABDOMINAL PAIN UPPER | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| CONSTIPATION | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| DIARRHOEA | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| CHEST PAIN | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| FATIGUE | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| NON-CARDIAC CHEST PAIN | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| PAIN | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| NASOPHARYNGITIS | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| URINARY TRACT INFECTION | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| ROAD TRAFFIC ACCIDENT | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| GAMMA-GLUTAMYLTRANSFERASE INCREASED | Investigations | MedDRA (21.0) | Systematic Assessment |
| |
| PROTHROMBIN TIME PROLONGED | Investigations | MedDRA (21.0) | Systematic Assessment |
| |
| HYPOKALAEMIA | Metabolism and nutrition disorders | MedDRA (21.0) | Systematic Assessment |
| |
| ARTHRALGIA | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| BACK PAIN | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| MUSCULOSKELETAL CHEST PAIN | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| MUSCULOSKELETAL PAIN | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| MYALGIA | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| OSTEONECROSIS | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| DIZZINESS | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| HEADACHE | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| DYSPNOEA | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| OROPHARYNGEAL PAIN | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| PRURITUS | Skin and subcutaneous tissue disorders | MedDRA (21.0) | Systematic Assessment |
|
The Safety population includes all randomized patients who received at least one dose of study drug.
Although the safety is now coded with MedDRA 21.1, the CSR, when first published 1in 2016, used MedDRA 16.1 CFB = Change from Baseline
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 | novartis.email@novartis.com |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D011317 | Priapism |
| 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 |
| D010409 | Penile Diseases |
| D005832 | Genital Diseases, Male |
| D000091662 | Genital Diseases |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| White |
|
| Other |
|
with HU therapy (yes, no) and categorized crises history (2 to 4, 5 to 10) as recorded in the IXRS system as the strata. |
| = 0.180 |
| Hodges-Lehmann median absolute diff. |
| -0.69 |
| 2-Sided |
| 95 |
| -1.84 |
| 0.02 |
| Other |
|
|
|
| Participants |
|
|
|
|
|
|
|
|
|
| Participants |
|
|
|
| Counts |
|---|
| Participants |
|
|
|
IV Infusion, once every 4 weeks through Week 50
SelG1
| OG002 | Placebo | IV Infusion, once every 4 weeks through Week 50 Placebo |
|
|