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Drug & research development » Clinical trials » NCT01549314

Cystic Fibrosis Related Bone Disease: the Role of CFTR

Official title: Cystic Fibrosis Related Bone Disease: the Role of CFTR

Clinical Trials gov number: NCT01549314

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Purpose: To determine whether ivacaftor improves bone micro-architecture and strength in CF patients with ≥1 G551D CFTR mutation.

Phase: NA

Type: Observational, prospective, cohort

Study sponsor: Massachusetts General Hospital; Children’s Hospital Boston

 

Cohort 1

– Established diagnosis of CF with ≥1 abnormal G551D-CFTR allele

– Eligibility for and intent to start treatment with ivacaftor or started treatment with ivacaftor within previous 6 months

Cohort 2

– Grouped by gender, age and race to match subjects in Cohort 1 within 2 years

– Pubertal subjects will be matched by Tanner stage

– Established diagnosis of CF

Cohort 3

– Grouped by gender, age and race to match subjects in Cohort 2 within 2 years

– Pubertal subjects will be matched by Tanner stage

– Clinically stable, deemed able to complete the screening, baseline, and scheduled study visits

Intervention
Ivacaftor

Geographical Location
United States

Number of Participants
51-100 (6-75 years)

Primary Endpoint

Bone microarchitecture and strength measures of the radius and tibia at 24 months

Secondary Endpoint

At 24 months

  • Areal bone mineral density as measured by DXA
  • Bone turnover markers

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Cohort 1 will receive ivacaftor, other cohorts will not be treated

View Trial Results