Study of Circulating Blood Cell Monocytes as Predictive Biomarker of Bone Cystic Fibrosis Disease. Impact of CFTR Correctors. (MUCO-OS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03492567
Recruitment Status : Completed
First Posted : April 10, 2018
Last Update Posted : November 21, 2018
Vaincre la Mucoviscidose
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Brief Summary:

The aim of the study is to look for risk factors of developing osteoporosis in patients with cystic fibrosis.

Blood cells called monocytes will be obtained from a blood sample. These monocytes will make possible to form another class of cells called osteoclasts in the laboratory. These latter cells are responsible for bone degradation and therefore promote the increase of bone fragility. The research will focus on the characterization of these cells to better understand their potential (low, medium or high) to increase bone fragility in patients with cystic fibrosis.

Finally the action of CFTR modulators on these bone cells will be tested.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Biological: Blood test Not Applicable

Detailed Description:

The aim of the study is to characterize the profile of circulating monocytes as predictive biomarker of osteoclastogenesis in CF related bone disease.

Investigators will evaluate the presence and regulation of CD115 (MCSF receptor) and CD265 (RANK) membrane receptors of circulating monocytes of cystic fibrosis patients.

They will also analyze the impact of the F508del CFTR mutation on the differentiation of monocytes into osteoclasts. We will 1) Quantify the training and functional activity of osteoclasts and, 2) Test in vitro the effects of correctors and potentiators of CFTR on the resorption activity of differentiated osteoclasts.

The study will be explained to patients and they only will have one blood test.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Official Title: Study of Circulating Monocytes as Predictive Biomarker of Osteoclastogenesis in Cystic Fibrosis-related Bone Loss. Impact of CFTR Correctors.
Actual Study Start Date : April 25, 2018
Actual Primary Completion Date : July 4, 2018
Actual Study Completion Date : July 4, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Blood monocyte precursors/osteoclasts
Blood test
Biological: Blood test
Blood draw (max 15 ml)

Primary Outcome Measures :
  1. Phenotype of CD14 monocytes/osteoclasts [ Time Frame: 1 day ]

Secondary Outcome Measures :
  1. In vitro test of CFTR modulators [ Time Frame: 1 day ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • patients with cystic fibrosis
  • attending the Paris Cochin adult CF centre
  • with at least F508del mutation (homozygote or heterozygote patients)

Exclusion Criteria:

  • patients younger than 18
  • absence of signed consent
  • patients without a F508del mutation

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03492567

Cochin Hospital
Paris, France, 75014
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Vaincre la Mucoviscidose
Principal Investigator: Dominique HUBERT, PhD Assistance Publique - Hôpitaux de Paris

Responsible Party: Assistance Publique - Hôpitaux de Paris Identifier: NCT03492567     History of Changes
Other Study ID Numbers: K171004J
2017-A02965-48 ( Other Identifier: ID-RCB )
First Posted: April 10, 2018    Key Record Dates
Last Update Posted: November 21, 2018
Last Verified: March 2018

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Cystic fibrosis
CFTR modulators

Additional relevant MeSH terms:
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases