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

NCT ID: NCT01737814

Last Updated: 2016-10-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

388 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-05-31

Brief Summary

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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.

Detailed Description

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Conditions

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Vaso-occlusive Crisis Sickle Cell Disease

Keywords

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sickle cell disease vaso-occlusive crisis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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MST-188

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.

Group Type EXPERIMENTAL

MST-188

Intervention Type DRUG

Saline

Saline administered as a continuous infusion for up to 49 hours

Group Type PLACEBO_COMPARATOR

Saline

Intervention Type DRUG

Interventions

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Saline

Intervention Type DRUG

MST-188

Intervention Type DRUG

Other Intervention Names

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vepoloxamer

Eligibility Criteria

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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
Minimum Eligible Age

4 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Mast Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Edwin L. Parsley, D.O.

Role: STUDY_DIRECTOR

Mast Therapeutics, Inc.

Locations

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University of South Alabama

Mobile, Alabama, United States

Site Status

Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

UCSF Benioff Children's Hospital

Oakland, California, United States

Site Status

University of California Davis Health System

Sacramento, California, United States

Site Status

Rady Children's Hosptial

San Diego, California, United States

Site Status

Harbor-UCLA Medical Center

Torrence, California, United States

Site Status

Al DuPont Hospital for Children

Wilmington, Delaware, United States

Site Status

Howard University

Washington D.C., District of Columbia, United States

Site Status

Children's Hospital of SouthWest Florida

Fort Myers, Florida, United States

Site Status

Joe Dimaggio Children's Hospital

Hollywood, Florida, United States

Site Status

University of Miami

Miami, Florida, United States

Site Status

Miami Children's Hospital

Miami, Florida, United States

Site Status

All Children's Hospital

St. Petersburg, Florida, United States

Site Status

Tampa General Hospital

Tampa, Florida, United States

Site Status

Grady Memorial Hospital

Atlanta, Georgia, United States

Site Status

Georgia Regents University

Augusta, Georgia, United States

Site Status

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

Children's Hospital at the University of Illinois

Chicago, Illinois, United States

Site Status

Fort Wayne Lutheran Hospital

Fort Wayne, Indiana, United States

Site Status

Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

University of Iowa-Children's Hospital

Iowa City, Iowa, United States

Site Status

University of Louisville/Kosair Children's Hospital

Louisville, Kentucky, United States

Site Status

Our Lady of the Lake Children's Hospital

Baton Rouge, Louisiana, United States

Site Status

Children's Hospital of New Orleans

New Orleans, Louisiana, United States

Site Status

Johns Hopkins

Baltimore, Maryland, United States

Site Status

The Herman and Walter Samuelson Children's Hospital at Sinai

Baltimore, Maryland, United States

Site Status

Children's Hospital of Michigan-Wayne State University

Detroit, Michigan, United States

Site Status

Hurley Research Center

Flint, Michigan, United States

Site Status

University of Mississippi Medical Center

Jackson, Mississippi, United States

Site Status

Rutger's University

New Brunswick, New Jersey, United States

Site Status

NY Methodist

Brooklyn, New York, United States

Site Status

Cohen Children's Medical Center

New Hyde Park, New York, United States

Site Status

Golisano Children's Hospital at URMC

Rochester, New York, United States

Site Status

Bronx Lebanon Hospital

The Bronx, New York, United States

Site Status

University of North Carolina-Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

East Carolina University

Greenville, North Carolina, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Rainbow Babies and Children's Hospital

Cleveland, Ohio, United States

Site Status

Randall Children's Hospital

Portland, Oregon, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Site Status

The Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

T.C. Thompson Children's Hospital

Chattanooga, Tennessee, United States

Site Status

Cook's Children Hospital

Fort Worth, Texas, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

University of Virginia Medical Center

Charlottsville, Virginia, United States

Site Status

Children's Hospital of the King's Daughters

Norfolk, Virginia, United States

Site Status

Children's Hospital of Richmond

Richmond, Virginia, United States

Site Status

Reearch Center

Antwerp, , Belgium

Site Status

Research Center

Brussels, , Belgium

Site Status

Research Center

Edgem, , Belgium

Site Status

Research Center

Liège, , Belgium

Site Status

Research Center

Montegnée, , Belgium

Site Status

Resaerch Center

Rio de Janerio, , Brazil

Site Status

Research Center

São Paulo, , Brazil

Site Status

Research Center

Santo Domingo, , Dominican Republic

Site Status

Research Center

Santo Domingo, , Dominican Republic

Site Status

Research Center

Kingston, , Jamaica

Site Status

Research Center

Irbid, , Jordan

Site Status

Research Center

Beirut, , Lebanon

Site Status

Research Center

Tripoli, , Lebanon

Site Status

Research Center - Child Health Department

Muscat, , Oman

Site Status

Research Center

Muscat, , Oman

Site Status

Research Center Hospital del Nino

Panama City, , Panama

Site Status

Research Center Hospital of Pediatric Specialities

Panama City, , Panama

Site Status

Research Center Metropolitan Hospital

Panama City, , Panama

Site Status

Research Center

Madrid, , Spain

Site Status

Research Center

Adana, , Turkey (Türkiye)

Site Status

Research Center

Istanbul, , Turkey (Türkiye)

Site Status

Research Center

Mersin, , Turkey (Türkiye)

Site Status

Countries

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United States Belgium Brazil Dominican Republic Jamaica Jordan Lebanon Oman Panama Spain Turkey (Türkiye)

References

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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.

Reference Type DERIVED
PMID: 33877274 (View on PubMed)

Other Identifiers

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MST-188-01

Identifier Type: -

Identifier Source: org_study_id