Evaluation of Purified Poloxamer 188 in Subjects with Sickle Cell Disease Experiencing a Vaso-occlusive Crisis

• Register: EUCTR
• Last refreshed on: 17 October 2016
• Main ID: EUCTR2013-002986-21-BE
• Date of registration: 09/04/2014
• Prospective Registration: Yes
• Primary sponsor: Mast Therapeutics, Inc.
• Public title: Evaluation of Purified Poloxamer 188 in Subjects with Sickle Cell Disease Experiencing a Vaso-occlusive Crisis
• Scientific 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 - Evaluation of Purified Poloxamer 188 in Subjects in Crisis (EPIC)
• Date of first enrolment: 16/06/2014
• Target sample size: 388
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-002986-21
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Brazil, Dominican Republic, Jordan, Lebanon, Netherlands, Oman, Panama, Saudi Arabia, Spain, Turkey, United Kingdom, United States

Contacts

• Name: Country Manager
• Affiliation: Pharm-Olam International Belgium BVBA

• Name: Country Manager
• Affiliation: Pharm-Olam International Belgium BVBA

Key inclusion & exclusion criteria

Inclusion criteria:
1. Age 4 through 65 years
2. Subject has a confirmed diagnosis of HbSS, HbSC, HbSß+thal, or HbSß0thal
3. Subject is experiencing acute pain typical of vaso-occlusive crisis requiring treatment with parenteral analgesia
4. Subject requires hospitalization
Are the trial subjects under 18? yes
Number of subjects for this age range: 232
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 155
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion criteria:
1. Subject has acute chest syndrome
2. Subject's laboratory results indicate inadequate organ function
3. Subject is pregnant or nursing an infant
4. Subject had a painful crisis requiring hospitalization within the preceding 14 days or has experienced > 5 hospitalizations for VOC in the prior 6 months
5. Subject has been transfused within the past 14 days
6. Subject is hospitalized for a condition other than VOC
7. Subject has complications related to SCD


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Sickle Cell Disease with Vaso-Occlusive Crisis
MedDRA version: 18.0 Level: LLT Classification code 10040644 Term: Sickle cell disease System Organ Class: 100000004850

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Product Name: MST-188 (purified poloxamer 188) Injection
Product Code: MST-188
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Purified poloxamer 188
CAS Number: 9003-11-6
Current Sponsor code: Purified poloxamer 188 (vepoloxamer)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Solution for infusion
Route of administration of the placebo: Intravenous use

Primary Outcome(s)

Secondary Objective: The secondary objectives are:
o To compare the re-hospitalization rate (for VOC) between the treatment arms.
o To compare the occurrence of acute chest syndrome (ACS) between the treatment arms.

Timepoint(s) of evaluation of this end point: The time from randomization to last parenteral opioid analgesic dose for the treatment of VOC prior to hospital discharge.

Main Objective: The primary objective is to demonstrate the efficacy of MST-188 in reducing the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease (SCD). The duration of VOC will be measured from the time of randomization to the time at which a subject receives the last dose of parenteral opioid analgesia for the treatment of VOC prior to hospital discharge.

Primary end point(s): The primary endpoint of the study is the time from randomization to last parenteral opioid analgesic dose for the treatment of VOC prior to hospital discharge.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 1. Within 14 days after discharge from the hospital.
2. Within 120 hours of randomization

Secondary end point(s): Secondary Endpoints
1. Re-hospitalization for VOC: The number and percent of subjects who are re-hospitalized for VOC within 14 days after discharge from the hospital.
2. Occurrence of ACS: The number and percent of subjects who meet the protocol definition of ACS within 120 hours of randomization.

Secondary ID(s)

NCT01737814

31,246

MST-188-01

Source(s) of Monetary Support

Mast Therapeutics, Inc.