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

• Register: EUCTR
• Last refreshed on: 10 October 2016
• Main ID: EUCTR2013-002986-21-ES
• Date of registration: 21/07/2014
• Prospective Registration: No
• 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: 16/07/2014
• 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: Regulatory Department
• Affiliation: Pharm-Olam International (Spain), S.L.U.

• Name: Regulatory Department
• Affiliation: Pharm-Olam International (Spain), S.L.U.

Key inclusion & exclusion criteria

Inclusion criteria:
1. Written documentation of informed consent and assent as applicable. Note: Minors must provide assent to participate in this study at an age-appropriate level determined and approved by the Institutional Review Board (IRB)/Independent Ethics Committee (IEC) for the study center.
2. Subject is ?4 and ?65 years of age.
3. Subject has confirmed diagnosis of HbSS, HbSC, HbS?+thal, or HbS?0thal.
4. Subject is experiencing acute pain typical of VOC and requires treatment with parenteral opioid analgesia.
5. Subject has been in moderate to severe pain as a result of the current VOC for no more than 24 hours at the time of presentation to the study center and for at least 4 hours prior to randomization.
6. Subject is hospitalized or in the process of admission for VOC at time of randomization.
7. If the subject is taking hydroxyurea, the dose is expected to remain stable through discharge.
8. If sexually active, the subject agrees to use reliable contraception while participating in this study and for at least 30 days after discontinuation of blinded study drug infusion.
9. If the subject is female and of child-bearing potential, must have negative pregnancy test (urine or serum).
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 suspected ACS, including either:
a) baseline chest X-ray indicating a new pulmonary infiltrate or
b) subject has acute respiratory symptoms consistent with ACS or with acute asthma attack.
2. Subject has platelet count <80,000/mm3.
3. Subject has a known or suspected bleeding disorder.
4. Subject has inadequate liver function defined as ALT >3X the institution?s upper limit of normal.
5. Subject has the following serum creatinine value:
? Age ?4-7 years: >0.8 mg/dL (>70.7 ?mol/L)
? Age ?8-13 years: >0.9 mg/dL (>79.6 ?mol/L)
? Age ?14 years: >1.0 mg/dL (>88.4 ?mol/L)
6. Subject is pregnant or nursing.
7. Subject has had an episode of painful crisis requiring hospitalization within the preceding 14 days.
8. Subject has been transfused within the past 14 days.
9. Subject is already hospitalized for any condition other than the current VOC.
10. Subject uses opioid analgesia on a daily basis for any reason.
11. Subject is currently receiving another investigational drug or has received any investigational drug within 30 days prior to randomization.
12. Subject presents with complications related to SCD, such as: aplastic crisis, priapism, sepsis, stroke, hepatic or splenic sequestration, or any complication expected to require surgical intervention.
13. Subject has experienced >5 hospitalizations for VOC in the prior 6 months.
14. Investigator believes subject is suffering from chronic pain (e.g., necrotic tissue resulting from repeated prior VOCs) and not acute pain associated with an ongoing VOC.
15. Subject is otherwise not an appropriate study candidate, in the Investigator's judgment.
16. Subject has been previously enrolled in the present trial or any prior MST-188 clinical trial.


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

Health Condition(s) or Problem(s) studied

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

Sickle Cell Disease with Vaso-Occlusive Crisis
MedDRA version: 17.0 Level: LLT Classification code 10040644 Term: Sickle cell disease System Organ Class: 100000004850

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
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)

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.

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.

Main Objective: The primary objective is to demostrate 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)

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.

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

Secondary ID(s)

2013-002986-21-BE

NCT01737814

31,246

MST-188-01

Source(s) of Monetary Support

Mast Therapeutics, Inc.