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 MST-188, our lead product candidate, for serious or life-threatening diseases with significant unmet needs.
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Evaluation of Purified Poloxamer 188 in Children in Crisis (EPIC)
This study is currently recruiting participants.
Verified July 2013 by Mast Therapeutics, Inc.
Information provided by (Responsible Party):
Mast Therapeutics, Inc.
First received: November 27, 2012
Last updated: July 22, 2013
Last verified: July 2013
The purpose of this study is to evaluate whether ANX-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease. The study will also evaluate whether ANX-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 ANX-188 to those who do not receive ANX-188.
| Vaso-occlusive Crisis |
Sickle Cell Disease
| Drug: ANX-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 Children in Crisis (EPIC): A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial of ANX-188 (Purified Poloxamer 188) Injection in Children With Sickle Cell Disease Experiencing Vaso Occlusive Crisis |
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) |
| Experimental: ANX-188 |
ANX-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: ANX-188 |
| Placebo Comparator: Saline |
Saline administered as a continuous infusion for up to 49 hours
| Drug: Saline |
Please refer to this study by its ClinicalTrials.gov identifier: NCT01737814
| Contact: Mast Therapeutics CT.gov Call Center || 1-888-965-1238 || |
| Responsible Party: || Mast Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: || NCT01737814 History of Changes |
| Other Study ID Numbers: || ANX-188-01 |
| Study First Received: || November 27, 2012 |
| Last Updated: || July 22, 2013 |
| Health Authority: || United States: Food and Drug Administration |
Keywords provided by Mast Therapeutics, Inc.:
| sickle cell disease |
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on July 25, 2013
| Anemia, Sickle Cell |
Anemia, Hemolytic, Congenital
| Hematologic Diseases |
Genetic Diseases, Inborn
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.
Exelbine™, or ANX-530 (vinorelbine injectable emulsion), is a novel emulsion formulation of the chemotherapy drug vinorelbine (Navelbine®). We are seeking a partner or outside investor to continue development of the Exelbine program.
ANX-514 (docetaxel for injectable emulsion) is a novel, detergent-free emulsion formulation of the chemotherapy drug docetaxel. We are seeking a partner or outside investor to continue development of the ANX-514 program. 2013 Activities
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
Submit Applications for U.S. Government Funding for Phase
2 Study in Resuscitation of Shock following Major Trauma Q3
Request Orphan Designation for MST-188 for ALI in U.S. Q3
Initiate Nonclinical Proof-of-Concept Study in Heart Failure Q3
Open First Ex-U.S. Clinical Site in Phase 3 Study Q4
Initiate Phase 2 Study in ALI Q4 ‘13/Q1 ‘14
Video of Management of Mast Therapeutics $MSTX speaking about MST-188 for Sickle Cell Disease ,orphan drug
Schedule 13D and 13G filings submitted since 03/31/2013
| Reported via || Event Date || Name || Ticker || Shares || Change || % of Portfolio |
| 13F || 2013-03-31 || MAST THERAPEUTICS INC || MSTX || 875,000 || New || 0.11 |
| 13F || 2013-06-30 || MAST THERAPEUTICS INC || MSTX || 1,848,045 || 973,045 || 0.14 |