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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by Duke University
Sponsor:
Information provided by (Responsible Party):
Duke University
ClinicalTrials.gov Identifier:
NCT01895998
First received: July 8, 2013
Last updated: April 8, 2014
Last verified: March 2014
  Purpose

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 Intervention Phase
Sickle Cell Disease
Drug: Albuterol
Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Diagnostic
Official Title: Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease

Resource links provided by NLM:


Further study details as provided by Duke University:

Primary Outcome Measures:
  • Presence of obstructive or restrictive lung disease [ Time Frame: One testing session- approximately 3 hours total ] [ Designated as safety issue: No ]
    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:
  • Presence of pulmonary hypertension [ Time Frame: one testing session- approximately 15 minutes ] [ Designated as safety issue: No ]
    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.


Estimated Enrollment: 32
Study Start Date: August 2013
Estimated Study Completion Date: February 2020
Estimated Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
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.
  Eligibility

Ages Eligible for Study:   5 Years to 12 Years
Genders Eligible for Study:   Both
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01895998

Contacts
Contact: Stacey L Peterson-Carmichael, MD 919-668-4760 stacey.peterson-carmichael@duke.edu
Contact: Kathy Auten, MSHS 919-684-2281 kathy.auten@duke.edu

Locations
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
Contact: Stacey Peterson-Carmichael, MD    919-668-4760    stacey.peterson-carmichael@duke.edu   
Contact: Kathy Auten, MSHS    919-684-2281    kathy.auten@duke.edu   
Sponsors and Collaborators
Duke University
Investigators
Principal Investigator: Stacey Peterson-Carmichael, MD Duke University
  More Information

No publications provided

Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT01895998     History of Changes
Other Study ID Numbers: Pro00040933
Study First Received: July 8, 2013
Last Updated: April 8, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Duke University:
sickle cell disease
pulmonary hypertension
airway hyperreactivity
acute chest syndrome

Additional relevant MeSH terms:
Anemia, Sickle Cell
Hypertension
Hypertension, Pulmonary
Lung Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Vascular Diseases
Cardiovascular Diseases
Respiratory Tract Diseases
Albuterol
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Pharmacologic Actions
Anti-Asthmatic Agents
Respiratory System Agents
Therapeutic Uses
Tocolytic Agents
Reproductive Control Agents
Adrenergic beta-2 Receptor Agonists
Adrenergic beta-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on August 19, 2014