Bi-Level Positive Airway Ventilation for Acute Chest Syndrome

• Sponsor: Albert Einstein College of Medicine (Other)
• Overall Status: Terminated
• CT.gov ID: NCT01589926
• Collaborator: (none)
• Enrollment: 3
• Location: 1
• Arms: 2
• Actual Duration (Months): 50.3
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief 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. 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.

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Anemia; Acute Chest Syndrome	Device: Bi-level positive airway pressure device; Device: Sham CPAP	N/A

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

• Study Type: Interventional
• Actual Enrollment: 3 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Supportive Care
• Official Title: Early Bi-Level Positive Airway Pressure (BiPAP) Ventilation for Acute Chest Syndrome (ACS) - a Double-Blind Randomized Controlled Pilot Study
• Actual Study Start Date: Jul 1, 2012
• Actual Primary Completion Date: Sep 8, 2016
• Actual Study Completion Date: Sep 8, 2016

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Bi-level Positive Airway Pressure Device BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Device: Bi-level positive airway pressure device; BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Sham Comparator: Sham CPAP Physiologic continuous positive airway pressure (CPAP) initiated for at least 16 hours per day for a minimum of 48hrs.	Device: Sham CPAP; Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.

Outcome Measures

Primary Outcome Measures

1. Length of Stay as Measured by the Time From Initial Diagnosis of ACS Until Meeting Discharge Criteria. [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.

Secondary Outcome Measures

1. Rate of Exchange Transfusions. [Diagnosis until discharge. Average 7 days.]

2. Determine Parent and Patient Acceptability of BLPAP Administration in the Setting of ACS. [Upon completion of intervention at 48hrs.]

3. Rate of PCCU Transfers. [Diagnosis until discharge. Average 7 days.]

4. Difference in Respiratory Rate. [48hrs after initiation of treatment]

5. Difference in Pulmonary Function Tests. [48hrs after initiation of treatment]

6. Difference in Mean SpO2 Recording During Sleep. [48hrs after initiation of treatment]

   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).

Eligibility Criteria

Criteria

• Ages Eligible for Study: 4 Years to 21 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• 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

• 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

Exclusion Criteria:

• 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital @ Montefiore	Bronx	New York	United States	10467

Sponsors and Collaborators

• Albert Einstein College of Medicine

Investigators

• Principal Investigator: Deepa Manwani, MD, Albert Einstein College of Medicine
• Principal Investigator: Michael E Roth, MD, Albert Einstein College of Medicine
• Study Director: Kerry Morrone, MD, Albert Einstein College of Medicine
• Principal Investigator: Hiren Muzumdar, MD, Albert Einstein College of Medicine
• Principal Investigator: Ranaan Arens, MD, Albert Einstein College of Medicine

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Deepa Manwani, Professor, Pediatrics, Albert Einstein College of Medicine
• ClinicalTrials.gov Identifier: NCT01589926
• Other Study ID Numbers: 12-04-139
• First Posted: May 2, 2012
• Last Update Posted: Dec 11, 2018
• Last Verified: Nov 1, 2018

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: Yes
• Additional relevant MeSH terms: Acute Chest Syndrome; Anemia, Sickle Cell; Syndrome

Study Results

Participant Flow

Recruitment Details	Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.

Arm/Group Title	Bi-level Positive Airway Pressure	Sham CPAP
Arm/Group Description	BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Physiologic CPAP initiated for at least 16 hours per day for a minimum of 48hrs. Sham CPAP: Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Period Title: Overall Study
STARTED	0	0
COMPLETED	0	0
NOT COMPLETED	0	0

Baseline Characteristics

Arm/Group Title	Bi-level Positive Airway Pressure	Sham CPAP	Total
Arm/Group Description	BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Physiologic CPAP initiated for at least 16 hours per day for a minimum of 48hrs. Sham CPAP: Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Total of all reporting groups
Overall Participants	0	0	0
Age () []
<=18 years
Between 18 and 65 years
>=65 years
Age () []
Sex: Female, Male () []
Female
Male
Race and Ethnicity Not Collected () []
Region of Enrollment (participants) []

Outcome Measures

1. Primary Outcome

Title	Length of Stay as Measured by the Time From Initial Diagnosis of ACS Until Meeting Discharge Criteria.
Description	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.
Time Frame	From diagnosis of ACS until meeting discharge criteria- Average 7 days.

Outcome Measure Data

Analysis Population Description

Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.

Arm/Group Title	Bi-level Positive Airway Pressure	Sham CPAP
Arm/Group Description	BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Physiologic CPAP initiated for at least 16 hours per day for a minimum of 48hrs. Sham CPAP: Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Measure Participants	0	0

2. Secondary Outcome

Title	Rate of Exchange Transfusions.
Time Frame	Diagnosis until discharge. Average 7 days.

Outcome Measure Data

Analysis Population Description

Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.

Arm/Group Title	Bi-level Positive Airway Pressure	Sham CPAP
Arm/Group Description	BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Physiologic CPAP initiated for at least 16 hours per day for a minimum of 48hrs. Sham CPAP: Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Measure Participants	0	0

3. Secondary Outcome

Title	Determine Parent and Patient Acceptability of BLPAP Administration in the Setting of ACS.
Time Frame	Upon completion of intervention at 48hrs.

Outcome Measure Data

Analysis Population Description

Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.

Arm/Group Title	Bi-level Positive Airway Pressure	Sham CPAP
Arm/Group Description	BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Physiologic CPAP initiated for at least 16 hours per day for a minimum of 48hrs. Sham CPAP: Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Measure Participants	0	0

4. Secondary Outcome

Title	Rate of PCCU Transfers.
Time Frame	Diagnosis until discharge. Average 7 days.

Outcome Measure Data

Analysis Population Description

Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.

Arm/Group Title	Bi-level Positive Airway Pressure	Sham CPAP
Arm/Group Description	BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Physiologic CPAP initiated for at least 16 hours per day for a minimum of 48hrs. Sham CPAP: Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Measure Participants	0	0

5. Secondary Outcome

Title	Difference in Respiratory Rate.
Time Frame	48hrs after initiation of treatment

Outcome Measure Data

Analysis Population Description

Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.

Arm/Group Title	Bi-level Positive Airway Pressure	Sham CPAP
Arm/Group Description	BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Physiologic CPAP initiated for at least 16 hours per day for a minimum of 48hrs. Sham CPAP: Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Measure Participants	0	0

6. Secondary Outcome

Title	Difference in Pulmonary Function Tests.
Time Frame	48hrs after initiation of treatment

Outcome Measure Data

Analysis Population Description

Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.

Arm/Group Title	Bi-level Positive Airway Pressure	Sham CPAP
Arm/Group Description	BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Physiologic CPAP initiated for at least 16 hours per day for a minimum of 48hrs. Sham CPAP: Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Measure Participants	0	0

7. Secondary Outcome

Title	Difference in Mean SpO2 Recording During Sleep.
Description	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).
Time Frame	48hrs after initiation of treatment

Outcome Measure Data

Analysis Population Description

Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.

Arm/Group Title	Bi-level Positive Airway Pressure	Sham CPAP
Arm/Group Description	BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Physiologic CPAP initiated for at least 16 hours per day for a minimum of 48hrs. Sham CPAP: Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Measure Participants	0	0

Adverse Events

Time Frame	Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.
Adverse Event Reporting Description	Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.
Arm/Group Title	Bi-level Positive Airway Pressure		Sham CPAP
Arm/Group Description	BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.		Physiologic CPAP initiated for at least 16 hours per day for a minimum of 48hrs. Sham CPAP: Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.
All Cause Mortality
	Bi-level Positive Airway Pressure		Sham CPAP
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/0 (NaN)		0/0 (NaN)
Serious Adverse Events
	Bi-level Positive Airway Pressure		Sham CPAP
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/0 (NaN)		0/0 (NaN)
Other (Not Including Serious) Adverse Events
	Bi-level Positive Airway Pressure		Sham CPAP
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/0 (NaN)		0/0 (NaN)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.