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MST-188

Phase 3

Sickle Cell Disease | Small molecule | Hematology |Savara, Inc.|Last Updated: Oct 28, 2016

Development status

Highest phase Phase 3
Registered trials 3 across 1 sponsor since Nov 2012

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment16

FDA Designations

No designations recorded

Clinical trial landscape

MST-188 · 2 trials · 2 indications

Phase 3 2
NCT02449616Evaluation of Repeat Administration of Purified Poloxamer 188Sickle Cell Disease
COMPLETED16 Analytics
NCT01737814Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)Vaso-occlusive Crisis
COMPLETED388 Analytics
PHASE3COMPLETED
Evaluation of Repeat Administration of Purified Poloxamer 188
Sickle Cell DiseaseUnlock trial analytics
PHASE3COMPLETED
Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)
Vaso-occlusive CrisisUnlock trial analytics

Study Endpoints

Primary Endpoints

Safety as measured as the incidence of adverse events
30 days after administration of study drug
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

Secondary Endpoints

Rate of re-hospitalization for recurrence of VOC
Within 14 days of the date of discharge
Occurrence of acute chest syndrome
Within 120 hours of registration
Re-hospitalization rate for VOC
Hospital discharge to 14 days post-discharge
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Study DrugEXPERIMENTALMST-188
MST-188EXPERIMENTALMST-188 injection administered as a continuous infusion 100 mg/kg for 1 hour followed by 30 mg/kg/hr for up to 48 hours.
SalinePLACEBO_COMPARATORSaline administered as a continuous infusion for up to 49 hours

Interventions

NameTypeDescription
MST-188DRUGIntravenous administration as 100 mg/kg for 1 hour followed by 30 mg/kg/hour for up to 48 hours.
SalineDRUG -
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Eligibility Criteria

Age Range4 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: * Completed participation in study MST-188-01 (EPIC study) * Subject age 4 through 65 years * Subject is experiencing acute pain typical of VOC and requires treatment with parenteral opioid analgesia * Subject requires hospitalization Exclusion Criteria: * Subject has acute ch...

Countries:United StatesBelgiumBrazilDominican RepublicJamaicaJordanLebanonOmanPanamaSpainTurkey (Türkiye)
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Frequently asked questions about MST-188

What is MST-188 used for?

MST-188 is an investigational small molecule being developed for vaso-occlusive crisis in sickle cell disease. It is designed to address the acute painful episodes caused by blocked blood flow. MST-188 is currently in clinical development and has not been approved by the FDA.

Who makes MST-188?

MST-188 is being developed by Savara, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker SVRA. Savara is conducting clinical trials to evaluate the safety and efficacy of MST-188 for the treatment of vaso-occlusive crisis in sickle cell disease.

What phase is MST-188 in?

MST-188 is in Phase 3 clinical development. Two Phase 3 trials have been completed, including a large study with 388 participants and a smaller repeat-administration study with 16 participants. MST-188 remains investigational and is not yet approved for any use.

What clinical trials is MST-188 in?

MST-188 has been studied in two completed Phase 3 trials. The primary trial, NCT01737814, enrolled 388 participants with vaso-occlusive crisis of sickle cell disease across multiple countries. A second trial, NCT02449616, evaluated repeat administration in 16 participants with sickle cell disease in the United States.

How does MST-188 work?

MST-188 is a purified poloxamer 188, a small molecule that targets vaso-occlusive crisis in sickle cell disease. It is designed to improve blood flow by reducing the adhesion of sickled red blood cells to blood vessel walls, thereby helping to prevent and resolve the blockages that cause painful crises.