Bone Microarchitecture at the Radius: a Pilot Comparison Between Children With Cystic Fibrosis and Healthy Controls

NCT01331980 | Completed DMC

• 1 other identifier: 113442
• Study Type: observational
• Target: 39
• Locations: 1 country
• Sites: 1

Brief Summary

Cystic fibrosis (CF) affects an estimated 30,000 people in the United States and is caused by a mutation in the gene encoding a protein called CF transmembrane regulator (CFTR). The hallmarks of CF are recurrent pulmonary exacerbations and declining pulmonary function. However, there are other problems in CF that affect both health and quality of life. These include CF related diabetes, liver disease, and bone disease. The median age of survival for patients with CF has been increasing steadily and is currently more than 37 years. With this improvement in life expectancy, it has become increasingly important to address the long-term complications of CF. Currently, patients with CF are evaluated annually for bone disease with dual X-ray absorptiometry (DXA), and screening usually starts at age 12. However, this may not be sufficient to detect early bone changes that may impact fracture risk. Furthermore, bone disease in children may manifest earlier than adolescence, which would suggest that screening should start at an earlier age in these vulnerable patients. The following study is therefore proposed to examine the potential role of peripheral quantitative computed tomography (pQCT) as a screening approach for bone disease in children with CF. The investigators expect to find bone problems by pQCT but not DXA.

Conditions

Cystic Fibrosis

Keywords

cystic fibrosis; bone

Outcome Measures

Primary Outcomes (3)

• Peripheral quantitative computed tomography (pQCT) - cortex width

  pQCT scan of the non-dominant radius at the 4% and 65% sites will be conducted and parameters will include cortex width, trabecular bone mineral density (BMD), and total BMD

  Day 1

• pQCT parameters - trabecular BMD

  pQCT scan of the non-dominant radius at the 4% and 65% sites will be conducted and parameters will include cortex width, trabecular BMD, and total BMD

  Day 1

• pQCT parameters - total BMD

  pQCT scan of the non-dominant radius at the 4% and 65% sites will be conducted and parameters will include cortex width, trabecular BMD, and total BMD.

  Day 1

Study Arms (2)

cystic fibrosis

children aged 6-12 years of age and Tanner stage 1 with a diagnosis of cystic fibrosis

Device: Peripheral quantitative computed tomography (pQCT); Device: Dual X-ray absorptiometry (DXA)

healthy controls

children ages 6-12 years and Tanner stage 1 without cystic fibrosis or other chronic disease that affects bone health

Device: Peripheral quantitative computed tomography (pQCT); Device: Dual X-ray absorptiometry (DXA)

Interventions

Peripheral quantitative computed tomography (pQCT) DEVICE

A pQCT scan of the non-dominant radius at the 4% and 65% sites will be conducted at a single study visit

Also known as: Stratec XCT 2000 series pQCT

cystic fibrosis; healthy controls

Dual X-ray absorptiometry (DXA) DEVICE

A total body DXA scan will be conducted at a single study visit

Also known as: Hologic QDR 4500A

cystic fibrosis; healthy controls

Eligibility Criteria

• Age: 6 Years - 12 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Participants for the cystic fibrosis (CF) group will be recruited from the pulmonary clinic at Arkansas Children's Hospital. Healthy controls will be recruited from Arkansas Children's Hospital outpatient clinics and the community.

You may qualify if:

• Diagnosis of CF by sweat test and/or genotyping for CF subjects (for CF group only)
• years of age at time of study visit
• Body mass index of at least the 3rd percentile
• Tanner stage 1

You may not qualify if:

• Body mass index (BMI) greater than the 95th percentile
• Recent fracture (within the past 6 months)
• Lung transplant recipient
• Current pulmonary exacerbation or current infection
• History of bisphosphonate or growth hormone therapy (in the past 5 years)
• Glucocorticoid therapy within the past 6 months
• Severe pulmonary dysfunction (forced expiratory volume in 1 second \< 40% predicted) if subjects are performing spirometry
• Concomitant disease known to cause bone disease (e.g. chronic kidney disease, CF-related diabetes)
• Inability or unwillingness of individual or legal guardian/representative to give written informed consent

Sponsors & Collaborators

• University of Arkansas: lead

Study Sites (1)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Serum samples will be frozen and assayed together.

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Absorptiometry, Photon

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Radiography; Diagnostic Imaging; Diagnostic Techniques and Procedures; Diagnosis; Densitometry; Photometry; Chemistry Techniques, Analytical; Investigative Techniques

Study Officials

• Catherine O'Brien, PharmD

  University of Arkansas for Medical Sciences and Arkansas Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2011
• First Posted: April 8, 2011
• Study Start: January 1, 2011
• Primary Completion: July 1, 2015
• Study Completion: July 1, 2015
• Last Updated: July 3, 2015

Record last verified: 2015-07

Locations

US (1)