NCT01737814

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.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
388

participants targeted

Target at P50-P75 for phase_3

Geographic Reach
11 countries

72 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 27, 2012

Completed
3 days until next milestone

First Posted

Study publicly available on registry

November 30, 2012

Completed
5 months until next milestone

Study Start

First participant enrolled

May 1, 2013

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2016

Completed
Last Updated

October 28, 2016

Status Verified

October 1, 2016

Enrollment Period

2.8 years

First QC Date

November 27, 2012

Last Update Submit

October 26, 2016

Conditions

Keywords

sickle cell diseasevaso-occlusive crisis

Outcome Measures

Primary Outcomes (1)

  • 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

Secondary Outcomes (2)

  • Re-hospitalization rate for VOC

    Hospital discharge to 14 days post-discharge

  • Occurence of acute chest syndrome

    Randomization to 120 hours after randomization

Study Arms (2)

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.

Drug: MST-188

Saline

PLACEBO COMPARATOR

Saline administered as a continuous infusion for up to 49 hours

Drug: Saline

Interventions

SalineDRUG
Saline
Also known as: vepoloxamer
MST-188

Eligibility Criteria

Age4 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (72)

University of South Alabama

Mobile, Alabama, 36688, United States

Location

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

UCSF Benioff Children's Hospital

Oakland, California, 94609, United States

Location

University of California Davis Health System

Sacramento, California, 95817, United States

Location

Rady Children's Hosptial

San Diego, California, 92123, United States

Location

Harbor-UCLA Medical Center

Torrence, California, 90502, United States

Location

Al DuPont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Howard University

Washington D.C., District of Columbia, 20060, United States

Location

Children's Hospital of SouthWest Florida

Fort Myers, Florida, 33908, United States

Location

Joe Dimaggio Children's Hospital

Hollywood, Florida, 33021, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Tampa General Hospital

Tampa, Florida, 33606, United States

Location

Grady Memorial Hospital

Atlanta, Georgia, 30303, United States

Location

Georgia Regents University

Augusta, Georgia, 30912, United States

Location

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

Chicago, Illinois, 60611, United States

Location

Children's Hospital at the University of Illinois

Chicago, Illinois, 60612, United States

Location

Fort Wayne Lutheran Hospital

Fort Wayne, Indiana, 46804, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

University of Iowa-Children's Hospital

Iowa City, Iowa, 52242, United States

Location

University of Louisville/Kosair Children's Hospital

Louisville, Kentucky, 40202, United States

Location

Our Lady of the Lake Children's Hospital

Baton Rouge, Louisiana, 70808, United States

Location

Children's Hospital of New Orleans

New Orleans, Louisiana, 70118, United States

Location

Johns Hopkins

Baltimore, Maryland, 21205, United States

Location

The Herman and Walter Samuelson Children's Hospital at Sinai

Baltimore, Maryland, 21215, United States

Location

Children's Hospital of Michigan-Wayne State University

Detroit, Michigan, 48201, United States

Location

Hurley Research Center

Flint, Michigan, 48503, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

Rutger's University

New Brunswick, New Jersey, 08903, United States

Location

NY Methodist

Brooklyn, New York, 11215, United States

Location

Cohen Children's Medical Center

New Hyde Park, New York, 11040, United States

Location

Golisano Children's Hospital at URMC

Rochester, New York, 14642, United States

Location

Bronx Lebanon Hospital

The Bronx, New York, 10457, United States

Location

University of North Carolina-Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

East Carolina University

Greenville, North Carolina, 27834, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Randall Children's Hospital

Portland, Oregon, 97227, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15232, United States

Location

The Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

T.C. Thompson Children's Hospital

Chattanooga, Tennessee, 37403, United States

Location

Cook's Children Hospital

Fort Worth, Texas, 76104, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

University of Virginia Medical Center

Charlottsville, Virginia, 22908, United States

Location

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23507, United States

Location

Children's Hospital of Richmond

Richmond, Virginia, 23298, United States

Location

Reearch Center

Antwerp, Belgium

Location

Research Center

Brussels, Belgium

Location

Research Center

Edgem, Belgium

Location

Research Center

Liège, Belgium

Location

Research Center

Montegnée, Belgium

Location

Resaerch Center

Rio de Janerio, Brazil

Location

Research Center

São Paulo, Brazil

Location

Research Center

Santo Domingo, 10101, Dominican Republic

Location

Research Center

Santo Domingo, 10514, Dominican Republic

Location

Research Center

Kingston, Jamaica

Location

Research Center

Irbid, Jordan

Location

Research Center

Beirut, Lebanon

Location

Research Center

Tripoli, Lebanon

Location

Research Center - Child Health Department

Muscat, Oman

Location

Research Center

Muscat, Oman

Location

Research Center Hospital del Nino

Panama City, Panama

Location

Research Center Hospital of Pediatric Specialities

Panama City, Panama

Location

Research Center Metropolitan Hospital

Panama City, Panama

Location

Research Center

Madrid, Spain

Location

Research Center

Adana, Turkey (Türkiye)

Location

Research Center

Istanbul, Turkey (Türkiye)

Location

Research Center

Mersin, Turkey (Türkiye)

Location

Related Publications (1)

  • 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.

MeSH Terms

Conditions

Vaso-Occlusive CrisesAnemia, Sickle Cell

Interventions

Sodium Chloride

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

ChloridesHydrochloric AcidChlorine CompoundsInorganic ChemicalsSodium Compounds

Study Officials

  • Edwin L. Parsley, D.O.

    Mast Therapeutics, Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 27, 2012

First Posted

November 30, 2012

Study Start

May 1, 2013

Primary Completion

February 1, 2016

Last Updated

October 28, 2016

Record last verified: 2016-10

Locations