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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Albuterol Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease
Study Start Date : August 2013
Actual Primary Completion Date : April 2014
Actual Study Completion Date : April 2014



Intervention Details:
  • 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.


Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   5 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01895998


Locations
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United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Duke University
Investigators
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Principal Investigator: Stacey Peterson-Carmichael, MD Duke University
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Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT01895998    
Other Study ID Numbers: Pro00040933
First Posted: July 11, 2013    Key Record Dates
Last Update Posted: December 3, 2014
Last Verified: December 2014
Keywords provided by Duke University:
sickle cell disease
pulmonary hypertension
airway hyperreactivity
acute chest syndrome
Additional relevant MeSH terms:
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Lung Diseases
Hypertension, Pulmonary
Hypertension
Anemia, Sickle Cell
Vascular Diseases
Cardiovascular Diseases
Respiratory Tract Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Albuterol
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Tocolytic Agents
Reproductive Control Agents
Adrenergic beta-2 Receptor Agonists
Adrenergic beta-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action