Cystic Fibrosis Related Bone Disease: the Role of CFTR

Description

The purpose of this study is to determine whether ivacaftor, a recently FDA-approved CFTR potentiator, improves bone micro-architecture and strength in patients with cystic fibrosis with at least one G551D CFTR mutation.

Study Start Date

April 2012

Estimated Completion Date

December 2017

Interventions

No interventions cited

Specialties

  • Internal Medicine: Clinical Pharmacology,Orthopedic/Physical Med,Pulmonology
  • Pulmonology: Clinical Pharmacology,Cystic Fibrosis
  • Orthopedics: Bone Metabolism,Clinical Pharmacology

MeSH Terms

  • Bone Diseases
  • CFTR
  • Cystic Fibrosis

Study ID

Massachusetts General Hospital -- MGH 2012P000269

Status

Unknown

Trial ID

NCT01549314

Study Type

Observational

Trial Phase

N/A

Enrollment Quota

90

Sponsor

Massachusetts General Hospital

COHORT 1

Inclusion Criteria

  • Age 6 to 75 years old
  • Established diagnosis of CF with at least one abnormal G551D-CFTR allele
  • Eligibility for and intent to start treatment with ivacaftor or started treatment with ivacaftor within previous 6 months

Exclusion Criteria

  • Psychiatric or mental incapacity that would preclude subject from assenting to study participation
  • Current pregnancy
  • History of organ transplantation
  • History of Burkholderia dolosa infection COHORT 2: Subjects will be grouped by gender, age and race to match subjects in Cohort 1 within two years. Pubertal subjects will be matched by Tanner stage.

    Inclusion Criteria

    • Age 6 to 75 years old
    • Established diagnosis of CF

    Exclusion Criteria

    • Psychiatric or mental incapacity that would preclude subject from assenting to study participation
    • Current pregnancy
    • History of organ transplantation
    • History of Burkholderia dolosa infection COHORT 3: Subjects will be grouped by gender, age and race to match subjects in Cohort 2 within two years. Pubertal subjects will be matched by Tanner stage. Inclusion criteria:
    • Age 6 to 75 years old
    • Clinically stable, deemed able to complete the screening, baseline, and scheduled study visits. Exclusion criteria:
    • History of significant cardiac, renal, pulmonary, hepatic, or malignant disease, current alcohol or illicit drug abuse, or major psychiatric disorder
    • Current diagnoses known to affect bone metabolism, including cystic fibrosis, osteoporosis, amenorrhea >3 months (in menstruating women who are not taking oral contraceptives or have an IUD), hyperthyroidism, diabetes, hyperparathyroidism, Paget's disease, kidney stones, chronic inflammatory diseases, malabsorptive disorders, malnutrition, prolonged immobility, and skeletal dysplasias
    • History of a non-digital fracture in the previous 6 months, history of one pathologic fracture, or greater than four total lifetime non-digital fractures
    • Cumulative lifetime use of oral glucocorticoids for greater than 2 months
    • Current or prior use of medications known to affect bone metabolism including hormone replacement therapy, anti-estrogens, bisphosphonates, calcitonin, fluoride, lithium, suppressive doses of levothyroxine, or anticonvulsants.
    • Pregnancy
    • BMI less than 18.5 or greater than 25 kg/m2 in subjects 18 years and older, or BMI less than 5th or greater than 85th percentile in subjects under the age of 18 years.
    • Any medical or psychiatric condition or situation that would compromise subject safety, informed consent/assent, treatment compliance, follow-up measurements, or data quality

Gender

Both

Ages

6 Years to 75 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Study Locations and Contact Information (1)

Study Location Distance Name Phone Email
Massachusetts General Hospital - Boston, Massachusetts 2.8 miles Melissa S Putman MD 857-218-5017 msputman@partners.org

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