Clinical trial NCT01737814: Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)
Official title: Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC): A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial of MST-188 (Purified Poloxamer 188) Injection in Subjects With Sickle Cell Disease Experiencing Vaso Occlusive Crisis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Vaso-occlusive Crisis
Sickle Cell Disease
Keywords: sickle cell disease
vaso-occlusive crisis
Lead sponsor: Mast Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2012-11-30
Enrollment: 388
Sex: ALL
Minimum age (years): 4
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* Age 4 through 65 years
* Subject has a confirmed diagnosis of HbSS, HbSC, HbSβ+thal, or HbSβ0thal
* Subject is experiencing acute pain typical of vaso-occlusive crisis requiring treatment with parenteral analgesia
* Subject requires hospitalization

Exclusion Criteria:

* Subject has acute chest syndrome
* Subject's laboratory results indicate inadequate organ function
* Subject is pregnant or nursing an infant
* Subject had a painful crisis requiring hospitalization within the preceding 14 days or has experienced \> 5 hospitalizations for VOC in the prior 6 months
* Subject has been transfused within the past 14 days
* Subject is hospitalized for a condition other than VOC
* Subject has complications related to SCD

Primary outcomes:
measure: Reduction of the duration of vaso occlusive crisis (VOC) in subjects with sickle cell disease.
timeFrame: Study participants will be followed for the duration of hospital stay, an expected average of 4 days
Secondary outcomes:
measure: Re-hospitalization rate for VOC
timeFrame: Hospital discharge to 14 days post-discharge
measure: Occurence of acute chest syndrome
timeFrame: Randomization to 120 hours after randomization
Other outcomes:
Locations:
city: Mobile
country: United States
facility: University of South Alabama
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital
state: California
city: Sacramento
country: United States
facility: University of California Davis Health System
state: California
city: San Diego
country: United States
facility: Rady Children's Hosptial
state: California
city: Torrence
country: United States
facility: Harbor-UCLA Medical Center
state: California
city: Wilmington
country: United States
facility: Al DuPont Hospital for Children
state: Delaware
city: Washington
country: United States
facility: Howard University
state: District of Columbia
city: Fort Myers
country: United States
facility: Children's Hospital of SouthWest Florida
state: Florida
city: Hollywood
country: United States
facility: Joe Dimaggio Children's Hospital
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: St. Petersburg
country: United States
facility: All Children's Hospital
state: Florida
city: Tampa
country: United States
facility: Tampa General Hospital
state: Florida
city: Atlanta
country: United States
facility: Grady Memorial Hospital
state: Georgia
city: Augusta
country: United States
facility: Georgia Regents University
state: Georgia
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Chicago
country: United States
facility: Children's Hospital at the University of Illinois
state: Illinois
city: Ft. Wayne
country: United States
facility: Fort Wayne Lutheran Hospital
state: Indiana
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa-Children's Hospital
state: Iowa
city: Louisville
country: United States
facility: University of Louisville/Kosair Children's Hospital
state: Kentucky
city: Baton Rouge
country: United States
facility: Our Lady of the Lake Children's Hospital
state: Louisiana
city: New Orleans
country: United States
facility: Children's Hospital of New Orleans
state: Louisiana
city: Baltimore
country: United States
facility: Johns Hopkins
state: Maryland
city: Baltimore
country: United States
facility: The Herman and Walter Samuelson Children's Hospital at Sinai
state: Maryland
city: Detroit
country: United States
facility: Children's Hospital of Michigan-Wayne State University
state: Michigan
city: Flint
country: United States
facility: Hurley Research Center
state: Michigan
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: New Brunswick
country: United States
facility: Rutger's University
state: New Jersey
city: Bronx
country: United States
facility: Bronx Lebanon Hospital
state: New York
city: Brooklyn
country: United States
facility: NY Methodist
state: New York
city: New Hyde Park
country: United States
facility: Cohen Children's Medical Center
state: New York
city: Rochester
country: United States
facility: Golisano Children's Hospital at URMC
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina-Chapel Hill
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Randall Children's Hospital
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Charleston
country: United States
facility: The Medical University of South Carolina
state: South Carolina
city: Chattanooga
country: United States
facility: T.C. Thompson Children's Hospital
state: Tennessee
city: Fort Worth
country: United States
facility: Cook's Children Hospital
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Charlottsville
country: United States
facility: University of Virginia Medical Center
state: Virginia
city: Norfolk
country: United States
facility: Children's Hospital of the King's Daughters
state: Virginia
city: Richmond
country: United States
facility: Children's Hospital of Richmond
state: Virginia
city: Antwerp
country: Belgium
facility: Reearch Center
city: Brussels
country: Belgium
facility: Research Center
city: Edgem
country: Belgium
facility: Research Center
city: Liege
country: Belgium
facility: Research Center
city: Montegnee
country: Belgium
facility: Research Center
city: Rio de Janerio
country: Brazil
facility: Resaerch Center
city: Sao Paulo
country: Brazil
facility: Research Center
city: Santo Domingo
country: Dominican Republic
facility: Research Center
city: Santo Domingo
country: Dominican Republic
facility: Research Center
city: Kingston
country: Jamaica
facility: Research Center
city: Irbid
country: Jordan
facility: Research Center
city: Beirut
country: Lebanon
facility: Research Center
city: Tripoli
country: Lebanon
facility: Research Center
city: Muscat
country: Oman
facility: Research Center - Child Health Department
city: Muscat
country: Oman
facility: Research Center
city: Panama
country: Panama
facility: Research Center Hospital del Nino
city: Panama
country: Panama
facility: Research Center Hospital of Pediatric Specialities
city: Panama
country: Panama
facility: Research Center Metropolitan Hospital
city: Madrid
country: Spain
facility: Research Center
city: Adana
country: Turkey
facility: Research Center
city: Istanbul
country: Turkey
facility: Research Center
city: Mersin
country: Turkey
facility: Research Center
Study officials:
affiliation: Mast Therapeutics, Inc.
name: Edwin L. Parsley, D.O.
role: STUDY_DIRECTOR
References:
citation: 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.