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Novel outcome measures for clinical trials in cystic fibrosis

Harm A W M Tiddens1, Michael Puderbach2, Jose G Venegas3

  • 1Department of Pediatric Pulmonology and Allergology, Department of Radiology, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.

Pediatric Pulmonology
|February 3, 2015
PubMed

Insights

This review summarizes novel outcome measures for cystic fibrosis (CF) clinical trials. It highlights advances in assessing disease progression and treatment response, addressing limitations of current endpoints.

Area of Science:

  • Pulmonology
  • Genetics
  • Clinical Trials

Background:

  • Cystic fibrosis (CF) is an inherited disorder caused by CF transmembrane regulator gene mutations.
  • Understanding CF molecular mechanisms and therapies necessitates improved outcome measures.
  • Current regulatory endpoints for CF trials have limitations.

Purpose of the Study:

  • To discuss novel endpoints for cystic fibrosis clinical trials.
  • To critically evaluate the pros and cons of potential new outcome measures.
  • To summarize advances informing the development of CF clinical trial endpoints.

Main Methods:

  • A workshop was held in November 2011 to discuss novel CF trial endpoints.
  • The review critically evaluated novel outcome measures for CF treatments.
  • Subsequent technological and technical advances were considered.

Main Results:

  • Novel outcome measures were discussed for assessing CF disease progression.
  • The utility of these measures for evaluating new CF therapies was evaluated.
  • Advances in technology and techniques relevant to endpoint development were highlighted.

Conclusions:

  • There is a need for novel outcome measures in cystic fibrosis clinical trials.
  • Advances in technology support the development of better CF endpoints.
  • This review summarizes key discussions and subsequent developments in CF trial endpoint research.
Keywords:
CFTR activitycystic fibrosisendpointsimagingoutcome measuressputum biomarkers

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