About Mast Therapeutics

We are a publicly traded biopharmaceutical company headquartered in San Diego, California.

We are leveraging the MAST (Molecular Adhesion and Sealant Technology) platform, derived from over two decades of clinical, nonclinical and manufacturing experience with purified and non-purified poloxamers, to develop vepoloxamer, our lead product candidate, for serious or life-threatening diseases with significant unmet needs.

To learn more about our company, please see our Letter from the CEO.

Investors

http://www.masttherapeutics.com/investors/

Evaluation of Purified Poloxamer 188 in Children in Crisis (EPIC)

https://clinicaltrials.gov/show/NCT01737814

This study is currently recruiting participants. (see Contacts and Locations)

Verified September 2015 by Mast Therapeutics, Inc.

Sponsor:

Mast Therapeutics, Inc.

Information provided by (Responsible Party):

Mast Therapeutics, Inc.

ClinicalTrials.gov Identifier:

NCT01737814

First received: November 27, 2012

Last updated: September 24, 2015

Last verified: September 2015

History of Changes

 

  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: Saline Drug: MST-188	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:

 

Genetics Home Reference related topics: sickle cell disease

MedlinePlus related topics: Sickle Cell Anemia

Drug Information available for: Morphine sulfate

Genetic and Rare Diseases Information Center resources: Sickle Cell Anemia

U.S. FDA Resources

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 Other Name: vepoloxamer
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 72 Study Locations

Sponsors and Collaborators

Mast Therapeutics, Inc.

Investigators

Study Director:	Edwin L. Parsley, D.O.	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:	September 24, 2015
Health Authority:	United States: Food and Drug Administration Belgium: Federal Agency for Medicines and Health Products, FAMHP Brazil: National Committee of Ethics in Research Dominican Republic: Consejo Nacional de Bioetica en Salud Jordan: Jordanian Food and Drug Administration Lebanon: Institutional Review Board Oman: Academic Accreditation Authority Panama: Ministry of Health Spain: Ministerio de Sanidad, Servicios Sociales e Igualdad Saudi Arabia: Saudi Food and Drug Administration Turkey: Drug and Medical Device Institution Jamaica: Ministry of Health

Keywords provided by Mast Therapeutics, Inc.:

sickle cell disease vaso-occlusive crisis

Additional relevant MeSH terms:

Anemia, Sickle Cell Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital	Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies

ClinicalTrials.gov processed this record on December 30, 2015

Technology

Hematology

MST-188 (lead product candidate)

MST-188 (purified poloxamer 188) is an investigational agent that has potential to reduce ischemic tissue injury and end-organ damage by restoring microvascular function, which is compromised in a wide range of serious and life-threatening diseases and conditions. We initially are developing MST-188 as a treatment for complications arising from sickle cell disease.

AIR001

This page contains Forward Looking Statements.*

AIR001 is a sodium nitrite solution for intermittent inhalation via nebulizer. Nitrite is a direct vasodilator and can be recycled in vivo to form nitric oxide (NO) independent of the classical NO synthase (NOS) pathway. Nitrite mediated NO formation has several beneficial effects, including dilation of blood vessels and reduction of inflammation and undesirable cell growth. Generation of NO from sodium nitrite is not dependent upon endothelial function and is enhanced in the setting of tissue hypoxia and acidosis, conditions in which NOS activity typically is depressed. In early clinical studies, AIR001 demonstrated positive hemodynamic effects with reductions observed in right atrial pressure and pulmonary capillary wedge pressure, as well as improvements in mean pulmonary artery pressures and cardiac output.

We are developing AIR001 for treatment of heart failure with preserved ejection fraction (HFpEF), a condition that affects millions in the U.S. and for which no proven therapeutic agent is available. Numerous lines of evidence suggest that HFpEF is a disease of NO deficiency. We are supporting two institution-sponsored Phase 2a studies in patients with HFpEF to evaluate acute hemodynamic effects of AIR001 and to evaluate its acute effects versus placebo on submaximal oxygen consumption and exercise hemodynamics.

Potential Applications of AIR001

Hemodynamic improvements, improvements in pulmonary artery pressures, and cardiac lusitropic effects observed in earlier studies of AIR001 support AIR001’s potential to benefit patients with heart failure with preserved ejection fraction (HFpEF). We currently are pursuing clinical development of AIR001 for that patient population.
 

2013 Activities

Event Timing

Initiate tQT/QTc Study Completed

Secure Orphan Designation for MST-188 for SCD in EU Completed

Activate First Site in Phase 3 Study Completed

File New Patent Applications Completed

Dose First Subject in Phase 3 Study Completed

Report data from tQT/QTc Study Q3 Completed and Primary Endpoints Met

Request Orphan Designation for MST-188 for ALI in U.S. Q3 Completed and Designation Granted By FDA

Initiate Nonclinical Proof-of-Concept Study in Heart Failure Q3 Completed and POC Data to be released Q1 '14

2014 Activities

Event Timing

Present Data From Nonclinical Proof-of-Concept Study In Heart Failure  Q1 '14  Data Released 01/06/14'Showed Significant Statistical Improvement"

Initiate Phase 2 Study in ALI Q1 '14 Initiated

Outcome of review of application for Unique Name Designation For Purified Poloxamer 188 From U.S. Adopted Names Counsel Q1 '14 Granted and Named VEPOLOXAMER

Open First Ex-U.S. Clinical Sites in Phase 3 Trial Q1 '14. Initiated and On Schedule

Submit Abstract with Heart Failure Study Biomarker Data to Major Medical Conference Q2 '14

Request Orphan Designation for ALI in EU Q2 '14  Application Submitted

Initiate Nonclinical POC Study in Stroke Q2 '14 Study Initiated

Initiate EPIC Sub-Study Q2 '14 Initiated On Schedule 06/14/14

Report Results from Phase 2 Study of AIR001 in PAH Q3 '14 Positive Top line Data released 9/8/14

Initiate study with Dept of Defense: MST-188 Resuscitation from major trauma '14 CRADA with US Military Initiated Q3 '14

Receive protocol from FDA for Phase 2 Heart Failure Study Q4'14 FDA Approves Heart Failure Study 12/9/14

2015 Event Schedule

Initiate dosing Phase2a study in AIR001 Q1 '15.  INITIATED 2/4/15

Nonclinical Study EMBOLIC STROKE Data Q1 '15:  POSITIVE DATA RELEASED 2/11/15

Report nonclinical data for repeat dose on Vepoloxamer in Heart Failure Q1 '15 POSITIVE DATA RELEASE 03/02/15

Phase 2 Study HEART FAILURE Initiate Enrollment Q2 '15 INITIATED 3/23/15

EPIC EXTENSION STUDY (Repeat Exposure) Initiate Enrollment 1st Half '15 INITIATED 5/26/15 

Phase 2a Study of AIR001 (WHO GROUP 2) in HFpEF Preliminary Data 2nd Half '15

Phase 2 Study Heart Failure Interim Safety Analysis 2nd Half '15

EPIC STUDY COMPLETE ENROLLMENT Q4 '15

2016 EVENTS

EPIC Top Line Data Q1 2016







 

Video of Management of Mast Therapeutics $MSTX speaking about MST-188 for Sickle Cell Disease ,orphan drug 
http://www.youtube.com/watch?v=eI7inPOiyZM&feature=youtu.be 
http://www.masttherapeutics.com/wp-content/uploads/2013/04/EPIC-An-Ongoing-Pivotal-Phase-3-Study-in-Patients-with-Sickle-Cell-Disease-Aug-21-2013.

Schedule 13D and 13G filings submitted since 09/30/14

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Contact Information