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

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

Tracking Information
First Submitted Date  ICMJE July 8, 2013
First Posted Date  ICMJE July 11, 2013
Last Update Posted Date December 3, 2014
Study Start Date  ICMJE August 2013
Actual Primary Completion Date April 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (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  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE 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  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 2, 2014)
5
Original Estimated Enrollment  ICMJE
 (submitted: July 8, 2013)
32
Actual Study Completion Date  ICMJE April 2014
Actual Primary Completion Date April 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01895998
Other Study ID Numbers  ICMJE Pro00040933
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Duke University
Study Sponsor  ICMJE Duke University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Stacey Peterson-Carmichael, MD Duke University
PRS Account Duke University
Verification Date December 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP