Vaso-occlusive CrisisPhase 3EPICNCT01737814
Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)
A Phase 3 study testing Saline and MST-188 in people with Vaso-occlusive Crisis and Sickle Cell Disease, run by Mast Therapeutics, Inc. at 72 sites in 11 countries.
Trial summary
The purpose of this study is to evaluate whether MST-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease. The study will also evaluate whether MST-188 can reduce the frequency of rehospitalization of subjects due to a recurrence of VOC. Read more about this study
- Who is it for?
- Ages 4 to 65 years
- Conditions the study is for
- Vaso-occlusive Crisis, Sickle Cell Disease
- What is being tested?
- Saline and MST-188
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 388 participants
- Where
- 72 sitesin 11 countries
- Study dates
- May 2013to Feb 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
- Age 4 through 65 years
- Subject has a confirmed diagnosis of HbSS, HbSC, HbSβ+thal, or HbSβ0thal
- Subject is experiencing acute pain typical of vaso-occlusive crisis requiring treatment with parenteral 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 or has experienced > 5 hospitalizations for VOC in the prior 6 months
- Subject has been transfused within the past 14 days
- Subject is hospitalized for a condition other than VOC
- Subject has complications related to SCD
Trial locations and contacts
72 sites in 11 countries.
72 sites in 11 countries
United States50 sites
Grady Memorial Hospital
Atlanta, Georgia, 30303
Georgia Regents University
Augusta, Georgia, 30912
Johns Hopkins
Baltimore, Maryland, 21205
The Herman and Walter Samuelson Children's Hospital at Sinai
Baltimore, Maryland, 21215
Our Lady of the Lake Children's Hospital
Baton Rouge, Louisiana, 70808
NY Methodist
Brooklyn, New York, 11215
University of North Carolina-Chapel Hill
Chapel Hill, North Carolina, 27599
The Medical University of South Carolina
Charleston, South Carolina, 29425
University of Virginia Medical Center
Charlottsville, Virginia, 22908
T.C. Thompson Children's Hospital
Chattanooga, Tennessee, 37403
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Children's Hospital at the University of Illinois
Chicago, Illinois, 60612
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Rainbow Babies and Children's Hospital
Cleveland, Ohio, 44106
Children's Hospital of Michigan-Wayne State University
Detroit, Michigan, 48201
Duke University Medical Center
Durham, North Carolina, 27710
Hurley Research Center
Flint, Michigan, 48503
Children's Hospital of SouthWest Florida
Fort Myers, Florida, 33908
Fort Wayne Lutheran Hospital
Fort Wayne, Indiana, 46804
Cook's Children Hospital
Fort Worth, Texas, 76104
East Carolina University
Greenville, North Carolina, 27834
Joe Dimaggio Children's Hospital
Hollywood, Florida, 33021
Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital for Children
Indianapolis, Indiana, 46202
University of Iowa-Children's Hospital
Iowa City, Iowa, 52242
University of Mississippi Medical Center
Jackson, Mississippi, 39216
University of Louisville/Kosair Children's Hospital
Louisville, Kentucky, 40202
University of Miami
Miami, Florida, 33136
Miami Children's Hospital
Miami, Florida, 33155
University of South Alabama
Mobile, Alabama, 36688
Rutger's University
New Brunswick, New Jersey, 08903
Cohen Children's Medical Center
New Hyde Park, New York, 11040
Children's Hospital of New Orleans
New Orleans, Louisiana, 70118
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
UCSF Benioff Children's Hospital
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15232
Randall Children's Hospital
Portland, Oregon, 97227
Children's Hospital of Richmond
Richmond, Virginia, 23298
Golisano Children's Hospital at URMC
Rochester, New York, 14642
University of California Davis Health System
Sacramento, California, 95817
Rady Children's Hosptial
San Diego, California, 92123
All Children's Hospital
St. Petersburg, Florida, 33701
Tampa General Hospital
Tampa, Florida, 33606
Bronx Lebanon Hospital
The Bronx, New York, 10457
Harbor-UCLA Medical Center
Torrence, California, 90502
Howard University
Washington D.C., District of Columbia, 20060
Al DuPont Hospital for Children
Wilmington, Delaware, 19803
Belgium5 sites
Reearch Center
Antwerp
Research Center
Brussels
Research Center
Edgem
Research Center
Liège
Research Center
Montegnée
Panama3 sites
Research Center Hospital del Nino
Panama City
Research Center Hospital of Pediatric Specialities
Panama City
Research Center Metropolitan Hospital
Panama City
Turkey (Türkiye)3 sites
Research Center
Adana
Research Center
Istanbul
Research Center
Mersin
Dominican Republic2 sites
Research Center
Santo Domingo, 10101
Research Center
Santo Domingo, 10514
Oman2 sites
Research Center - Child Health Department
Muscat
Research Center
Muscat
About this study
Official 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
The purpose of this study is to evaluate whether MST-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease. The study will also evaluate whether MST-188 can reduce the frequency of rehospitalization of subjects due to a recurrence of VOC. Additionally, this study will compare the development of acute chest syndrome during VOC in subjects who receive MST-188 to those who do not receive MST-188.
- Conditions
- Vaso-occlusive Crisis, Sickle Cell Disease
- Being tested
- Saline, MST-188
- Keywords
- sickle cell disease, vaso-occlusive crisis
- Medical subject headings
- Vaso-Occlusive Crises, Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SalineDrug
MST-188Drug
Also called vepoloxamer
| Study group | Role | What they receive |
|---|---|---|
| MST-188 | Experimental | MST-188 injection administered as a continuous infusion 100 mg/kg for 1 hour followed by 30 mg/kg/hr for up to 48 hours. Receives: Drug: MST-188 |
| Saline | Placebo comparator | Saline administered as a continuous infusion for up to 49 hours Receives: Drug: Saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction of the duration of vaso occlusive crisis (VOC) in subjects with sickle cell disease.
Measured: Study participants will be followed for the duration of hospital stay, an expected average of 4 days
Secondary outcomes (2)
Re-hospitalization rate for VOC
Measured: Hospital discharge to 14 days post-discharge
Occurence of acute chest syndrome
Measured: Randomization to 120 hours after randomization
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Nov 2012 (estimated)
- Study startMay 2013
- Primary completionFeb 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Vaso-occlusive CrisisStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 388 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
- NCT01737814
- Sponsor's protocol ID
- MST-188-01
- Acronym
- EPIC
- Status last verified
- Oct 2016
- First submitted
- 27 Nov 2012
- Last updated
- 28 Oct 2016
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Casella JF, Barton BA, Kanter J, Black LV, Majumdar S, Inati A, Wali Y, Drachtman RA, Abboud MR, Kilinc Y, Fuh BR, Al-Khabori MK, Takemoto CM, Salman E, Sarnaik SA, Shah N, Morris CR, Keates-Baleeiro J, Raj A, Alvarez OA, Hsu LL, Thompson AA, Sisler IY, Pace BS, Noronha SA, Lasky JL 3rd, de Julian EC, Godder K, Thornburg CD, Kamberos NL, Nuss R, Marsh AM, Owen WC, Schaefer A, Tebbi CK, Chantrain CF, Cohen DE, Karakas Z, Piccone CM, George A, Fixler JM, Singleton TC, Moulton T, Quinn CT, de Castro Lobo CL, Almomen AM, Goyal-Khemka M, Maes P, Emanuele M, Gorney RT, Padgett CS, Parsley E, Kronsberg SS, Kato GJ, Gladwin MT. Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial. JAMA. 2021 Apr 20;325(15):1513-1523. doi: 10.1001/jama.2021.3414.
PubMed 33877274
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 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
- ClinicalTrials.gov ID
- NCT01737814
- Acronym / sponsor ID
- EPIC / MST-188-01
- Conditions
- Vaso-occlusive Crisis, Sickle Cell Disease
- Keywords
- sickle cell disease, vaso-occlusive crisis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 388 participants (actual)
- Lead sponsor
- Mast Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- May 2013
- Primary completion
- Feb 2016 (actual)
- First posted
- 30 Nov 2012 (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 whether MST-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease. The study will also evaluate whether MST-188 can reduce the frequency of rehospitalization of subjects due to a recurrence of VOC. Additionally, this study will compare the development of acute chest syndrome during VOC in subjects who receive MST-188 to those who do not receive MST-188.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MST-188 | Experimental | Drug: MST-188MST-188 injection administered as a continuous infusion 100 mg/kg for 1 hour followed by 30 mg/kg/hr for up to 48 hours. |
| Saline | Placebo comparator | Drug: SalineSaline administered as a continuous infusion for up to 49 hours |
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)
- Age 4 through 65 years
- Subject has a confirmed diagnosis of HbSS, HbSC, HbSβ+thal, or HbSβ0thal
- Subject is experiencing acute pain typical of vaso-occlusive crisis requiring treatment with parenteral analgesia
- Subject requires hospitalization
Key exclusion criteria (7 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 or has experienced > 5 hospitalizations for VOC in the prior 6 months
- 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 |
|---|---|---|
| Saline | Drug | Saline |
| MST-188Also: vepoloxamer | Drug | MST-188 |
Primary outcome
Reduction of the duration of vaso occlusive crisis (VOC) in subjects with sickle cell disease.
Time frame: Study participants will be followed for the duration of hospital stay, an expected average of 4 days
2 secondary outcomes
Re-hospitalization rate for VOC
Time frame: Hospital discharge to 14 days post-discharge
Occurence of acute chest syndrome
Time frame: Randomization to 120 hours after randomization
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.
- Derived from this study: PubMed 33877274Casella JF, Barton BA, Kanter J, Black LV, Majumdar S, Inati A, Wali Y, Drachtman RA, Abboud MR, Kilinc Y, Fuh BR, Al-Khabori MK, Takemoto CM, Salman E, Sarnaik SA, Shah N, Morris CR, Keates-Baleeiro J, Raj A, Alvarez OA, Hsu LL, Thompson AA, Sisler IY, Pace BS, Noronha SA, Lasky JL 3rd, de Julian EC, Godder K, Thornburg CD, Kamberos NL, Nuss R, Marsh AM, Owen WC, Schaefer A, Tebbi CK, Chantrain CF, Cohen DE, Karakas Z, Piccone CM, George A, Fixler JM, Singleton TC, Moulton T, Quinn CT, de Castro Lobo CL, Almomen AM, Goyal-Khemka M, Maes P, Emanuele M, Gorney RT, Padgett CS, Parsley E, Kronsberg SS, Kato GJ, Gladwin MT. Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial. JAMA. 2021 Apr 20;325(15):1513-1523. doi: 10.1001/jama.2021.3414.
Related trials for Vaso-occlusive Crisis
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
RecruitingPhase 2
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
Sickle Cell Disease
Not yet recruiting
Hypoxic Red Blood Cells in Sickle Cell Anemia
Sickle Cell Anaemia, Sickle Cell Anemia Crisis
Recruiting
Virtual Reality for SCD VOC
Sickle Cell Disease
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
Sickle Cell Disease
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.
72 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Saline and MST-188 in people with Vaso-occlusive Crisis and Sickle Cell Disease, run by Mast Therapeutics, Inc. at 72 sites in 11 countries.
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 7 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 72 sites in United States, Belgium, Panama, Turkey (Türkiye), Brazil and 6 more countries. 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. Some participants in this study may receive a placebo. 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?
Reduction of the duration of vaso occlusive crisis (VOC) in subjects with sickle cell disease. (time frame: Study participants will be followed for the duration of hospital stay, an expected average of 4 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01737814, which also lists 1 linked publication.
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.