Sickle Cell DiseasePhase 2IMPACTS-2NCT01522196
A Study of Varespladib Infusion in Subjects With Sickle Cell Disease.
A Phase 2 study testing Varespladib in people with Sickle Cell Disease and Vaso-occlusive Crisis, run by Anthera Pharmaceuticals at 1 site in United States.
Trial summary
The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP). Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Sickle Cell Disease, Vaso-occlusive Crisis
- What is being tested?
- Varespladib
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2012to Mar 2012
- Phase
- Phase 2
- 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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Genotypes Hb SS, Hb SC, sickle β°-thalassemia, sickle β+ -thalassemia
- Pain consistent with vaso-occlusive crisis (clinical judgment) and not attributable to other causes
- Serum CRP ≥5.0 mg/L at time of screening
- Fever defined as oral temperature ≥38.0°C at time of screening
- Age ≥5 years
Who cannot join
- New or suspected new pulmonary infiltrate diagnosed by chest radiography
- Females who are nursing, pregnant or intend to become pregnant
- Renal dysfunction defined as a creatinine level >1.2 mg/dL for subjects aged 18 or less or a creatinine level >1.5 mg/dL for subjects over the age of 18
- Hepatic dysfunction (alanine aminotransferase [ALT] or aspartate aminotransferase [AST] >3 × upper limit of normal)
- Acute neurologic dysfunction
- Any medical condition for which transfusion may be needed imminently, and/or hemoglobin <5 g/dL
- Red blood cell transfusion within 30 days prior to screening
- Parenteral or oral corticosteroid therapy (inhaled steroids acceptable) within 7 days prior to screening
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Double-blind, Placebo-Controlled Study With Varespladib Infusion (A-001) in Subjects With Sickle Cell Disease and Vaso-Occlusive Crisis for the Prevention of Acute Chest Syndrome At-Risk Subjects.
The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis
- Being tested
- Varespladib, Placebo (Normal Saline)
- Keywords
- SCD
- 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.
VarespladibDrug
48 hour continuous infusion delivered intravenously (IV)at a rate of 110µg/kg/hr.
Placebo (Normal Saline)Other
48 hour continuous infusion delivered intravenously (IV)at a rate of 110µg/kg/hr.
| Study group | Role | What they receive |
|---|---|---|
| Varespladib | Active comparator | 48 hour continuous infusion delivered intravenously (IV) Receives: Drug: Varespladib |
| Placebo | Placebo comparator | 48 hour continuous infusion delivered intravenously (IV) Receives: Other: Placebo (Normal Saline) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with the combination of vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).
Measured: Various time points up to Day 30
Secondary outcomes (4)
Safety and tolerability of A 001 therapy when administered as a 48-hour continuous infusion to SCD subjects at-risk for acute chest syndrome.
Measured: Various time points up to Day 30
Impact of A-001 treatment on sPLA2 levels and CRP.
Measured: Various time points up to Day 30
Impact of A-001 treatment on the signs and symptoms of vaso-occlusive crisis.
Measured: Various time points up to Day 30
Impact of A-001 treatment on opioid use for pain in the treatment of vaso-occlusive crisis.
Measured: Various time points up to Day 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2012 (estimated)
- Study startFeb 2012
- Primary completionMar 2012
- Study completionMar 2012
Why it stopped: change in company plans
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 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Anthera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01522196
- Sponsor's protocol ID
- AN-SCD1122
- Acronym
- IMPACTS-2
- Status last verified
- Jan 2014
- First submitted
- 18 Jan 2012
- Last updated
- 4 Mar 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-Controlled Study With Varespladib Infusion (A-001) in Subjects With Sickle Cell Disease and Vaso-Occlusive Crisis for the Prevention of Acute Chest Syndrome At-Risk Subjects.
- ClinicalTrials.gov ID
- NCT01522196
- Acronym / sponsor ID
- IMPACTS-2 / AN-SCD1122
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis
- Keywords
- SCD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: change in company plans
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Anthera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Feb 2012
- Primary completion
- Mar 2012 (actual)
- Study completion
- Mar 2012 (actual)
- First posted
- 31 Jan 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 determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Varespladib | Active comparator | Drug: Varespladib48 hour continuous infusion delivered intravenously (IV) |
| Placebo | Placebo comparator | Other: Placebo (Normal Saline)48 hour continuous infusion delivered intravenously (IV) |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Genotypes Hb SS, Hb SC, sickle β°-thalassemia, sickle β+ -thalassemia
- Pain consistent with vaso-occlusive crisis (clinical judgment) and not attributable to other causes
- Serum CRP ≥5.0 mg/L at time of screening
- Fever defined as oral temperature ≥38.0°C at time of screening
- Age ≥5 years
Key exclusion criteria (8 listed)
- New or suspected new pulmonary infiltrate diagnosed by chest radiography
- Females who are nursing, pregnant or intend to become pregnant
- Renal dysfunction defined as a creatinine level >1.2 mg/dL for subjects aged 18 or less or a creatinine level >1.5 mg/dL for subjects over the age of 18
- Hepatic dysfunction (alanine aminotransferase [ALT] or aspartate aminotransferase [AST] >3 × upper limit of normal)
- Acute neurologic dysfunction
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 |
|---|---|---|
| Varespladib48 hour continuous infusion delivered intravenously (IV)at a rate of 110µg/kg/hr. | Drug | Varespladib |
| Placebo (Normal Saline)48 hour continuous infusion delivered intravenously (IV)at a rate of 110µg/kg/hr. | Other | Placebo |
Primary outcome
Efficacy of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with the combination of vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).
Time frame: Various time points up to Day 30
4 secondary outcomes
Safety and tolerability of A 001 therapy when administered as a 48-hour continuous infusion to SCD subjects at-risk for acute chest syndrome.
Time frame: Various time points up to Day 30
Impact of A-001 treatment on sPLA2 levels and CRP.
Time frame: Various time points up to Day 30
Impact of A-001 treatment on the signs and symptoms of vaso-occlusive crisis.
Time frame: Various time points up to Day 30
Impact of A-001 treatment on opioid use for pain in the treatment of vaso-occlusive crisis.
Time frame: Various time points up to Day 30
Results and safety
The study was stopped early (reason given: change in company plans). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2014.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Varespladib in people with Sickle Cell Disease and Vaso-occlusive Crisis, run by Anthera Pharmaceuticals at 1 site 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 5 inclusion and 8 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: stopped early, as of the sponsor's last update on 4 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early (reason given: change in company plans). Results are not available in the source record.
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?
Efficacy of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with the combination of vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP). (time frame: Various time points up to Day 30). 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 4 Mar 2014.