Treatment of Low Bone Density in Cystic Fibrosis. (OSCYF)

Istituto Auxologico Italiano

Status and phase: Completed; Phase 3

Conditions: Osteoporosis; Cystic Fibrosis

Treatments: Drug: Alendronate; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT01812551

02A001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 171 patients

Sex: All

Ages: 5 to 30 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• age 2-30 years
• clinically stable condition
• regular menses in females
• low Bone Mineral Apparent Density for age (defined as BMAD Z-score ≤-2.0 if age ≤18 years or ≤-2.5 if age >18 years).

Exclusion criteria

• two or more episodes of hypercalcemia and/or hypercalciuria
• contraindications to 25-OH vitamin D or alendronate treatment
• recent transplantation
• other diseases or medications (glucocorticoids excepted) associated with bone loss.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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171 participants in 2 patient groups, including a placebo group

Alendronate

Active Comparator group

Description:

128 subjects participated in the study's Phase 2 (1-year double-blind, randomized, placebo-controlled, parallel group study). 65 subjects were randomized to this arm. Oral alendronate dose: 5 mg/day, if body weight ≤25 kg; 10 mg/day, if body weight \>25 kg.

Treatment:

Drug: Alendronate

Placebo

Placebo Comparator group

Description:

128 subjects participated in the study's Phase 2 (1-year double-blind, randomized, placebo-controlled, parallel group study). 63 subjects were randomized to this arm. Oral placebo (inactive pills).

Treatment:

Drug: Placebo