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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Vaso-occlusive Crisis, Sickle Cell Disease

Intervention / Treatment
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* Drug: Saline
* Drug: MST-188


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 4 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MST-188<br>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<br> <br> * Other names: vepoloxamer;|
| Placebo Comparator: Saline<br>Saline administered as a continuous infusion for up to 49 hours | Drug: Saline<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Re-hospitalization rate for VOC |  | Hospital discharge to 14 days post-discharge | 
| Occurence of acute chest syndrome |  | Randomization to 120 hours after randomization | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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sickle cell disease, vaso-occlusive crisis