Zoledronic Acid in Cystic Fibrosis

NCT01702415 | Withdrawn Phase 4 DMC

• 1 other identifier: P01612
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

Null hypotheses: zoledronic acid does not improve bone density in cystic fibrosis. Low bone mineral density (osteoporosis) is prevalent in adults with cystic fibrosis (CF); they have an increased rate of bone fractures in comparison to the general population. CF patients start to lose bone density in adolescence/early adulthood due to an imbalance in bone breakdown and formation. Predicted survival for patients with CF has increased from 16 years in 1970 to 36.5 years in 2009 which has resulted in an increase in comorbidities associated with increased longevity in CF e.g. decreased bone density. Oral and intravenous bisphophosphonates are known to increase bone density in CF; the current licensed oral preparations require daily or weekly dosing which are difficult to maintain. Zoledronate, which is licensed for use, is administered intravenously once a year which should be easier to administer. The current evidence relates to its use in other disease groups e.g. glucocorticoid induced osteoporosis and oncology. The purpose of this study is to ascertain its efficacy in cystic fibrosis.

Conditions

Cystic Fibrosis

Keywords

Cystic fibrosis; Bone density

Outcome Measures

Primary Outcomes (1)

• Bone density

  Bone density measure by DEXA

  12 months

Secondary Outcomes (1)

• Effect of zoledronic acid on the number of bone fractures

  12 months

Other Outcomes (1)

• Bone pain

  12 months

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Zoledronic acid

EXPERIMENTAL

Active IMP

Drug: Zoledronic acid

Interventions

Zoledronic acid DRUG

Single dose, intravenous solution 5mg

Zoledronic acid

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of cystic fibrosis
• Aged at least 18 years
• Bone mineral density score of -1.5 or less at lumbar spine or total hip
• Able to give informed consent

You may not qualify if:

• Previous solid organ transplant
• on solid organ transplant waiting list
• Long trem oral glucocorticosteroids
• CRP \>20mg on day of randomisation
• Hypocalcaemia
• Poor dental hygiene

Sponsors & Collaborators

• Papworth Hospital NHS Foundation Trust: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (1)

Papworth Hospital NHS Foundation Trust

Cambridge, Cambridgeshire, CB23 3RE, United Kingdom

Location

Related Publications (1)

• Jeffery TC, Chang AB, Conwell LS. Bisphosphonates for osteoporosis in people with cystic fibrosis. Cochrane Database Syst Rev. 2023 Jan 10;1(1):CD002010. doi: 10.1002/14651858.CD002010.pub5.

  PMID: 36625789 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Zoledronic Acid

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Diphosphonates; Organophosphonates; Organophosphorus Compounds; Organic Chemicals; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Charles Dr Haworth, FRCP

  Papworth Hospital NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 4, 2012
• First Posted: October 8, 2012
• Study Start: October 1, 2013
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: October 21, 2015

Record last verified: 2015-10

Locations

GB (1)