NCT02449616

Brief Summary

The purpose of this study is to evaluate the safety of repeat administration of MST-188 during vaso-occlusive crisis of sickle cell disease. Additionally, this study will evaluate the development of acute chest syndrome during VOC and re-hospitalization for recurrence of VOC.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_3

Geographic Reach
1 country

9 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 18, 2015

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 20, 2015

Completed
12 days until next milestone

Study Start

First participant enrolled

June 1, 2015

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2016

Completed
Last Updated

October 28, 2016

Status Verified

October 1, 2016

Enrollment Period

1.3 years

First QC Date

May 18, 2015

Last Update Submit

October 26, 2016

Conditions

Keywords

Sickle cellvaso-occlusive crisis

Outcome Measures

Primary Outcomes (1)

  • Safety as measured as the incidence of adverse events

    30 days after administration of study drug

Secondary Outcomes (2)

  • Rate of re-hospitalization for recurrence of VOC

    Within 14 days of the date of discharge

  • Occurrence of acute chest syndrome

    Within 120 hours of registration

Study Arms (1)

Study Drug

EXPERIMENTAL

MST-188

Drug: MST-188

Interventions

Intravenous administration as 100 mg/kg for 1 hour followed by 30 mg/kg/hour for up to 48 hours.

Also known as: vepoloxamer
Study Drug

Eligibility Criteria

Age4 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Completed participation in study MST-188-01 (EPIC study)
  • Subject age 4 through 65 years
  • Subject is experiencing acute pain typical of VOC and requires treatment with parenteral opioid analgesia
  • Subject requires hospitalization

You may not qualify if:

  • 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
  • Subject has been transfused within the past 14 days
  • Subject has complications related to SCD

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Rady Children's Hospital

San Diego, California, United States

Location

Children's Hospital of Southwest Florida

Fort Myers, Florida, 33908, United States

Location

Joe Dimaggio Children's Hospital

Hollywood, Florida, 33021, United States

Location

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Location

University of Iowa Children's Hospital

Iowa City, Iowa, United States

Location

Our Lady of the Lake Children's Hospital

Baton Rouge, Louisiana, 70808, United States

Location

Rutgers University

New Brunswick, New Jersey, United States

Location

Medical University of South Carolina

Charleston, South Carolina, United States

Location

T. C. Thompson Children's Hospital

Chattanooga, Tennessee, United States

Location

MeSH Terms

Conditions

Anemia, Sickle CellVaso-Occlusive Crises

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Edwin L Parsley, D. O.

    Mast Therapeutics, Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 18, 2015

First Posted

May 20, 2015

Study Start

June 1, 2015

Primary Completion

September 1, 2016

Last Updated

October 28, 2016

Record last verified: 2016-10

Locations