Cystic Fibrosis Related Bone Disease: the Role of CFTR
Completed
Conditions studied: Cystic Fibrosis Related Bone Disease
In brief
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.
Key facts
- Study ID
- NCT01549314
- Run by
- Massachusetts General Hospital
- People needed
- 79
- Starts
- 2012-04-01
- Expected to finish
- 2020-02-01
- Last updated by the study team
- 2020-04-01
Who can join
Age: 6 and older, up to 75. Sex: any. Healthy volunteers: accepted.
You may qualify if…
- 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
You may not qualify if…
- 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.
Where it is running
- Massachusetts General Hospital — Boston, Massachusetts, United States
Full record on ClinicalTrials.gov
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