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

This study has been completed.
Sponsor:
Collaborator:
Fondazione Telethon
Information provided by (Responsible Party):
Maria Luisa Bianchi, Istituto Auxologico Italiano
ClinicalTrials.gov Identifier:
NCT01812551
First received: March 14, 2013
Last updated: March 5, 2014
Last verified: March 2014

March 14, 2013
March 5, 2014
October 2002
July 2006   (final data collection date for primary outcome measure)
Bone mineral density increase at lumbar spine. [ Time Frame: up to 24 months ] [ Designated as safety issue: No ]

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.

Same as current
Complete list of historical versions of study NCT01812551 on ClinicalTrials.gov Archive Site
  • Changes in bone turnover markers. [ Time Frame: baseline and up to 24 months ] [ Designated as safety issue: No ]
    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).
  • Fracture rate. [ Time Frame: at 12th and 24th month ] [ Designated as safety issue: No ]

    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.

  • Adverse effects of alendronate. [ Time Frame: continuously throughout Phase 2 (2nd year of study) ] [ Designated as safety issue: Yes ]
    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.)
Same as current
Not Provided
Not Provided
 
Treatment of Low Bone Density in Cystic Fibrosis.
Osteoporosis in Cystic Fibrosis: Study of Bone Mass and Bone Metabolism, and Prospective Randomized Therapeutic Trial.

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.

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.

Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Osteoporosis
  • Cystic Fibrosis
  • 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).
    Other Name: Alendros
  • Drug: Placebo
    Placebo was distributed to the patients in plain bottles and boxes (bearing only the center and patient codes).
  • Active Comparator: Alendronate

    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.

    Intervention: Drug: Alendronate
  • Placebo Comparator: Placebo

    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).

    Intervention: Drug: Placebo

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
171
July 2007
July 2006   (final data collection date for primary outcome measure)

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.
Both
5 Years to 30 Years
No
Contact information is only displayed when the study is recruiting subjects
Holy See (Vatican City State),   Italy
 
NCT01812551
02A001
No
Maria Luisa Bianchi, Istituto Auxologico Italiano
Istituto Auxologico Italiano
Fondazione Telethon
Principal Investigator: Maria Luisa Bianchi, M.D. Istituto Auxologico Italiano
Istituto Auxologico Italiano
March 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP