NCT01812551

Treatment of Low Bone Density in Cystic Fibrosis.

Osteoporosis in Cystic Fibrosis: Study of Bone Mass and Bone Metabolism, and Prospective Randomized Therapeutic Trial.

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 171 (actual)
• Sponsor: Istituto Auxologico Italiano · Academic / Other
• Sex: All
• Age: 5 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

Cystic fibrosis (CF) -- an autosomal recessive genetic disease affecting about 60,000 individuals worldwide, including about 3,800 in Italy -- is often associated with low bone mineral mass. The current aggressive therapies have ensured a much longer survival of CF patients but this has led to a higher frequency of osteoporosis and bone fractures, a serious problem which not only affects quality of life, but also hinders further therapeutic measures. The aim of this study, conducted on a large group of children, adolescents and young adults with CF, has been the evaluation of bone mass changes after 1 year of a simple treatment with RDA-adjusted dietary calcium plus 25-OH vitamin D supplementation, and the feasibility and efficacy of alendronate treatment (for another year) in patients not responding to calcium + 25-OH vitamin D alone.

Detailed description

The study included 2 phases. Phase 1 (1-year open-label observational study): following baseline evaluation, bone mass changes have been studied with a simple therapy of adequate calcium intake and 25-OH vitamin D supplements in all eligible subjects (N=171). Phase 2 (1-year double-blind, randomized, placebo-controlled, parallel group study): the 128 subjects showing an insufficient response to calcium + 25-OH vitamin D alone (bone mass increase \<5%) at the end of Phase 1, were randomized into 2 groups and assigned to alendronate treatment (N=65) or placebo (N=63) (in addition to calcium and 25-OH vitamin D as during Phase 1). The study has been carried out by the Coordinator's Institution (Istituto Auxologico Italiano)in collaboration with most Regional Reference Centers for CF in Italy.

Conditions

• Osteoporosis
• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	Alendronate	As active drug, we used Alendros (Abiogen Pharma, Pisa, Italy), distributed to the patients in plain bottles and boxes (bearing only the center and patient codes).
DRUG	Placebo	Placebo was distributed to the patients in plain bottles and boxes (bearing only the center and patient codes).

Timeline

• Start date: 2002-10-01
• Primary completion: 2006-07-01
• Completion: 2007-07-01
• First posted: 2013-03-18
• Last updated: 2014-03-06

Locations

11 sites across 2 countries: Holy See, Italy