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| ID | Type | Description | Link |
|---|---|---|---|
| RESET | Other Identifier | Alias Study Number |
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| Name | Class |
|---|---|
| GlycoMimetics Incorporated | INDUSTRY |
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This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis necessitating hospitalization.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Rivipansel Treatment Arm | Experimental |
| |
| Placebo Treatment Arm | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Rivipansel | Drug | Rivipansel (GMI-1070) will be infused intravenously every 12 hours up to 15 doses maximum. Subjects aged 12 and over who weigh more than 40 kilograms will receive a dose of 1680 mg of rivipansel, followed by a dose of 840 mg of rivipansel every 12 hours. All subjects aged 6 to 11 years and any subject who weighs 40 kilograms or less, will receive weight-based dosing (mg/kg) of 40 mg/kg of rivipansel (maximum of 1680 mg) followed by a dose of 20 mg/kg of rivipansel (maximum of 840 mg) every 12 hours. |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Readiness for Discharge From Hospital | Time to readiness-for-discharge from hospital was defined as the difference (in hours) between the time and date when all criteria for readiness-for-discharge were met and the start time and date of the first infusion (loading dose) of study drug. Criteria for readiness-for-discharge were met when all of the applicable 6 criteria (in relation to treatment of VOC and complications related to the VOC) were documented to have occurred. The six criteria were: 1) only oral pain medication was required, 2) acute complications related to the VOC (such as acute chest syndrome, stroke, priapism) had resolved to the extent that management could be in an outpatient setting, 3) IV opioids had been discontinued, 4) IV hydration had been discontinued, 5) IV antibiotics had been discontinued and 6) red blood cell (RBC) transfusion was no longer required for treatment of this VOC. | Day 1 up to the latest day when all 6 criteria of readiness-for-discharge were met (up to an average of Day 8) |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Discharge From Hospital | Time to discharge from hospital was defined as the difference (in hours) between the time and date of the hospital discharge order from a qualified healthcare provider and the start time and date of the first infusion (loading dose) of study drug. | Day 1 up to the latest day when the order of hospital discharge was issued by a qualified healthcare provider (up to an average of Day 8) |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) as Per Genotype | AE: any untoward medical occurrence in a participant who received investigational product without regard to possibility of a causal relationship. SAE: an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; medically important events. An adverse event was considered a treatment-emergent adverse event (TEAE) if the event started during the effective duration of treatment (all events that started on or after the first dosing). AEs included both serious and non-serious adverse events. Participants with AEs and SAEs were categorized by genotype categories. Category 1= participants with hemoglobin SS, hemoglobin S beta0 thalassemia and hemoglobin SD; category 2= participants with hemoglobin SC, hemoglobin S beta+ thalassemia and hemoglobin S-Variant (other than HbSD). |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of South Alabama Children's and Women's Hospital | Mobile | Alabama | 36604 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35981565 | Derived | Dampier CD, Telen MJ, Wun T, Brown RC, Desai P, El Rassi F, Fuh B, Kanter J, Pastore Y, Rothman J, Taylor JG, Readett D, Sivamurthy KM, Tammara B, Tseng LJ, Lozier JN, Thackray H, Magnani JL, Hassell KL; RESET Investigators. A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis. Blood. 2023 Jan 12;141(2):168-179. doi: 10.1182/blood.2022015797. | |
| 35120836 |
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Due to the short duration of time allowed to dose upon a VOC event, an optional screening may have occurred while the participant was well. Patients were neither enrolled nor randomized to study treatment until a VOC occurred, at which time the formal assessment of eligibility for enrollment into the study was performed. Of the 475 screened participants, 345 participants experiencing an acute VOC event were randomized.
This was a randomized, blinded study to treat subjects >=6 years of age with SCD, experiencing an acute VOC event requiring hospitalization. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population in alignment with the primary study objective.
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| ID | Title | Description |
|---|---|---|
| FG000 | Rivipansel Treatment Arm | Participants with sickle cell disease (SCD) received intravenous (IV) infusion of rivipansel while hospitalized for treatment of a vaso-occlusive crisis (VOC). Participants aged >=12 years, with body weight >40 kilogram (kg) received a loading dose of 1680 milligram (mg) rivipansel on Day 1 followed by maintenance doses of 840 mg rivipansel administered at 12 hourly intervals. Participants aged 6 to 11 years or any participant weighing <=40 kg received a loading dose of 40 milligram per kilogram body weight (mg/kg) rivipansel on Day 1 (maximum of 1680 mg) followed by maintenance doses of 20 mg/kg rivipansel (maximum of 840 mg) administered at 12 hourly intervals. Participants received rivipansel until they met the protocol-defined criteria for readiness-for-discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 1, 2019 | Feb 14, 2025 |
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|
|
| Placebo | Other | Placebo (phosphate buffered saline) will be infused intravenously every 12 hours up to 15 doses maximum. |
|
|
| Cumulative Intravenous (IV) Opioids Consumption From Time of Loading Dose of Study Drug to Discharge From Hospital | Cumulative IV opioid consumption was reported as cumulative IV opioid use (standardized using morphine equivalent units [MEU]), from the start of the first infusion (loading dose) of study drug until hospital discharge. | Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8) |
| Time to Discontinuation of Intravenous (IV) Opioids | Time to discontinuation of IV opioids was defined as the difference (in hours) between the stop time and date of the latest IV opioid dose and the start time and date of the first infusion (loading dose) of study drug. | Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8) |
| Cumulative Intravenous (IV) Opioids Consumption Within First 24 Hours Post-Loading Dose of Study Drug | Cumulative IV opioid consumption (standardized using MEU) was reported as IV opioid use in the first 24 hours from the start time of the first IV infusion (loading dose) of study drug. | 24 hours post first IV infusion of loading dose of study drug on Day 1 |
| Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 3 Days of Discharge From Hospital | Percentage of participants who were re-hospitalized for a vaso-occlusive crisis (VOC) within 3 days of discharge from hospital are reported. | Within 3 days of discharge from hospital, where discharge from hospital was any day from Day 1 to an average of Day 8 |
| Day 1 up to the 35-day post discharge visit (up to an average of Day 43) |
| Number of Participants With Treatment Emergent Adverse Event (AEs) as Per Severity | AE: any untoward medical occurrence in a participant who received investigational product without regard to possibility of a causal relationship. AEs were classified according to the severity in 3 categories a) mild - AEs did not interfere with participant's usual function, b) moderate - AEs interfered to some extent with participant's usual function, c) severe - AEs interfered significantly with participant's usual function. | Day 1 up to the 35-day post discharge visit (up to an average of Day 43) |
| Number of Participants With Clinical Laboratory Abnormalities | Hematology: hemoglobin, hematocrit, erythrocytes <0.8*lower limit of normal (LLN), reticulocytes <0.5*LLN >1.5*ULN, platelets<0.5*LLN>1.75*upper limit of normal (ULN), reticulocytes/erythrocytes<0.5*LLN>1.5*ULN, leukocytes <0.6*LLN >1.5*ULN, lymphocytes, lymphocytes/leukocytes, neutrophils, neutrophils/leukocytes <0.8*LLN >1.2*ULN, basophils, basophils/leukocytes, eosinophils, eosinophils/leukocytes, monocytes monocytes/leukocytes >1.2*ULN. Clinical chemistry: bilirubin, direct, bilirubin, indirect bilirubin>1.5*ULN, aspartate aminotransferase (AT), alanine AT, lactate dehydrogenase, alkaline phosphatase>3.0*ULN, urea nitrogen, urea, creatinine >1.3*ULN, sodium<0.95*LLN>1.05*ULN, potassium, chloride, bicarbonate<0.9*LLN>1.1*ULN, glucose<0.6*LLN>1.5*ULN, estimated glomerular filtration rate <=60. Urinalysis: urine glucose, ketones, urine protein, urine hemoglobin, nitrite, leukocyte esterase >=1. | Day 1 up to the 35-day post discharge visit (up to an average of Day 43) |
| Number of Participants With Increase in Grades From Baseline in Clinical Laboratory Parameters Over the Study | Hemoglobin(grade [G] 0:>11g/dl,G1:>9-11g/dl,G2:>7-9g/dl,G3:5-7g/dl,G4:<5g/dl),reticulocytes count(G0:<1%,G1:1-5%,G2:>5-10%,G3:>10-20%,G4:>20%),leukocytes(G0:<=upper limit of normal [ULN],G1:>ULN-15,000/mm^3,G2:>15,000- 20,000/mm^3,G3:>20,000- 50,000/mm^, G4:>50,000/mm^3),neutrophils(G0:>=LLN, G1:<LLN-1,500/mm^3, G2:<1,500-1,000/mm^3, G3:<1,000-500/mm^3, G4:<500/mm^3),blood urea nitrogen, creatinine(G0:<=ULN, G1:>ULN-1.5*UL, G2:>1.5-3.0*ULN, G3:>3.0*ULN, G4:>6.0*ULN), lactate dehydrogenase, alanine transaminase, aspartate aminotransferase(G0:<=ULN, G1:>ULN-3.0*ULN, G2:>3.0-5.0*ULN, G3:>5.0-20.0*ULN, G4:>20.0*ULN), bilirubin(G0:<=ULN, G1:>ULN-1.5*ULN, G2:>1.5-3.0*ULN, G3:>3.0-10.0*ULN, G4:>10.0*ULN), urine protein(G0:0-15mg/dL, G1:>15-30mg/dL, G2:>30-100mg/dL, G3:>100-300mg/dL, G4:>300mg/dL), platelet(G0:>=150K, G1:>=100K-<150K, G2:>=50K <100K, G3:<50K), eGFR (G0:>=90 mL/min/1.73m^2, G1:>=60-<90mL/min/1.73m^2, G2:>=30-<60mL/min/1.73m^2, G3:>=15-<30mL/min/1.73m^2, G4:<15 mL/min/1.73m^2) | Baseline up to the 35-day post discharge visit (up to an average of Day 43) |
| Number of Participants With Clinically Significant Changes in Physical Examination | Physical examination included assessment of the general appearance and the skin, head, ears, eyes, nose, mouth, throat, spine, neck, thyroid, chest, extremities, lymph nodes and abdomen (including liver and kidneys) plus the respiratory, cardiovascular, musculoskeletal, neurological and genitourinary systems. Clinical significance was assessed by the Investigator. | From Post-screening up to end of treatment (up to an average of Day 8), From Post-discharge up to 35 days post-discharge (up to an average of Day 43) |
| Number of Participants With Treatment Related Changes From Baseline in Vital Signs Over the Study | Vital signs included temperature, respiratory rate, pulse rate and systolic and diastolic blood pressure. Relatedness to treatment was assessed by the Investigator. | Baseline (Day 1) up to the 35-day post discharge visit (up to an average of Day 43) |
| Percentage of Participants With Adjudicated Acute Chest Syndrome (ACS) | Investigator reported events of Acute Chest Syndrome (ACS) and other reported respiratory events were sent for adjudication by the Acute Chest Syndrome Safety Endpoint Adjudication Committee. The committee, which consisted of physicians with relevant SCD expertise, evaluated these events and determined whether they were consistent with cases of ACS. | Day 1 up to the 35-day post discharge visit (up to an average of Day 43) |
| Percentage of Participants With Severe Adjudicated and/or Generalized Cutaneous Manifestations | Investigator reported cutaneous events were sent for adjudication by the Cutaneous Manifestations Safety Endpoint Adjudication Committee. The committee, which consisted of dermatologists, evaluated these events and determined whether they were cases of severe and/or generalized cutaneous manifestations and specifically whether any event was consistent with Acute Generalized Exanthematous Pustulosis. | Day 1 up to the 35-day post discharge visit (up to an average of Day 43) |
| Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 7, 14 and 30 Days of Discharge From Hospital | Percentage of participants re-hospitalized for VOC within 7, 14 and 30 days of hospital discharge. | Within 7 days of discharge; Within 14 days of discharge; Within 30 days of discharge, where discharge from hospital was any day from Day 1 to an average of Day 8 |
| Arkansas Children's Hospital Research Pharmacy |
| Little Rock |
| Arkansas |
| 72202 |
| United States |
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States |
| UCSF Benioff Children's Hospital Oakland | Oakland | California | 94609 | United States |
| UC Davis Medical Center Main Hospital | Sacramento | California | 95817 | United States |
| UC Davis Medical Center | Sacramento | California | 95817 | United States |
| University of California Davis Medical Center | Sacramento | California | 95817 | United States |
| University of Colorado CTRC | Aurora | Colorado | 80045 | United States |
| University of Colorado Hospital | Aurora | Colorado | 80045 | United States |
| Howard University Center for Sickle Cell Disease | Washington D.C. | District of Columbia | 20001 | United States |
| Medstar Health Research Institute | Washington D.C. | District of Columbia | 20010 | United States |
| Howard University Hospital | Washington D.C. | District of Columbia | 20060 | United States |
| Golisano Children's Hospital of Southwest Florida | Fort Myers | Florida | 33908 | United States |
| UF Health Davis Cancer Pavillion and Shands Med Plaza | Gainesville | Florida | 32608 | United States |
| UF Health Shands Cancer Hospital | Gainesville | Florida | 32608 | United States |
| UF Health Shands Hospital | Gainesville | Florida | 32610 | United States |
| Jackson Memorial Hospital | Miami | Florida | 33136 | United States |
| University Of Miami | Miami | Florida | 33136 | United States |
| Investigational Drug Service Tampa General Hospital | Tampa | Florida | 33606 | United States |
| Tampa General Hospital Center of Research Excellence | Tampa | Florida | 33606 | United States |
| Tampa General Hospital | Tampa | Florida | 33606 | United States |
| University of South Florida | Tampa | Florida | 33606 | United States |
| St. Joseph's Children's Hospital | Tampa | Florida | 33607 | United States |
| Children's Hematology and Oncology Associates | West Palm Beach | Florida | 33407 | United States |
| St. Mary's Medical Center | West Palm Beach | Florida | 33407 | United States |
| Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center | Atlanta | Georgia | 30303 | United States |
| Grady Health System | Atlanta | Georgia | 30303 | United States |
| Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center | Atlanta | Georgia | 30322 | United States |
| Emory Children's Center | Atlanta | Georgia | 30322 | United States |
| Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center | Atlanta | Georgia | 30342 | United States |
| Children's Healthcare of Atlanta: Scottish Rite Campus | Atlanta | Georgia | 30342 | United States |
| Augusta University Clinical Research Pharmacy | Augusta | Georgia | 30912 | United States |
| Augusta University Medical Center | Augusta | Georgia | 30912 | United States |
| Augusta University | Augusta | Georgia | 30912 | United States |
| Memorial Family Medicine Ctr. @Memorial Health Univ Med Ct | Savannah | Georgia | 31404 | United States |
| Memorial Health University Medical Center | Savannah | Georgia | 31404 | United States |
| University of Illinois at Chicago Clinical Research Center | Chicago | Illinois | 60612 | United States |
| University of Illinois Hospital and Health Sciences System | Chicago | Illinois | 60612 | United States |
| The University of Chicago/Comer Children's Hospital | Chicago | Illinois | 60637 | United States |
| University of Chicago, Investigational Drug Service Pharmacy | Chicago | Illinois | 60637 | United States |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | United States |
| Kosair Charities Pediatric Clinical Research Unit | Louisville | Kentucky | 40202 | United States |
| Norton Children´s Hospital | Louisville | Kentucky | 40202 | United States |
| The Novak Center for Children's Health | Louisville | Kentucky | 40202 | United States |
| University of Louisville Health Sciences Center | Louisville | Kentucky | 40202 | United States |
| University of Louisville Physicians Pediatric Hematology Oncology | Louisville | Kentucky | 40202 | United States |
| Our Lady of the Lake Regional Medical Center | Baton Rouge | Louisiana | 70808 | United States |
| St. Jude Affiliate Clinic | Baton Rouge | Louisiana | 70808 | United States |
| Our Lady of the Lake Physician Group-Medical Oncology | Baton Rouge | Louisiana | 70809 | United States |
| University of Maryland Medical System Investigational Pharmacy | Baltimore | Maryland | 21201 | United States |
| University of Maryland Medical System | Baltimore | Maryland | 21201 | United States |
| Johns Hopkins Medicine | Baltimore | Maryland | 21205 | United States |
| The Johns Hopkins University School of Medicine | Baltimore | Maryland | 21205 | United States |
| The Johns Hopkins Hospital | Baltimore | Maryland | 21287-6180 | United States |
| Johns Hopkins Medicine | Baltimore | Maryland | 21287 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Brigham and Womens Hospital | Boston | Massachusetts | 02115 | United States |
| Center for Clinical Investigation, Brigham & Women's Hospital | Boston | Massachusetts | 02115 | United States |
| Investigational Drug Services | Boston | Massachusetts | 02115 | United States |
| Boston Medical Center E7E | Boston | Massachusetts | 02118 | United States |
| Boston Medical Center | Boston | Massachusetts | 02118 | United States |
| Boston University Medical Center | Boston | Massachusetts | 02118 | United States |
| Children's Hospital of Michigan | Detroit | Michigan | 48201 | United States |
| Detroit Medical Center Pharmacy | Detroit | Michigan | 48201 | United States |
| Wayne State University / Detroit Receiving Hospital | Detroit | Michigan | 48201 | United States |
| University of Mississippi Medical Center-Outpatient Clinical Research Unit | Jackson | Mississippi | 39216 | United States |
| University Of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| Center for Outpatient Health | St Louis | Missouri | 63108 | United States |
| Barnes-Jewish Hospital Department of Pharmacy | St Louis | Missouri | 63110 | United States |
| Barnes-Jewish Hospital | St Louis | Missouri | 63110 | United States |
| Center for Advanced Medicine | St Louis | Missouri | 63110 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Bristol Myers Squibb Children's Hospital at Robert Wood Johnson University Hospital | New Brunswick | New Jersey | 08901 | United States |
| Rutgers-Robert Wood Johnson Medical School | New Brunswick | New Jersey | 08901 | United States |
| Rutgers Cancer Institute Of New Jersey | New Brunswick | New Jersey | 08903 | United States |
| Kings County Hospital Center - Pharmacy Department | Brooklyn | New York | 11203 | United States |
| Kings County Hospital Center | Brooklyn | New York | 11203 | United States |
| State University of New York (SUNY) - Downstate Medical Center | Brooklyn | New York | 11203 | United States |
| SUNY Downstate Medical Center University Hospital of Brooklyn | Brooklyn | New York | 11203 | United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| Columbia University Medical Center Research Pharmacy | New York | New York | 10032 | United States |
| MS CHONY Pediatric Emergency Department | New York | New York | 10032 | United States |
| MS CHONY Pediatric Hematology/Oncology Unit | New York | New York | 10032 | United States |
| Staten Island University Hospital | Staten Island | New York | 10305 | United States |
| Jacobi Medical Center | The Bronx | New York | 10461 | United States |
| Duke University Hospital | Durham | North Carolina | 27710 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| East Carolina University Brody School of Medicine | Greenville | North Carolina | 27834 | United States |
| Leo W. Jenkins Cancer Center | Greenville | North Carolina | 27834 | United States |
| Vidant Medical Center | Greenville | North Carolina | 27834 | United States |
| University of Cincinnati Medical Center/ Investigational Pharmacy | Cincinnati | Ohio | 45219 | United States |
| University of Cincinnati Medical Center/Research Office | Cincinnati | Ohio | 45219 | United States |
| University of Cincinnati Medical Center | Cincinnati | Ohio | 45219 | United States |
| University of Cincinnati Physicians Company LLC | Cincinnati | Ohio | 45219 | United States |
| University of Cincinnati- Hoxworth Building | Cincinnati | Ohio | 45219 | United States |
| UC Health Ridgeway Hospital | Cincinnati | Ohio | 45229 | United States |
| The Ohio State University Wexner Medical Center East | Columbus | Ohio | 43203 | United States |
| Nationwide Childrens Hospital | Columbus | Ohio | 43205 | United States |
| The Ohio State University Investigational Drug Services | Columbus | Ohio | 43210 | United States |
| The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute | Columbus | Ohio | 43210 | United States |
| The Ohio State University Wexner Medical Center | Columbus | Ohio | 43210 | United States |
| Five Rivers Medical Surgical Health Center | Dayton | Ohio | 45402 | United States |
| Miami Valley Hospital | Dayton | Ohio | 45409 | United States |
| Einstein Medical Center Philadelphia | Philadelphia | Pennsylvania | 19141 | United States |
| UPMC Presbyterian | Pittsburgh | Pennsylvania | 15213 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| UPMC Hillman Cancer Center | Pittsburgh | Pennsylvania | 15232 | United States |
| Hasbro Children's Hospital | Providence | Rhode Island | 02903 | United States |
| Rhode Island Hospital-Pharmacy Service | Providence | Rhode Island | 02903 | United States |
| Rhode Island Hospital | Providence | Rhode Island | 02903 | United States |
| The Miriam Hospital | Providence | Rhode Island | 02906 | United States |
| Medical University of South Carolina - Hospital | Charleston | South Carolina | 29425 | United States |
| Medical University of South Carolina Lifespan Comprehensive Sickle Cell Center | Charleston | South Carolina | 29425 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| MUSC Investigational Drug Services | Charleston | South Carolina | 29425 | United States |
| Cook Children's Hematology and Oncology Center | Fort Worth | Texas | 76104 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| Cook Children's Hematology and Oncology Center-Grapevine | Grapevine | Texas | 76051 | United States |
| Texas Children's Hospital - Clinical Research Center | Houston | Texas | 77030 | United States |
| Texas Children´s Hospital | Houston | Texas | 77030 | United States |
| University of Texas Medical School | Houston | Texas | 77030 | United States |
| Primary Children's Hospital | Salt Lake City | Utah | 84113 | United States |
| Main Hospital - Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| Virginia Commonwealth University-Investigational Drug Services | Richmond | Virginia | 23298 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| Royal Alexandra Hospital | Edmonton | Alberta | T5H 3V9 | Canada |
| Miseracordia Community Hospital | Edmonton | Alberta | T5R 4H5 | Canada |
| Kaye Edmonton Clinic 3 C | Edmonton | Alberta | T6G 1Z1 | Canada |
| University of Alberta Hospital, Pharmacy Services | Edmonton | Alberta | T6G 1Z1 | Canada |
| Stollery Children's Hospital | Edmonton | Alberta | T6G 2B7 | Canada |
| University of Alberta Hospital | Edmonton | Alberta | T6G 2B7 | Canada |
| Research transition Facility | Edmonton | Alberta | T6G 2V2 | Canada |
| Grey Nuns Community Hospital | Edmonton | Alberta | T6L 5X8 | Canada |
| St. Paul's Hospital | Vancouver | British Columbia | V6E 1M7 | Canada |
| St. Paul's Hospital | Vancouver | British Columbia | V6Z 1Y6 | Canada |
| Children's Hospital Of Eastern Ontario | Ottawa | Ontario | K1H 8L1 | Canada |
| University Health Network, Toronto General Hospital | Toronto | Ontario | M5G 2C4 | Canada |
| The Hospital for Sick Children | Toronto | Ontario | M5G1X8 | Canada |
| Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec | H3T 1C5 | Canada |
| McGill University Health Centre, Royal Victoria Hospital | Montreal | Quebec | H4A 3J1 | Canada |
| The Montreal Children's Hospital/ McGill University Health Centre | Montreal | Quebec | H4A 3J1 | Canada |
| Derived |
| Rebelo AL, Chevalier MT, Russo L, Pandit A. Role and therapeutic implications of protein glycosylation in neuroinflammation. Trends Mol Med. 2022 Apr;28(4):270-289. doi: 10.1016/j.molmed.2022.01.004. Epub 2022 Feb 1. |
| FG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
| Treated |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Full analysis set population included all participants who were randomized in the study. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population in alignment with the primary study objective.
Full analysis set population included all participants who were randomized in the study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Rivipansel Treatment Arm | Participants with sickle cell disease (SCD) received intravenous (IV) infusion of rivipansel while hospitalized for treatment of a vaso-occlusive crisis (VOC). Participants aged >=12 years, with body weight >40 kilogram (kg) received a loading dose of 1680 milligram (mg) rivipansel on Day 1 followed by maintenance doses of 840 mg rivipansel administered at 12 hourly intervals. Participants aged 6 to 11 years or any participant weighing <=40 kg received a loading dose of 40 milligram per kilogram body weight (mg/kg) rivipansel on Day 1 (maximum of 1680 mg) followed by maintenance doses of 20 mg/kg rivipansel (maximum of 840 mg) administered at 12 hourly intervals. Participants received rivipansel until they met the protocol-defined criteria for readiness-for-discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days. |
| BG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
| 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, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | 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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Time to Readiness for Discharge From Hospital | Time to readiness-for-discharge from hospital was defined as the difference (in hours) between the time and date when all criteria for readiness-for-discharge were met and the start time and date of the first infusion (loading dose) of study drug. Criteria for readiness-for-discharge were met when all of the applicable 6 criteria (in relation to treatment of VOC and complications related to the VOC) were documented to have occurred. The six criteria were: 1) only oral pain medication was required, 2) acute complications related to the VOC (such as acute chest syndrome, stroke, priapism) had resolved to the extent that management could be in an outpatient setting, 3) IV opioids had been discontinued, 4) IV hydration had been discontinued, 5) IV antibiotics had been discontinued and 6) red blood cell (RBC) transfusion was no longer required for treatment of this VOC. | Full analysis set population included all participants who were randomized in the study. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population in alignment with the primary study objective. | Posted | Median | 95% Confidence Interval | Hours | Day 1 up to the latest day when all 6 criteria of readiness-for-discharge were met (up to an average of Day 8) |
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| Secondary | Time to Discharge From Hospital | Time to discharge from hospital was defined as the difference (in hours) between the time and date of the hospital discharge order from a qualified healthcare provider and the start time and date of the first infusion (loading dose) of study drug. | Full analysis set population included all participants who were randomized in the study. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population in alignment with the primary study objective. | Posted | Median | 95% Confidence Interval | Hours | Day 1 up to the latest day when the order of hospital discharge was issued by a qualified healthcare provider (up to an average of Day 8) |
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| Secondary | Cumulative Intravenous (IV) Opioids Consumption From Time of Loading Dose of Study Drug to Discharge From Hospital | Cumulative IV opioid consumption was reported as cumulative IV opioid use (standardized using morphine equivalent units [MEU]), from the start of the first infusion (loading dose) of study drug until hospital discharge. | Full analysis set population included all participants who were randomized in the study. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population in alignment with the primary study objective. | Posted | Median | Inter-Quartile Range | MEU per kg | Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8) |
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| Secondary | Time to Discontinuation of Intravenous (IV) Opioids | Time to discontinuation of IV opioids was defined as the difference (in hours) between the stop time and date of the latest IV opioid dose and the start time and date of the first infusion (loading dose) of study drug. | Full analysis set population included all participants who were randomized in the study. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population in alignment with the primary study objective. | Posted | Median | 95% Confidence Interval | Hours | Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8) |
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| Secondary | Cumulative Intravenous (IV) Opioids Consumption Within First 24 Hours Post-Loading Dose of Study Drug | Cumulative IV opioid consumption (standardized using MEU) was reported as IV opioid use in the first 24 hours from the start time of the first IV infusion (loading dose) of study drug. | Full analysis set population included all participants who were randomized in the study. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population in alignment with the primary study objective. | Posted | Median | Inter-Quartile Range | MEU per kg | 24 hours post first IV infusion of loading dose of study drug on Day 1 |
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| Secondary | Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 3 Days of Discharge From Hospital | Percentage of participants who were re-hospitalized for a vaso-occlusive crisis (VOC) within 3 days of discharge from hospital are reported. | Full analysis set population included all participants who were randomized in the study. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population in alignment with the primary study objective. | Posted | Count of Participants | Participants | Within 3 days of discharge from hospital, where discharge from hospital was any day from Day 1 to an average of Day 8 |
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| Other Pre-specified | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) as Per Genotype | AE: any untoward medical occurrence in a participant who received investigational product without regard to possibility of a causal relationship. SAE: an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; medically important events. An adverse event was considered a treatment-emergent adverse event (TEAE) if the event started during the effective duration of treatment (all events that started on or after the first dosing). AEs included both serious and non-serious adverse events. Participants with AEs and SAEs were categorized by genotype categories. Category 1= participants with hemoglobin SS, hemoglobin S beta0 thalassemia and hemoglobin SD; category 2= participants with hemoglobin SC, hemoglobin S beta+ thalassemia and hemoglobin S-Variant (other than HbSD). | Safety analysis set population included all participants who received at least 1 infusion of study drug. Here, "Number Analyzed" signifies number of participants evaluable for specified rows. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population. | Posted | Count of Participants | Participants | No | Day 1 up to the 35-day post discharge visit (up to an average of Day 43) |
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| Other Pre-specified | Number of Participants With Treatment Emergent Adverse Event (AEs) as Per Severity | AE: any untoward medical occurrence in a participant who received investigational product without regard to possibility of a causal relationship. AEs were classified according to the severity in 3 categories a) mild - AEs did not interfere with participant's usual function, b) moderate - AEs interfered to some extent with participant's usual function, c) severe - AEs interfered significantly with participant's usual function. | Safety analysis set population included all participants who received at least 1 infusion of study drug. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population. | Posted | Count of Participants | Participants | No | Day 1 up to the 35-day post discharge visit (up to an average of Day 43) |
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| Other Pre-specified | Number of Participants With Clinical Laboratory Abnormalities | Hematology: hemoglobin, hematocrit, erythrocytes <0.8*lower limit of normal (LLN), reticulocytes <0.5*LLN >1.5*ULN, platelets<0.5*LLN>1.75*upper limit of normal (ULN), reticulocytes/erythrocytes<0.5*LLN>1.5*ULN, leukocytes <0.6*LLN >1.5*ULN, lymphocytes, lymphocytes/leukocytes, neutrophils, neutrophils/leukocytes <0.8*LLN >1.2*ULN, basophils, basophils/leukocytes, eosinophils, eosinophils/leukocytes, monocytes monocytes/leukocytes >1.2*ULN. Clinical chemistry: bilirubin, direct, bilirubin, indirect bilirubin>1.5*ULN, aspartate aminotransferase (AT), alanine AT, lactate dehydrogenase, alkaline phosphatase>3.0*ULN, urea nitrogen, urea, creatinine >1.3*ULN, sodium<0.95*LLN>1.05*ULN, potassium, chloride, bicarbonate<0.9*LLN>1.1*ULN, glucose<0.6*LLN>1.5*ULN, estimated glomerular filtration rate <=60. Urinalysis: urine glucose, ketones, urine protein, urine hemoglobin, nitrite, leukocyte esterase >=1. | Safety analysis set population included all participants who received at least 1 infusion of study drug. Here 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population. | Posted | Count of Participants | Participants | No | Day 1 up to the 35-day post discharge visit (up to an average of Day 43) |
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| Other Pre-specified | Number of Participants With Increase in Grades From Baseline in Clinical Laboratory Parameters Over the Study | Hemoglobin(grade [G] 0:>11g/dl,G1:>9-11g/dl,G2:>7-9g/dl,G3:5-7g/dl,G4:<5g/dl),reticulocytes count(G0:<1%,G1:1-5%,G2:>5-10%,G3:>10-20%,G4:>20%),leukocytes(G0:<=upper limit of normal [ULN],G1:>ULN-15,000/mm^3,G2:>15,000- 20,000/mm^3,G3:>20,000- 50,000/mm^, G4:>50,000/mm^3),neutrophils(G0:>=LLN, G1:<LLN-1,500/mm^3, G2:<1,500-1,000/mm^3, G3:<1,000-500/mm^3, G4:<500/mm^3),blood urea nitrogen, creatinine(G0:<=ULN, G1:>ULN-1.5*UL, G2:>1.5-3.0*ULN, G3:>3.0*ULN, G4:>6.0*ULN), lactate dehydrogenase, alanine transaminase, aspartate aminotransferase(G0:<=ULN, G1:>ULN-3.0*ULN, G2:>3.0-5.0*ULN, G3:>5.0-20.0*ULN, G4:>20.0*ULN), bilirubin(G0:<=ULN, G1:>ULN-1.5*ULN, G2:>1.5-3.0*ULN, G3:>3.0-10.0*ULN, G4:>10.0*ULN), urine protein(G0:0-15mg/dL, G1:>15-30mg/dL, G2:>30-100mg/dL, G3:>100-300mg/dL, G4:>300mg/dL), platelet(G0:>=150K, G1:>=100K-<150K, G2:>=50K <100K, G3:<50K), eGFR (G0:>=90 mL/min/1.73m^2, G1:>=60-<90mL/min/1.73m^2, G2:>=30-<60mL/min/1.73m^2, G3:>=15-<30mL/min/1.73m^2, G4:<15 mL/min/1.73m^2) | Safety analysis set population included all participants who received at least 1 infusion of drug. Overall Number of Participants Analyzed signifies participants evaluable for this outcome measure; Number Analyzed signifies participants evaluable for specific rows. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population. | Posted | Count of Participants | Participants | No | Baseline up to the 35-day post discharge visit (up to an average of Day 43) |
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| Other Pre-specified | Number of Participants With Clinically Significant Changes in Physical Examination | Physical examination included assessment of the general appearance and the skin, head, ears, eyes, nose, mouth, throat, spine, neck, thyroid, chest, extremities, lymph nodes and abdomen (including liver and kidneys) plus the respiratory, cardiovascular, musculoskeletal, neurological and genitourinary systems. Clinical significance was assessed by the Investigator. | Safety analysis set population included all participants who received at least 1 infusion of study drug. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population. | Posted | Count of Participants | Participants | No | From Post-screening up to end of treatment (up to an average of Day 8), From Post-discharge up to 35 days post-discharge (up to an average of Day 43) |
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| Other Pre-specified | Number of Participants With Treatment Related Changes From Baseline in Vital Signs Over the Study | Vital signs included temperature, respiratory rate, pulse rate and systolic and diastolic blood pressure. Relatedness to treatment was assessed by the Investigator. | Safety analysis set population included all participants who received at least 1 infusion of study drug. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population. | Posted | Count of Participants | Participants | No | Baseline (Day 1) up to the 35-day post discharge visit (up to an average of Day 43) |
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| Other Pre-specified | Percentage of Participants With Adjudicated Acute Chest Syndrome (ACS) | Investigator reported events of Acute Chest Syndrome (ACS) and other reported respiratory events were sent for adjudication by the Acute Chest Syndrome Safety Endpoint Adjudication Committee. The committee, which consisted of physicians with relevant SCD expertise, evaluated these events and determined whether they were consistent with cases of ACS. | Safety analysis set population included all participants who received at least 1 infusion of study drug. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population. | Posted | Number | Percentage of participants | Day 1 up to the 35-day post discharge visit (up to an average of Day 43) |
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| Other Pre-specified | Percentage of Participants With Severe Adjudicated and/or Generalized Cutaneous Manifestations | Investigator reported cutaneous events were sent for adjudication by the Cutaneous Manifestations Safety Endpoint Adjudication Committee. The committee, which consisted of dermatologists, evaluated these events and determined whether they were cases of severe and/or generalized cutaneous manifestations and specifically whether any event was consistent with Acute Generalized Exanthematous Pustulosis. | Safety analysis set population included all participants who received at least 1 infusion of study drug. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population. | Posted | Number | Percentage of participants | Day 1 up to the 35-day post discharge visit (up to an average of Day 43) |
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| Other Pre-specified | Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 7, 14 and 30 Days of Discharge From Hospital | Percentage of participants re-hospitalized for VOC within 7, 14 and 30 days of hospital discharge. | Safety analysis set population included all participants who received at least 1 infusion of study drug. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population. | Posted | Number | Percentage of participants | Within 7 days of discharge; Within 14 days of discharge; Within 30 days of discharge, where discharge from hospital was any day from Day 1 to an average of Day 8 |
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Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
Same event may appear as AE and SAE, what is presented are distinct events. Safety analysis set population included all participants who received at least one 1 infusion of study drug. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population.
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 | Rivipansel Treatment Arm | Participants with sickle cell disease (SCD) received intravenous (IV) infusion of rivipansel while hospitalized for treatment of a vaso-occlusive crisis (VOC). Participants aged >=12 years, with body weight >40 kilogram (kg) received a loading dose of 1680 milligram (mg) rivipansel on Day 1 followed by maintenance doses of 840 mg rivipansel administered at 12 hourly intervals. Participants aged 6 to 11 years or any participant weighing <=40 kg received a loading dose of 40 milligram per kilogram body weight (mg/kg) rivipansel on Day 1 (maximum of 1680 mg) followed by maintenance doses of 20 mg/kg rivipansel (maximum of 840 mg) administered at 12 hourly intervals. Participants received rivipansel until they met the protocol-defined criteria for readiness-for-discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days. | 0 | 162 | 52 | 162 | 137 | 162 |
| EG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days | 0 | 158 | 49 | 158 | 124 | 158 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA v22.0 | Non-systematic Assessment |
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| Thrombocytosis | Blood and lymphatic system disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Lip blister | Gastrointestinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Systemic inflammatory response syndrome | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Beta haemolytic streptococcal infection | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Parainfluenzae virus infection | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pilonidal cyst | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Thrombophlebitis septic | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Vascular device infection | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Delayed haemolytic transfusion reaction | Injury, poisoning and procedural complications | MedDRA v22.0 | Non-systematic Assessment |
| |
| Blood culture positive | Investigations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Chest X-ray abnormal | Investigations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Liver function test increased | Investigations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Fluid overload | Metabolism and nutrition disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Bone infarction | Musculoskeletal and connective tissue disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Hallucination | Psychiatric disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Priapism | Reproductive system and breast disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Acute generalised exanthematous pustulosis | Skin and subcutaneous tissue disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Swelling face | Skin and subcutaneous tissue disorders | MedDRA v22.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Sickle cell disease | Congenital, familial and genetic disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Chest pain | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Infusion site extravasation | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Ocular icterus | Hepatobiliary disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Haematocrit decreased | Investigations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA v22.0 | Non-systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA v22.0 | Non-systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA v22.0 | Non-systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Head of Regulatory | Biossil Inc. | 978-245-7397 | debora@biossil.ai |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 10, 2019 | Feb 14, 2025 | SAP_001.pdf |
| 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 |
|---|---|
| C553182 | rivipansel |
Not provided
Not provided
Not provided
| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Race: White |
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| Race: Other |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| Rivipansel Treatment Arm |
Participants with sickle cell disease (SCD) received intravenous (IV) infusion of rivipansel while hospitalized for treatment of a vaso-occlusive crisis (VOC). Participants aged >=12 years, with body weight >40 kilogram (kg) received a loading dose of 1680 milligram (mg) rivipansel on Day 1 followed by maintenance doses of 840 mg rivipansel administered at 12 hourly intervals. Participants aged 6 to 11 years or any participant weighing <=40 kg received a loading dose of 40 milligram per kilogram body weight (mg/kg) rivipansel on Day 1 (maximum of 1680 mg) followed by maintenance doses of 20 mg/kg rivipansel (maximum of 840 mg) administered at 12 hourly intervals. Participants received rivipansel until they met the protocol-defined criteria for readiness-for-discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days. |
| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| OG001 | Placebo Treatment Arm | Participants with SCD received IV infusion of placebo (matched to rivipansel infusion) while hospitalized for treatment of a VOC. Participants received placebo on the same schedule used for rivipansel until they met the protocol-defined criteria for readiness-for- discharge from hospital or up to a maximum of 15 doses (1 loading dose and 14 maintenance doses), whichever occurred first. Dosing therefore continued for a maximum of 8 days |
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| Genotype Category 2 |
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