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

Phase 1

Conditions: Sickle Cell Disease with Vaso-Occlusive Crisis
MedDRA version: 17.0Level: LLTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2013-002986-21-ES

Lead Sponsor: Mast Therapeutics, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 388

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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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.;Secondary Objective: The secondary objectives are:<br>o To compare the re-hospitalization rate (for VOC) between the treatment arms.<br>o To compare the occurrence of acute chest syndrome (ACS) between the treatment arms.;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.;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 Outcome Measures

Name	Time	Method
Secondary end point(s): Secondary Endpoints <br>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. <br>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. <br>2. Within 120 hours of randomization

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