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Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC) (EPIC)

Primary Purpose

Vaso-occlusive Crisis, Sickle Cell Disease

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Saline
MST-188
Sponsored by
Mast Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Vaso-occlusive Crisis focused on measuring sickle cell disease, vaso-occlusive crisis

Eligibility Criteria

4 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of South Alabama
  • Phoenix Children's Hospital
  • UCSF Benioff Children's Hospital
  • University of California Davis Health System
  • Rady Children's Hosptial
  • Harbor-UCLA Medical Center
  • Al DuPont Hospital for Children
  • Howard University
  • Children's Hospital of SouthWest Florida
  • Joe Dimaggio Children's Hospital
  • University of Miami
  • Miami Children's Hospital
  • All Children's Hospital
  • Tampa General Hospital
  • Grady Memorial Hospital
  • Georgia Regents University
  • Ann and Robert H. Lurie Children's Hospital of Chicago
  • Children's Hospital at the University of Illinois
  • Fort Wayne Lutheran Hospital
  • Riley Hospital for Children
  • University of Iowa-Children's Hospital
  • University of Louisville/Kosair Children's Hospital
  • Our Lady of the Lake Children's Hospital
  • Children's Hospital of New Orleans
  • Johns Hopkins
  • The Herman and Walter Samuelson Children's Hospital at Sinai
  • Children's Hospital of Michigan-Wayne State University
  • Hurley Research Center
  • University of Mississippi Medical Center
  • Rutger's University
  • Bronx Lebanon Hospital
  • NY Methodist
  • Cohen Children's Medical Center
  • Golisano Children's Hospital at URMC
  • University of North Carolina-Chapel Hill
  • Duke University Medical Center
  • East Carolina University
  • Cincinnati Children's Hospital Medical Center
  • Rainbow Babies and Children's Hospital
  • Randall Children's Hospital
  • Children's Hospital of Philadelphia
  • Children's Hospital of Pittsburgh
  • University of Pittsburgh Medical Center
  • The Medical University of South Carolina
  • T.C. Thompson Children's Hospital
  • Cook's Children Hospital
  • Texas Children's Hospital
  • University of Virginia Medical Center
  • Children's Hospital of the King's Daughters
  • Children's Hospital of Richmond
  • Reearch Center
  • Research Center
  • Research Center
  • Research Center
  • Research Center
  • Resaerch Center
  • Research Center
  • Research Center
  • Research Center
  • Research Center
  • Research Center
  • Research Center
  • Research Center
  • Research Center - Child Health Department
  • Research Center
  • Research Center Hospital del Nino
  • Research Center Hospital of Pediatric Specialities
  • Research Center Metropolitan Hospital
  • Research Center
  • Research Center
  • Research Center
  • Research Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

MST-188

Saline

Arm Description

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 administered as a continuous infusion for up to 49 hours

Outcomes

Primary Outcome Measures

Reduction of the duration of vaso occlusive crisis (VOC) in subjects with sickle cell disease.

Secondary Outcome Measures

Re-hospitalization rate for VOC
Occurence of acute chest syndrome

Full Information

First Posted
November 27, 2012
Last Updated
October 26, 2016
Sponsor
Mast Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01737814
Brief Title
Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)
Acronym
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

2. Study Status

Record Verification Date
October 2016
Overall Recruitment Status
Completed
Study Start Date
May 2013 (undefined)
Primary Completion Date
February 2016 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mast Therapeutics, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Vaso-occlusive Crisis, Sickle Cell Disease
Keywords
sickle cell disease, vaso-occlusive crisis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
388 (Actual)

8. Arms, Groups, and Interventions

Arm Title
MST-188
Arm Type
Experimental
Arm Description
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.
Arm Title
Saline
Arm Type
Placebo Comparator
Arm Description
Saline administered as a continuous infusion for up to 49 hours
Intervention Type
Drug
Intervention Name(s)
Saline
Intervention Type
Drug
Intervention Name(s)
MST-188
Other Intervention Name(s)
vepoloxamer
Primary Outcome Measure Information:
Title
Reduction of the duration of vaso occlusive crisis (VOC) in subjects with sickle cell disease.
Time Frame
Study participants will be followed for the duration of hospital stay, an expected average of 4 days
Secondary Outcome Measure Information:
Title
Re-hospitalization rate for VOC
Time Frame
Hospital discharge to 14 days post-discharge
Title
Occurence of acute chest syndrome
Time Frame
Randomization to 120 hours after randomization

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Edwin L. Parsley, D.O.
Organizational Affiliation
Mast Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of South Alabama
City
Mobile
State/Province
Alabama
ZIP/Postal Code
36688
Country
United States
Facility Name
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
UCSF Benioff Children's Hospital
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
University of California Davis Health System
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Rady Children's Hosptial
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Harbor-UCLA Medical Center
City
Torrence
State/Province
California
ZIP/Postal Code
90502
Country
United States
Facility Name
Al DuPont Hospital for Children
City
Wilmington
State/Province
Delaware
ZIP/Postal Code
19803
Country
United States
Facility Name
Howard University
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20060
Country
United States
Facility Name
Children's Hospital of SouthWest Florida
City
Fort Myers
State/Province
Florida
ZIP/Postal Code
33908
Country
United States
Facility Name
Joe Dimaggio Children's Hospital
City
Hollywood
State/Province
Florida
ZIP/Postal Code
33021
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Miami Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
All Children's Hospital
City
St. Petersburg
State/Province
Florida
ZIP/Postal Code
33701
Country
United States
Facility Name
Tampa General Hospital
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States
Facility Name
Grady Memorial Hospital
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30303
Country
United States
Facility Name
Georgia Regents University
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
Ann and Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Children's Hospital at the University of Illinois
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Fort Wayne Lutheran Hospital
City
Ft. Wayne
State/Province
Indiana
ZIP/Postal Code
46804
Country
United States
Facility Name
Riley Hospital for Children
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
University of Iowa-Children's Hospital
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Louisville/Kosair Children's Hospital
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Our Lady of the Lake Children's Hospital
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70808
Country
United States
Facility Name
Children's Hospital of New Orleans
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70118
Country
United States
Facility Name
Johns Hopkins
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
The Herman and Walter Samuelson Children's Hospital at Sinai
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21215
Country
United States
Facility Name
Children's Hospital of Michigan-Wayne State University
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Hurley Research Center
City
Flint
State/Province
Michigan
ZIP/Postal Code
48503
Country
United States
Facility Name
University of Mississippi Medical Center
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
Rutger's University
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08903
Country
United States
Facility Name
Bronx Lebanon Hospital
City
Bronx
State/Province
New York
ZIP/Postal Code
10457
Country
United States
Facility Name
NY Methodist
City
Brooklyn
State/Province
New York
ZIP/Postal Code
11215
Country
United States
Facility Name
Cohen Children's Medical Center
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11040
Country
United States
Facility Name
Golisano Children's Hospital at URMC
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
University of North Carolina-Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
East Carolina University
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27834
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Rainbow Babies and Children's Hospital
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Randall Children's Hospital
City
Portland
State/Province
Oregon
ZIP/Postal Code
97227
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
University of Pittsburgh Medical Center
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15232
Country
United States
Facility Name
The Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
T.C. Thompson Children's Hospital
City
Chattanooga
State/Province
Tennessee
ZIP/Postal Code
37403
Country
United States
Facility Name
Cook's Children Hospital
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Virginia Medical Center
City
Charlottsville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Children's Hospital of the King's Daughters
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23507
Country
United States
Facility Name
Children's Hospital of Richmond
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
Reearch Center
City
Antwerp
Country
Belgium
Facility Name
Research Center
City
Brussels
Country
Belgium
Facility Name
Research Center
City
Edgem
Country
Belgium
Facility Name
Research Center
City
Liege
Country
Belgium
Facility Name
Research Center
City
Montegnee
Country
Belgium
Facility Name
Resaerch Center
City
Rio de Janerio
Country
Brazil
Facility Name
Research Center
City
Sao Paulo
Country
Brazil
Facility Name
Research Center
City
Santo Domingo
ZIP/Postal Code
10101
Country
Dominican Republic
Facility Name
Research Center
City
Santo Domingo
ZIP/Postal Code
10514
Country
Dominican Republic
Facility Name
Research Center
City
Kingston
Country
Jamaica
Facility Name
Research Center
City
Irbid
Country
Jordan
Facility Name
Research Center
City
Beirut
Country
Lebanon
Facility Name
Research Center
City
Tripoli
Country
Lebanon
Facility Name
Research Center - Child Health Department
City
Muscat
Country
Oman
Facility Name
Research Center
City
Muscat
Country
Oman
Facility Name
Research Center Hospital del Nino
City
Panama
Country
Panama
Facility Name
Research Center Hospital of Pediatric Specialities
City
Panama
Country
Panama
Facility Name
Research Center Metropolitan Hospital
City
Panama
Country
Panama
Facility Name
Research Center
City
Madrid
Country
Spain
Facility Name
Research Center
City
Adana
Country
Turkey
Facility Name
Research Center
City
Istanbul
Country
Turkey
Facility Name
Research Center
City
Mersin
Country
Turkey

12. IPD Sharing Statement

Citations:
PubMed Identifier
33877274
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.
Results Reference
derived

Learn more about this trial

Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)

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