Sickle Cell DiseasePhase 3EPIC-ENCT02449616
Evaluation of Repeat Administration of Purified Poloxamer 188
A Phase 3 study testing MST-188 in people with Sickle Cell Disease, run by Mast Therapeutics, Inc. at 9 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 65 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- MST-188
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 9 sitesUnited States
- Study dates
- Jun 2015to Sep 2016
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Our Lady of the Lake Children's Hospital
Baton Rouge, Louisiana, 70808
Medical University of South Carolina
Charleston, South Carolina
T. C. Thompson Children's Hospital
Chattanooga, Tennessee
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois
Children's Hospital of Southwest Florida
Fort Myers, Florida, 33908
Joe Dimaggio Children's Hospital
Hollywood, Florida, 33021
University of Iowa Children's Hospital
Iowa City, Iowa
Rutgers University
New Brunswick, New Jersey
Rady Children's Hospital
San Diego, California
About this study
Official title: Evaluation of Repeat Administration of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC-E): An Open-Label Safety Extension Trial Assessing Repeat Administration of MST-188 (Purified Poloxamer 188) Injection in Subjects With Sickle Cell Disease Experiencing Vaso Occlusive Crisis
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.
- Conditions
- Sickle Cell Disease
- Being tested
- MST-188
- Keywords
- Sickle cell, vaso-occlusive crisis
- Medical subject headings
- Anemia, Sickle Cell, Vaso-Occlusive Crises
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MST-188Drug
Also called vepoloxamer
Intravenous administration as 100 mg/kg for 1 hour followed by 30 mg/kg/hour for up to 48 hours.
| Study group | Role | What they receive |
|---|---|---|
| Study Drug | Experimental | MST-188 Receives: Drug: MST-188 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as measured as the incidence of adverse events
Measured: 30 days after administration of study drug
Secondary outcomes (2)
Rate of re-hospitalization for recurrence of VOC
Measured: Within 14 days of the date of discharge
Occurrence of acute chest syndrome
Measured: Within 120 hours of registration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2015 (estimated)
- Study startJun 2015
- Primary completionSep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mast Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Edwin L Parsley, D. O., Study director, Mast Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02449616
- Sponsor's protocol ID
- MST-188-04
- Acronym
- EPIC-E
- Status last verified
- Oct 2016
- First submitted
- 18 May 2015
- Last updated
- 28 Oct 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Repeat Administration of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC-E): An Open-Label Safety Extension Trial Assessing Repeat Administration of MST-188 (Purified Poloxamer 188) Injection in Subjects With Sickle Cell Disease Experiencing Vaso Occlusive Crisis
- ClinicalTrials.gov ID
- NCT02449616
- Acronym / sponsor ID
- EPIC-E / MST-188-04
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle cell, vaso-occlusive crisis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Mast Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2015
- Primary completion
- Sep 2016 (actual)
- First posted
- 20 May 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Study Drug | Experimental | Drug: MST-188MST-188 |
Who may qualify
- Ages
- 4 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MST-188Also: vepoloxamerIntravenous administration as 100 mg/kg for 1 hour followed by 30 mg/kg/hour for up to 48 hours. | Drug | Study Drug |
Primary outcome
Safety as measured as the incidence of adverse events
Time frame: 30 days after administration of study drug
2 secondary outcomes
Rate of re-hospitalization for recurrence of VOC
Time frame: Within 14 days of the date of discharge
Occurrence of acute chest syndrome
Time frame: Within 120 hours of registration
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Edwin L Parsley, D. O. study director
Mast Therapeutics, Inc.
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing MST-188 in people with Sickle Cell Disease, run by Mast Therapeutics, Inc. at 9 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety as measured as the incidence of adverse events (time frame: 30 days after administration of study drug). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Oct 2016.