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

Sickle Cell Disease Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01737814
• Other study ID #: MST-188-01
• Status: Completed
• Phase: Phase 3
• First received: November 27, 2012
• Last updated: October 26, 2016
• Start date: May 2013

Study information

• Verified date: October 2016
• Source: Mast Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationBelgium: Federal Agency for Medicines and Health Products, FAMHPBrazil: National Committee of Ethics in ResearchDominican Republic: Consejo Nacional de Bioetica en SaludJordan: Jordanian Food and Drug AdministrationLebanon: Institutional Review BoardOman: Academic Accreditation AuthorityPanama: Ministry of HealthSpain: Ministerio de Sanidad, Servicios Sociales e IgualdadSaudi Arabia: Saudi Food and Drug AdministrationTurkey: Drug and Medical Device InstitutionJamaica: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 388
• Est. primary completion date: February 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 4 Years to 65 Years

Eligibility

Inclusion Criteria:

• Age 4 through 65 years

• Subject has a confirmed diagnosis of HbSS, HbSC, HbSß+thal, or HbSß0thal

• 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

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease
• Vaso-occlusive Crisis

Intervention

Drug:
• Saline

• MST-188

Locations

Country	Name	City	State
Belgium	Reearch Center	Antwerp
Belgium	Research Center	Brussels
Belgium	Research Center	Edgem
Belgium	Research Center	Liege
Belgium	Research Center	Montegnee
Brazil	Resaerch Center	Rio de Janerio
Brazil	Research Center	Sao Paulo
Dominican Republic	Research Center	Santo Domingo
Dominican Republic	Research Center	Santo Domingo
Jamaica	Research Center	Kingston
Jordan	Research Center	Irbid
Lebanon	Research Center	Beirut
Lebanon	Research Center	Tripoli
Oman	Research Center	Muscat
Oman	Research Center - Child Health Department	Muscat
Panama	Research Center Hospital del Nino	Panama
Panama	Research Center Hospital of Pediatric Specialities	Panama
Panama	Research Center Metropolitan Hospital	Panama
Spain	Research Center	Madrid
Turkey	Research Center	Adana
Turkey	Research Center	Istanbul
Turkey	Research Center	Mersin
United States	Grady Memorial Hospital	Atlanta	Georgia
United States	Georgia Regents University	Augusta	Georgia
United States	Johns Hopkins	Baltimore	Maryland
United States	The Herman and Walter Samuelson Children's Hospital at Sinai	Baltimore	Maryland
United States	Our Lady of the Lake Children's Hospital	Baton Rouge	Louisiana
United States	Bronx Lebanon Hospital	Bronx	New York
United States	NY Methodist	Brooklyn	New York
United States	University of North Carolina-Chapel Hill	Chapel Hill	North Carolina
United States	The Medical University of South Carolina	Charleston	South Carolina
United States	University of Virginia Medical Center	Charlottsville	Virginia
United States	T.C. Thompson Children's Hospital	Chattanooga	Tennessee
United States	Ann and Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Children's Hospital at the University of Illinois	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Rainbow Babies and Children's Hospital	Cleveland	Ohio
United States	Children's Hospital of Michigan-Wayne State University	Detroit	Michigan
United States	Duke University Medical Center	Durham	North Carolina
United States	Hurley Research Center	Flint	Michigan
United States	Children's Hospital of SouthWest Florida	Fort Myers	Florida
United States	Cook's Children Hospital	Fort Worth	Texas
United States	Fort Wayne Lutheran Hospital	Ft. Wayne	Indiana
United States	East Carolina University	Greenville	North Carolina
United States	Joe Dimaggio Children's Hospital	Hollywood	Florida
United States	Texas Children's Hospital	Houston	Texas
United States	Riley Hospital for Children	Indianapolis	Indiana
United States	University of Iowa-Children's Hospital	Iowa City	Iowa
United States	University of Mississippi Medical Center	Jackson	Mississippi
United States	University of Louisville/Kosair Children's Hospital	Louisville	Kentucky
United States	Miami Children's Hospital	Miami	Florida
United States	University of Miami	Miami	Florida
United States	University of South Alabama	Mobile	Alabama
United States	Rutger's University	New Brunswick	New Jersey
United States	Cohen Children's Medical Center	New Hyde Park	New York
United States	Children's Hospital of New Orleans	New Orleans	Louisiana
United States	Children's Hospital of the King's Daughters	Norfolk	Virginia
United States	UCSF Benioff Children's Hospital	Oakland	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania
United States	Randall Children's Hospital	Portland	Oregon
United States	Children's Hospital of Richmond	Richmond	Virginia
United States	Golisano Children's Hospital at URMC	Rochester	New York
United States	University of California Davis Health System	Sacramento	California
United States	Rady Children's Hosptial	San Diego	California
United States	All Children's Hospital	St. Petersburg	Florida
United States	Tampa General Hospital	Tampa	Florida
United States	Harbor-UCLA Medical Center	Torrence	California
United States	Howard University	Washington	District of Columbia
United States	Al DuPont Hospital for Children	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Mast Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Belgium,  Brazil,  Dominican Republic,  Jamaica,  Jordan,  Lebanon,  Oman,  Panama,  Spain,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Secondary	Re-hospitalization rate for VOC		Hospital discharge to 14 days post-discharge	No
Secondary	Occurence of acute chest syndrome		Randomization to 120 hours after randomization	No