Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease

• ClinicalTrials.gov Identifier: NCT01895998
• Recruitment Status: Completed
• First Posted: July 11, 2013
• Last Update Posted: December 3, 2014
• Sponsor: Duke University
• Information provided by (Responsible Party): Duke University

Tracking Information

• First Submitted Date: July 8, 2013
• First Posted Date: July 11, 2013
• Last Update Posted Date: December 3, 2014
• Study Start Date: August 2013
• Actual Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 8, 2013)

Presence of obstructive or restrictive lung disease [ Time Frame: One testing session- approximately 3 hours total ]

Obstructive lung disease will be defined as forced expiratory volume in 1 second (FEV1), or forced vital capacity (FVC) < 80% predicted based on normative data for age, sex, height, weight and race. Restrictive lung disease will be defined as total lung capacity (TLC) <80% predicted based on normative data for age, sex, height, weight and race.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: July 8, 2013)

Presence of pulmonary hypertension [ Time Frame: one testing session- approximately 15 minutes ]

Pulmonary hypertension will be defined as a tricuspid regurgitation (TR) jet gradient that predicts a pulmonary artery (PA) systolic pressure greater than half of the systemic systolic pressure.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease
• Official Title: Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease

Brief Summary

The proposed research study is a cross-sectional study enrolling young children with sickle cell disease between 5 and 12 years of age. They will be screened as outpatients for consent to perform pulmonary function testing (PFT) and echocardiography. In addition, the degree of bronchodilator response will be assessed at each session. To estimate presence of pulmonary hypertension, echocardiography will be performed at the time of PFT measures.

Study Design:

1. Enroll children aged 5 to 12 years of age with sickle cell disease (HbSS, HbSC, HbS beta plus thalassemia, HbS beta zero thalassemia, and HbS OArab) who are established patients within the Duke Pediatric Sickle Cell Clinic.
2. Perform a chart review of all enrolled subjects to obtain specific details regarding birth history, nutritional status (weight, height), family history, sickle cell genotype, parental smoking history, recent laboratory parameters, parental smoking history, any concurrent conditions (atopy, asthma, airway anomaly), history of sickle cell complications and prescribed medications.
3. Perform spirometry and plethysmography with the administration of albuterol.
4. Before or after completion the PFT session, the patient will have echocardiography in the PFT lab area
5. Using medical record information, determine number of hospitalizations for any pulmonary symptoms indicative of acute chest syndrome (ACS) (dyspnea, fever, wheezing, hypoxia, cough, chest pain). In addition, we will track any respiratory or cardiac symptoms or therapies for each subject 6 years after enrollment up to age 18 years using the registry.
6. As standard of care, refer any child identified as having lung disease or pulmonary hypertension to a pediatric pulmonologist and/or cardiologist for monitoring, treatment and ongoing care.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Diagnostic
• Condition: Sickle Cell Disease

Intervention

Drug: Albuterol

This medication will be used as routine part of pulmonary function testing to assess for airway hyperreactivity. This is routinely done in a clinical capacity for pediatric patients with various pulmonary disease processes.

• Study Arms: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: December 2, 2014): 5
• Original Estimated Enrollment (submitted: July 8, 2013): 32
• Actual Study Completion Date: April 2014
• Actual Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• children aged 5 to 12 years of age with sickle cell disease (HbSS, HbSC, HbS beta plus thalassemia, HbS beta zero thalassemia, and HbS OArab)
• established patients within the Duke Pediatric Sickle Cell Clinic.
• Subjects must have been full-term at birth
• any race or gender

Exclusion Criteria:

• significant chromosomal/congenital anomalies
• hemodynamically significant congenital heart disease (arrhythmia requiring medication, defects with chronic hypoxia, single ventricle physiology, heart failure)
• any child within 3 weeks of a respiratory tract infection, an asthma attack, an episode of ACS or of a vaso-occlusive or hemolytic crisis.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 5 Years to 12 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01895998
• Other Study ID Numbers: Pro00040933
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Duke University
• Original Responsible Party: Same as current
• Current Study Sponsor: Duke University
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Stacey Peterson-Carmichael, MD; Duke University

• PRS Account: Duke University
• Verification Date: December 2014