Sickle Cell AnemiaNCT01589926
Bi-Level Positive Airway Ventilation for Acute Chest Syndrome
A study testing Bi-level positive airway pressure device in people with Sickle Cell Anemia and Acute Chest Syndrome, run by Albert Einstein College of Medicine at 1 site in United States.
Trial summary
Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by having findings on a chest x-ray and one of the following: chest pain, fever, or trouble breathing. Read more about this study
- Who is it for?
- Ages 4 to 21 years
- Conditions the study is for
- Sickle Cell Anemia, Acute Chest Syndrome
- What is being tested?
- Bi-level positive airway pressure device
- Type of treatment
- Medical device
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2012to 8 Sep 2016
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- patients diagnosed with Hemoglobin SS (HB SS), the most common type of sickle cell disease
- patients diagnosed with Hemoglobin SC (HB SC), the second most common type of sickle cell disease.
- patients diagnosed with Hemoglobin sickle beta-zero thalassemia ( HB SB0thal) or Hemoglobin sickle thalassemia (HB SBthal)
- Must meet clinical criteria for ACS- an infiltrate on Chest X-ray and one of the following:
- Respiratory symptoms/signs (patients pulse oximetry < 92% or oxygen saturation < 2% below their baseline, tachypnea, cough, and increased work of breathing)
- Fever
Show all 11Show fewer
- Chest pain AND
- Patients' eligible for a simple transfusion based on one of the following criteria:
- Hypoxemia (patients pulse oximetry < 92% or oxygen saturation < 2% below their baseline)
- Hemoglobin < 5 gm/dl
- Increased work of breathing
Who cannot join
- Patient requires exchange transfusion within first 24 hours of admission
- Patient requires PCCU transfer within first 24 hours of admission
- Hemoglobin > 9gm/dl secondary to these patients requiring an exchange transfusion
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital @ Montefiore
The Bronx, New York, 10467
About this study
Official title: Early Bi-Level Positive Airway Pressure (BiPAP) Ventilation for Acute Chest Syndrome (ACS) - a Double-Blind Randomized Controlled Pilot Study
Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by having findings on a chest x-ray and one of the following: chest pain, fever, or trouble breathing. Patients with Acute Chest Syndrome can get very sick and require an exchange transfusion (special large blood transfusion) and mechanical ventilation. Bi-level Positive Airway Pressure (also known as BLPAP or BiPAP) is a device that blows air into a patients lungs via a mask that covers the nose. The goal of this study is to determine whether giving children BiPAP when they have ACS, in addition to providing standard clinical care for ACS, alters the clinical course of these patients. The investigators hypothesize that patients receiving effective BiPAP will have milder clinical courses resulting in shorter hospital stays and fewer transfers to the intensive care unit and exchange transfusions.
Read the detailed description
Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by a new infiltrate on chest x-ray and one of the following: chest pain, fever, or respiratory signs or symptoms (tachypnea, cough, new onset hypoxemia, or increased work of breathing.)The treatment for acute chest syndrome is focused on supportive care with hydration, antibiotics, blood transfusions and respiratory support. Unfortunately, despite these treatments many patients fail to have improvements in their respiratory status, or have respiratory decompensation. These patients require more aggressive treatments, which frequently include exchange transfusions, pediatric intensive care unit (PCCU) management, and respiratory support.
The study objective is to perform a prospective double blind randomized control trial to investigate if early initiation of effective BiPAP in addition to providing standard clinical care for ACS alters the clinical course of these patients vs. sham BiPAP and standard clinical care. Investigators hypothesize that participants receiving effective BiPAP will have milder clinical courses resulting in shorter hospital stays and fewer transfers to PCCU and exchange transfusions.
- Conditions
- Sickle Cell Anemia, Acute Chest Syndrome
- Being tested
- Bi-level positive airway pressure device, Sham CPAP
- Medical subject headings
- Anemia, Sickle Cell, Acute Chest Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bi-level positive airway pressure deviceDevice
BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Sham CPAPDevice
Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.
| Study group | Role | What they receive |
|---|---|---|
| Bi-level Positive Airway Pressure Device | Experimental | BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Receives: Device: Bi-level positive airway pressure device |
| Sham CPAP | Sham comparator | Physiologic continuous positive airway pressure (CPAP) initiated for at least 16 hours per day for a minimum of 48hrs. Receives: Device: Sham CPAP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Length of Stay as Measured by the Time From Initial Diagnosis of ACS Until Meeting Discharge Criteria.
Length of stay as measured by the time from initial diagnosis of ACS until meeting discharge criteria. It is anticipated length of stay will correlate to efficacy of treatment: shorter stay is theorized to indicate more efficient treatment.
Measured: From diagnosis of ACS until meeting discharge criteria- Average 7 days.
Secondary outcomes (6)
Rate of Exchange Transfusions.
Measured: Diagnosis until discharge. Average 7 days.
Determine Parent and Patient Acceptability of BLPAP Administration in the Setting of ACS.
Measured: Upon completion of intervention at 48hrs.
Rate of PCCU Transfers.
Measured: Diagnosis until discharge. Average 7 days.
Difference in Respiratory Rate.
Measured: 48hrs after initiation of treatment
Difference in Pulmonary Function Tests.
Measured: 48hrs after initiation of treatment
Difference in Mean SpO2 Recording During Sleep.
Peripheral capillary oxygen saturation (SpO2) is an estimate of the amount of oxygen in the blood. It is the percentage of haemoglobin containing oxygen compared to the total amount of haemoglobin in the blood (i.e. oxygenated haemoglobin vs oxygenated and non-oxygenated haemoglobin).
Measured: 48hrs after initiation of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2012 (estimated)
- Study startJul 2012
- Primary completion8 Sep 2016
- Study completion8 Sep 2016
Why it stopped: Low enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sickle Cell AnemiaStudies 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 N/A
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Albert Einstein College of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Deepa Manwani, Albert Einstein College of Medicine
- Investigators
- Deepa Manwani, MD, Principal investigator, Albert Einstein College of MedicineMichael E Roth, MD, Principal investigator, Albert Einstein College of MedicineKerry Morrone, MD, Study director, Albert Einstein College of MedicineHiren Muzumdar, MD, Principal investigator, Albert Einstein College of MedicineRanaan Arens, MD, Principal investigator, Albert Einstein College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01589926
- Sponsor's protocol ID
- 12-04-139
- Status last verified
- Nov 2018
- First submitted
- 30 Apr 2012
- Last updated
- 11 Dec 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Bi-Level Positive Airway Pressure (BiPAP) Ventilation for Acute Chest Syndrome (ACS) - a Double-Blind Randomized Controlled Pilot Study
- ClinicalTrials.gov ID
- NCT01589926
- Acronym / sponsor ID
- 12-04-139
- Conditions
- Sickle Cell Anemia, Acute Chest Syndrome
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low enrollment
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Albert Einstein College of MedicineUniversity, hospital or other
- Study start
- Jul 2012 (actual)
- Primary completion
- 8 Sep 2016 (actual)
- Study completion
- 8 Sep 2016 (actual)
- First posted
- 2 May 2012 (estimated)
- Results
- Posted 11 Dec 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by having findings on a chest x-ray and one of the following: chest pain, fever, or trouble breathing. Patients with Acute Chest Syndrome can get very sick and require an exchange transfusion (special large blood transfusion) and mechanical ventilation. Bi-level Positive Airway Pressure (also known as BLPAP or BiPAP) is a device that blows air into a patients lungs via a mask that covers the nose. The goal of this study is to determine whether giving children BiPAP when they have ACS, in addition to providing standard clinical care for ACS, alters the clinical course of these patients. The investigators hypothesize that patients receiving effective BiPAP will have milder clinical courses resulting in shorter hospital stays and fewer transfers to the intensive care unit and exchange transfusions.
Detailed description
Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by a new infiltrate on chest x-ray and one of the following: chest pain, fever, or respiratory signs or symptoms (tachypnea, cough, new onset hypoxemia, or increased work of breathing.)The treatment for acute chest syndrome is focused on supportive care with hydration, antibiotics, blood transfusions and respiratory support. Unfortunately, despite these treatments many patients fail to have improvements in their respiratory status, or have respiratory decompensation. These patients require more aggressive treatments, which frequently include exchange transfusions, pediatric intensive care unit (PCCU) management, and respiratory support.
The study objective is to perform a prospective double blind randomized control trial to investigate if early initiation of effective BiPAP in addition to providing standard clinical care for ACS alters the clinical course of these patients vs. sham BiPAP and standard clinical care. Investigators hypothesize that participants receiving effective BiPAP will have milder clinical courses resulting in shorter hospital stays and fewer transfers to PCCU and exchange transfusions.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Bi-level Positive Airway Pressure Device | Experimental | Device: Bi-level positive airway pressure deviceBiPAP initiated for at least 16 hours per day for a minimum of 48hrs. |
| Sham CPAP | Sham comparator | Device: Sham CPAPPhysiologic continuous positive airway pressure (CPAP) initiated for at least 16 hours per day for a minimum of 48hrs. |
Who may qualify
- Ages
- 4 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- patients diagnosed with Hemoglobin SS (HB SS), the most common type of sickle cell disease
- patients diagnosed with Hemoglobin SC (HB SC), the second most common type of sickle cell disease.
- patients diagnosed with Hemoglobin sickle beta-zero thalassemia ( HB SB0thal) or Hemoglobin sickle thalassemia (HB SBthal)
- Must meet clinical criteria for ACS- an infiltrate on Chest X-ray and one of the following:
- Respiratory symptoms/signs (patients pulse oximetry < 92% or oxygen saturation < 2% below their baseline, tachypnea, cough, and increased work of breathing)
Key exclusion criteria (3 listed)
- Patient requires exchange transfusion within first 24 hours of admission
- Patient requires PCCU transfer within first 24 hours of admission
- Hemoglobin > 9gm/dl secondary to these patients requiring an exchange transfusion
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 |
|---|---|---|
| Bi-level positive airway pressure deviceBiPAP initiated for at least 16 hours per day for a minimum of 48hrs. | Device | Bi-level Positive Airway Pressure Device |
| Sham CPAPSham CPAP initiated for at least 16 hours per day for a minimum of 48hrs. | Device | Sham CPAP |
Primary outcome
Length of Stay as Measured by the Time From Initial Diagnosis of ACS Until Meeting Discharge Criteria.
Time frame: From diagnosis of ACS until meeting discharge criteria- Average 7 days.
Length of stay as measured by the time from initial diagnosis of ACS until meeting discharge criteria. It is anticipated length of stay will correlate to efficacy of treatment: shorter stay is theorized to indicate more efficient treatment.
6 secondary outcomes
Rate of Exchange Transfusions.
Time frame: Diagnosis until discharge. Average 7 days.
Determine Parent and Patient Acceptability of BLPAP Administration in the Setting of ACS.
Time frame: Upon completion of intervention at 48hrs.
Rate of PCCU Transfers.
Time frame: Diagnosis until discharge. Average 7 days.
Difference in Respiratory Rate.
Time frame: 48hrs after initiation of treatment
Difference in Pulmonary Function Tests.
Time frame: 48hrs after initiation of treatment
Difference in Mean SpO2 Recording During Sleep.
Time frame: 48hrs after initiation of treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Deepa Manwani, MD principal investigator
Albert Einstein College of Medicine
Michael E Roth, MD principal investigator
Albert Einstein College of Medicine
Kerry Morrone, MD study director
Albert Einstein College of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Bi-level positive airway pressure device in people with Sickle Cell Anemia and Acute Chest Syndrome, run by Albert Einstein College of Medicine 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 11 inclusion and 3 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 11 Dec 2018. 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Dec 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Length of Stay as Measured by the Time From Initial Diagnosis of ACS Until Meeting Discharge Criteria. (time frame: From diagnosis of ACS until meeting discharge criteria- Average 7 days.). 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 11 Dec 2018.