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

• ClinicalTrials.gov Identifier: NCT01737814
• Recruitment Status: Completed
• First Posted: November 30, 2012
• Last Update Posted: October 28, 2016
• Sponsor: Mast Therapeutics, Inc.
• Information provided by (Responsible Party): Mast Therapeutics, Inc.

Tracking Information

• First Submitted Date: November 27, 2012
• First Posted Date: November 30, 2012
• Last Update Posted Date: October 28, 2016
• Study Start Date: May 2013
• Actual Primary Completion Date: February 2016 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: November 29, 2012): 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 ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 29, 2012)

• Re-hospitalization rate for VOC [ Time Frame: Hospital discharge to 14 days post-discharge ]
• Occurence of acute chest syndrome [ Time Frame: Randomization to 120 hours after randomization ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)
• 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
• Brief Summary: 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment

Condition

• Vaso-occlusive Crisis
• Sickle Cell Disease

Intervention

• Drug: Saline
• Drug: MST-188
  Other Name: vepoloxamer

Study Arms

• 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.
  Intervention: Drug: MST-188
• Placebo Comparator: Saline
  Saline administered as a continuous infusion for up to 49 hours
  Intervention: Drug: Saline

• Publications *: Casella JF, Barton BA, Kanter J, Black LV, Majumdar S, Inati A, Wali Y, Drachtman RA, Abboud MR, Kilinc Y, Fuh BR, Al-Khabori MK, Takemoto CM, Salman E, Sarnaik SA, Shah N, Morris CR, Keates-Baleeiro J, Raj A, Alvarez OA, Hsu LL, Thompson AA, Sisler IY, Pace BS, Noronha SA, Lasky JL 3rd, de Julian EC, Godder K, Thornburg CD, Kamberos NL, Nuss R, Marsh AM, Owen WC, Schaefer A, Tebbi CK, Chantrain CF, Cohen DE, Karakas Z, Piccone CM, George A, Fixler JM, Singleton TC, Moulton T, Quinn CT, de Castro Lobo CL, Almomen AM, Goyal-Khemka M, Maes P, Emanuele M, Gorney RT, Padgett CS, Parsley E, Kronsberg SS, Kato GJ, Gladwin MT. Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial. JAMA. 2021 Apr 20;325(15):1513-1523. doi: 10.1001/jama.2021.3414.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: November 29, 2012): 388
• Original Estimated Enrollment: Same as current
• Study Completion Date: Not Provided
• Actual Primary Completion Date: February 2016 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 4 Years to 65 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Belgium, Brazil, Dominican Republic, Jamaica, Jordan, Lebanon, Oman, Panama, Spain, Turkey, United States

Administrative Information

• NCT Number: NCT01737814
• Other Study ID Numbers: MST-188-01
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Mast Therapeutics, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Mast Therapeutics, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Edwin L. Parsley, D.O.; Mast Therapeutics, Inc.

• PRS Account: Mast Therapeutics, Inc.
• Verification Date: October 2016