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The purpose of this study is to evaluate whether MST-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease. The study will also evaluate whether MST-188 can reduce the frequency of rehospitalization of subjects due to a recurrence of VOC. Additionally, this study will compare the development of acute chest syndrome during VOC in subjects who receive MST-188 to those who do not receive MST-188.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MST-188 | Experimental | MST-188 injection administered as a continuous infusion 100 mg/kg for 1 hour followed by 30 mg/kg/hr for up to 48 hours. |
|
| Saline | Placebo Comparator | Saline administered as a continuous infusion for up to 49 hours |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Saline | Drug |
| ||
| MST-188 |
| Measure | Description | Time Frame |
|---|---|---|
| Reduction of the duration of vaso occlusive crisis (VOC) in subjects with sickle cell disease. | Study participants will be followed for the duration of hospital stay, an expected average of 4 days |
| Measure | Description | Time Frame |
|---|---|---|
| Re-hospitalization rate for VOC | Hospital discharge to 14 days post-discharge | |
| Occurence of acute chest syndrome | Randomization to 120 hours after randomization |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Edwin L. Parsley, D.O. | Mast Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of South Alabama | Mobile | Alabama | 36688 | United States | ||
| Phoenix Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33877274 | Derived | Casella JF, Barton BA, Kanter J, Black LV, Majumdar S, Inati A, Wali Y, Drachtman RA, Abboud MR, Kilinc Y, Fuh BR, Al-Khabori MK, Takemoto CM, Salman E, Sarnaik SA, Shah N, Morris CR, Keates-Baleeiro J, Raj A, Alvarez OA, Hsu LL, Thompson AA, Sisler IY, Pace BS, Noronha SA, Lasky JL 3rd, de Julian EC, Godder K, Thornburg CD, Kamberos NL, Nuss R, Marsh AM, Owen WC, Schaefer A, Tebbi CK, Chantrain CF, Cohen DE, Karakas Z, Piccone CM, George A, Fixler JM, Singleton TC, Moulton T, Quinn CT, de Castro Lobo CL, Almomen AM, Goyal-Khemka M, Maes P, Emanuele M, Gorney RT, Padgett CS, Parsley E, Kronsberg SS, Kato GJ, Gladwin MT. Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial. JAMA. 2021 Apr 20;325(15):1513-1523. doi: 10.1001/jama.2021.3414. |
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|
|
| Phoenix |
| Arizona |
| 85016 |
| United States |
| UCSF Benioff Children's Hospital | Oakland | California | 94609 | United States |
| University of California Davis Health System | Sacramento | California | 95817 | United States |
| Rady Children's Hosptial | San Diego | California | 92123 | United States |
| Harbor-UCLA Medical Center | Torrence | California | 90502 | United States |
| Al DuPont Hospital for Children | Wilmington | Delaware | 19803 | United States |
| Howard University | Washington D.C. | District of Columbia | 20060 | United States |
| Children's Hospital of SouthWest Florida | Fort Myers | Florida | 33908 | United States |
| Joe Dimaggio Children's Hospital | Hollywood | Florida | 33021 | United States |
| University of Miami | Miami | Florida | 33136 | United States |
| Miami Children's Hospital | Miami | Florida | 33155 | United States |
| All Children's Hospital | St. Petersburg | Florida | 33701 | United States |
| Tampa General Hospital | Tampa | Florida | 33606 | United States |
| Grady Memorial Hospital | Atlanta | Georgia | 30303 | United States |
| Georgia Regents University | Augusta | Georgia | 30912 | United States |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Children's Hospital at the University of Illinois | Chicago | Illinois | 60612 | United States |
| Fort Wayne Lutheran Hospital | Fort Wayne | Indiana | 46804 | United States |
| Riley Hospital for Children | Indianapolis | Indiana | 46202 | United States |
| University of Iowa-Children's Hospital | Iowa City | Iowa | 52242 | United States |
| University of Louisville/Kosair Children's Hospital | Louisville | Kentucky | 40202 | United States |
| Our Lady of the Lake Children's Hospital | Baton Rouge | Louisiana | 70808 | United States |
| Children's Hospital of New Orleans | New Orleans | Louisiana | 70118 | United States |
| Johns Hopkins | Baltimore | Maryland | 21205 | United States |
| The Herman and Walter Samuelson Children's Hospital at Sinai | Baltimore | Maryland | 21215 | United States |
| Children's Hospital of Michigan-Wayne State University | Detroit | Michigan | 48201 | United States |
| Hurley Research Center | Flint | Michigan | 48503 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| Rutger's University | New Brunswick | New Jersey | 08903 | United States |
| NY Methodist | Brooklyn | New York | 11215 | United States |
| Cohen Children's Medical Center | New Hyde Park | New York | 11040 | United States |
| Golisano Children's Hospital at URMC | Rochester | New York | 14642 | United States |
| Bronx Lebanon Hospital | The Bronx | New York | 10457 | United States |
| University of North Carolina-Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| East Carolina University | Greenville | North Carolina | 27834 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Rainbow Babies and Children's Hospital | Cleveland | Ohio | 44106 | United States |
| Randall Children's Hospital | Portland | Oregon | 97227 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15232 | United States |
| The Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| T.C. Thompson Children's Hospital | Chattanooga | Tennessee | 37403 | United States |
| Cook's Children Hospital | Fort Worth | Texas | 76104 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| University of Virginia Medical Center | Charlottsville | Virginia | 22908 | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | United States |
| Children's Hospital of Richmond | Richmond | Virginia | 23298 | United States |
| Reearch Center | Antwerp | Belgium |
| Research Center | Brussels | Belgium |
| Research Center | Edgem | Belgium |
| Research Center | Liège | Belgium |
| Research Center | Montegnée | Belgium |
| Resaerch Center | Rio de Janerio | Brazil |
| Research Center | São Paulo | Brazil |
| Research Center | Santo Domingo | 10101 | Dominican Republic |
| Research Center | Santo Domingo | 10514 | Dominican Republic |
| Research Center | Kingston | Jamaica |
| Research Center | Irbid | Jordan |
| Research Center | Beirut | Lebanon |
| Research Center | Tripoli | Lebanon |
| Research Center - Child Health Department | Muscat | Oman |
| Research Center | Muscat | Oman |
| Research Center Hospital del Nino | Panama City | Panama |
| Research Center Hospital of Pediatric Specialities | Panama City | Panama |
| Research Center Metropolitan Hospital | Panama City | Panama |
| Research Center | Madrid | Spain |
| Research Center | Adana | Turkey (Türkiye) |
| Research Center | Istanbul | Turkey (Türkiye) |
| Research Center | Mersin | Turkey (Türkiye) |
| ID | Term |
|---|---|
| D000098644 | Vaso-Occlusive Crises |
| D000755 | Anemia, Sickle Cell |
| 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 |
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| ID | Term |
|---|---|
| D012965 | Sodium Chloride |
| ID | Term |
|---|---|
| D002712 | Chlorides |
| D006851 | Hydrochloric Acid |
| D017606 | Chlorine Compounds |
| D007287 | Inorganic Chemicals |
| D017670 | Sodium Compounds |
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