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.
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.
Mast Therapeutics, Inc.
Information provided by (Responsible Party):
Mast Therapeutics, Inc.
First received: November 27, 2012
Last updated: September 24, 2015
Last verified: September 2015
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.
| || |
|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 |
Other Name: vepoloxamer
|Placebo Comparator: Saline |
Saline administered as a continuous infusion for up to 49 hours
|Drug: Saline |
| || |
|Ages Eligible for Study: ||4 Years to 65 Years |
|Genders Eligible for Study: ||Both |
|Accepts Healthy Volunteers: ||No |
- 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
- 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
| || || || |
|Contact: Mast Therapeutics CT.gov Call Center ||1-888-965-1238 || || |
Sponsors and Collaborators
Mast Therapeutics, Inc.
| || || || |
|Study Director: ||Edwin L. Parsley, D.O. ||Mast Therapeutics, Inc. || |
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 |
Additional relevant MeSH terms:
| || |
|Anemia, Sickle Cell |
Anemia, Hemolytic, Congenital
|Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on December 30, 2015
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.
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.
AIR001 Inhalation Solution is sodium nitrite formulated with a phosphate buffer into an inhalation solution. Nitrite is a physiological signaling molecule with roles in intravascular endocrine nitric oxide (NO) production, hypoxic vasodilation, signaling, and cytoprotection after ischemia-reperfusion. Nitrite serves as the largest physiologic reservoir of NO and can be converted to NO independent of NO synthase (NOS) activity.
In experimental models, nitrite use demonstrated improved remodeling both in the pulmonary vasculature and right ventricle. Hemodynamic effects include venodilation with reductions in right atrial pressures, pulmonary and systemic vasodilation with reductions in pulmonary vascular resistance and left atrial pressures, and improved cardiac relaxation. In addition, recent clinical and nonclinical studies have demonstrated that nitrite can stimulate mitochondrial biogenesis and mitochondrial fusion and decrease mitochondrial oxygen consumption through a mechanism distinct from that of NO, which may have utility in treating heart failure.
We obtained the AIR001 program through our acquisition of Aires Pharmaceuticals, Inc. in February 2014. Prior to the acquisition, AIR001 had been tested in more than 120 healthy volunteers and patients with various forms of pulmonary hypertension in three Phase 1 studies and in one Phase 2 study in patients with pulmonary arterial hypertension. While the Phase 2 study was prematurely terminated due to Aires’ capital constraints prior to the acquisition, preliminary data from the study are positive, showing improvements in hemodynamic parameters and in exercise capacity from baseline. There were no treatment-related serious adverse events and methemoglobin levels remained normal (< 1.5%), which distinguishes AIR001 from safety concerns associated with intravenously-administered nitrite.
Data from these early clinical studies support continued clinical development of AIR001. Given the observed hemodynamic improvements of decreased right arterial pressure, decreased pulmonary capillary wedge (or left atrial pressure), as well as improvements in pulmonary vascular resistance and cardiac lusitropic effects, AIR001 may benefit patients with exercise impairment due to heart failure with preserved ejection fraction (HFpEF), for whom these are all desirable effects.
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.
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
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
EPIC Top Line Data Q1 2016
Video of Management of Mast Therapeutics $MSTX speaking about MST-188 for Sickle Cell Disease ,orphan drug
Schedule 13D and 13G filings submitted since 09/30/14
| ||Filing Company ||Date of |
|Total Reported |
|Shares Reported |
| ||Baker Bros LLC ||12/31/14 ||4,774,434 ||4,774,434 |
| ||BVF Inc. ||3/31/15 ||7,241,600 ||7,241,600! |
|SC 13G ||FRANKLIN RESOURCES INC ||02/04/15 ||11,946,100 ||11,946,100 |
|Reported via ||Event Date ||Name ||Ticker ||Shares * ||Change ||% of Portfolio |
|13F MPM Asset Management || || ||MSTX ||2,551,851 || || |
|13F Vanguard Group || || ||MSTX ||3,382,483 || || |
13F Proquest Associates MSTX. 2,551,851