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

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Mast Therapeutics, Inc.
Sponsor:
Information provided by (Responsible Party):
Mast Therapeutics, Inc.
ClinicalTrials.gov Identifier:
NCT01737814
First received: November 27, 2012
Last updated: August 2, 2014
Last verified: August 2014
  Purpose

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.


Condition Intervention Phase
Vaso-occlusive Crisis
Sickle Cell Disease
Drug: MST-188
Drug: Saline
Phase 3

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

Resource links provided by NLM:


Further study details as provided by Mast Therapeutics, Inc.:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Re-hospitalization rate for VOC [ Time Frame: Hospital discharge to 14 days post-discharge ] [ Designated as safety issue: No ]
  • Occurence of acute chest syndrome [ Time Frame: Randomization to 120 hours after randomization ] [ Designated as safety issue: No ]

Estimated Enrollment: 388
Study Start Date: May 2013
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 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.
Drug: MST-188
Placebo Comparator: Saline
Saline administered as a continuous infusion for up to 49 hours
Drug: Saline

  Eligibility

Ages Eligible for Study:   4 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01737814

Contacts
Contact: Mast Therapeutics CT.gov Call Center 1-888-965-1238

  Show 49 Study Locations
Sponsors and Collaborators
Mast Therapeutics, Inc.
Investigators
Study Director: Santosh Vetticaden, PhD, MD Mast Therapeutics, Inc.
  More Information

No publications provided

Responsible Party: Mast Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT01737814     History of Changes
Other Study ID Numbers: MST-188-01
Study First Received: November 27, 2012
Last Updated: August 2, 2014
Health Authority: United States: Food and Drug Administration
Belgium: Federal Agency for Medicinal Products and Health Products
Lebanon: Ministry of Public Health
Oman: Ministry of Health, Sultanate of Oman
Panama: Ministry of Health, Panama

Keywords provided by Mast Therapeutics, Inc.:
sickle cell disease
vaso-occlusive crisis

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on September 18, 2014