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Completed

NCT Number: NCT01812551

Treatment of Low Bone Density in Cystic Fibrosis.

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.

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Key information

Conditions

Osteoporosis Bone Diseases Bone Diseases, Metabolic Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cystic Fibrosis Digestive System Diseases Fractures, Bone Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Metabolic Diseases Musculoskeletal Diseases Nutritional and Metabolic Diseases Pancreatic Diseases Respiratory Tract Diseases Wounds and Injuries

Age range

5 year–30 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

CRR Fibrosi Cistica, Divisione Gastroenterologia, Ospedale Bambin Gesù, Vatican City, Holy See

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Alendronate

Drug

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).

Other names: Alendros

Placebo

Drug

Placebo was distributed to the patients in plain bottles and boxes (bearing only the center and patient codes).

Primary outcomes

  1. Bone mineral density increase at lumbar spine.

    Time frame: up to 24 months

    Bone mineral density evaluated by DXA. Bone mineral apparent density calculated to correct for bone size (growing subjects). Z-score calculated.

    Measurements: Phase 1 (171 subjects): Baseline, 6 months, 12 months. Phase 2 (128 subjects, randomized to 2 arms: placebo or alendronate): 18 months, 24 months.

Secondary outcomes

  1. Changes in bone turnover markers.

    Time frame: baseline and up to 24 months

    Bone turnover markers: (serum) osteocalcin (OC), bone specific alkaline phosphatase (BSAP), C-terminal telopeptide of procollagen 1 (CTx); (urine) terminal telopeptide of procollagen 1 (NTx).

  2. Fracture rate.

    Time frame: at 12th and 24th month

    Appendicular fractures were evaluated at baseline (previous fractures) and throughout the 2 years of study (incident fractures) with X-rays.

    Vertebral fractures were evaluated at the end of Phase 1 (12th month) and at the end of Phase 2 (24th month) with lateral thoracic and lumbar spine X-rays.

  3. Adverse effects of alendronate.

    Time frame: continuously throughout Phase 2 (2nd year of study)

    Evaluated on the basis of lab tests (calcemia, calciuria, blood cell count, liver and kidney function), FEV1 changes, and other signs/symptoms (e.g. pain, fever, etc.)

Sponsors and collaborators

Lead sponsor

Istituto Auxologico Italiano

Other

Collaborators

  • Fondazione Telethon

Registry information

Official study title

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

Acronym: OSCYF

Important dates

Study start
2002
Primary completion
2006
Study completion
2007
First posted
Mar 18, 2013
Registry last updated
Mar 6, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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