NCT02965326

Brief Summary

The purpose of this study is to determine which biological marker, or association of biological markers, best predict clinical response of cystic fibrosis patients to CFTR modulators.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
75

participants targeted

Target at P50-P75 for not_applicable

Timeline
5mo left

Started May 2016

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress96%
May 2016Oct 2026

Study Start

First participant enrolled

May 1, 2016

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

November 11, 2016

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 16, 2016

Completed
9.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2026

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2026

Expected
Last Updated

March 12, 2024

Status Verified

March 1, 2024

Enrollment Period

9.8 years

First QC Date

November 11, 2016

Last Update Submit

March 8, 2024

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

  • Forced Expiratory Volume in 1 second

    Respiratory Function test

    initiation, 7 days, 1 month, 6 months, 1 year and every 6 months

Secondary Outcomes (43)

  • Forced Vital Capacity

    initiation, 7 days, 1 month, 6 months, 1 year and every 6 months

  • Forced Expiratory Flow 25-75

    initiation, 7 days, 1 month, 6 months, 1 year and every 6 months

  • Residual Volume

    initiation, 7 days, 1 month, 6 months, 1 year and every 6 months

  • elastase in sputum

    initiation, 7 days, 1 month, 6 months, 1 year and every 6 months

  • calprotectin in sputum

    initiation, 7 days, 1 month, 6 months, 1 year and every 6 months

  • +38 more secondary outcomes

Study Arms (3)

Cystic fibrosis, treated

OTHER

Cystic fibrosis patients treated either by Ivacaftor or by the association Ivacaftor-Lumacaftor

Procedure: Nasal swab; rectal biopsy.

Cystic fibrosis, non treated

OTHER

Cystic fibrosis patients, non treated by a CFTR modulator

Procedure: Nasal swab; rectal biopsy.

Non-Cystic fibrosis

OTHER

Patients in whom cystic fibrosis diagnosis has been suspected, but excluded by physiological and genetic investigations

Procedure: Nasal swab; rectal biopsy.

Interventions

Nasal swab; rectal biopsy.PROCEDURE

Nasal epithelial cells will be obtained by nasal swabs from patients of the three arms; intestinal epithelial cells will be obtained, by rectal biopsy, only from patients treated by CFTR modulators.

Cystic fibrosis, non treatedCystic fibrosis, treatedNon-Cystic fibrosis

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Cystic fibrosis patients treated by CFTR modulators (Ivacaftor or the association Ivacaftor-Lumacaftor)
  • Cystic fibrosis patients non treated by CFTR modulators
  • Patients in whom cystic fibrosis diagnosis has been suspected, but excluded by physiological and genetic investigations

You may not qualify if:

  • pregnant or lactating women
  • contraindication to nasal swab
  • contraindication to rectal biopsy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Necker Hospital

Paris, 75014, France

RECRUITING

Related Publications (9)

  • Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordonez C, Elborn JS; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.

    PMID: 22047557BACKGROUND

  • Graeber SY, Hug MJ, Sommerburg O, Hirtz S, Hentschel J, Heinzmann A, Dopfer C, Schulz A, Mainz JG, Tummler B, Mall MA. Intestinal Current Measurements Detect Activation of Mutant CFTR in Patients with Cystic Fibrosis with the G551D Mutation Treated with Ivacaftor. Am J Respir Crit Care Med. 2015 Nov 15;192(10):1252-5. doi: 10.1164/rccm.201507-1271LE. No abstract available.

    PMID: 26568242BACKGROUND

  • Quittner A, Suthoff E, Rendas-Baum R, Bayliss MS, Sermet-Gaudelus I, Castiglione B, Vera-Llonch M. Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial. Health Qual Life Outcomes. 2015 Jul 2;13:93. doi: 10.1186/s12955-015-0293-6.

    PMID: 26135562BACKGROUND

  • De Boeck K, Munck A, Walker S, Faro A, Hiatt P, Gilmartin G, Higgins M. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros. 2014 Dec;13(6):674-80. doi: 10.1016/j.jcf.2014.09.005. Epub 2014 Sep 26.

    PMID: 25266159BACKGROUND

  • Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, Rodman D; VX09-809-102 study group. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24.

    PMID: 24973281BACKGROUND

  • Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA, Konstan MW, McColley SA, McCoy K, McKone EF, Munck A, Ratjen F, Rowe SM, Waltz D, Boyle MP; TRAFFIC Study Group; TRANSPORT Study Group. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. N Engl J Med. 2015 Jul 16;373(3):220-31. doi: 10.1056/NEJMoa1409547. Epub 2015 May 17.

    PMID: 25981758BACKGROUND

  • De Boeck K, Kent L, Davies J, Derichs N, Amaral M, Rowe SM, Middleton P, de Jonge H, Bronsveld I, Wilschanski M, Melotti P, Danner-Boucher I, Boerner S, Fajac I, Southern K, de Nooijer RA, Bot A, de Rijke Y, de Wachter E, Leal T, Vermeulen F, Hug MJ, Rault G, Nguyen-Khoa T, Barreto C, Proesmans M, Sermet-Gaudelus I; European Cystic Fibrosis Society Clinical Trial Network Standardisation Committee. CFTR biomarkers: time for promotion to surrogate end-point. Eur Respir J. 2013 Jan;41(1):203-16. doi: 10.1183/09031936.00057512. Epub 2012 Aug 9.

    PMID: 22878883BACKGROUND

  • Dekkers JF, Wiegerinck CL, de Jonge HR, Bronsveld I, Janssens HM, de Winter-de Groot KM, Brandsma AM, de Jong NW, Bijvelds MJ, Scholte BJ, Nieuwenhuis EE, van den Brink S, Clevers H, van der Ent CK, Middendorp S, Beekman JM. A functional CFTR assay using primary cystic fibrosis intestinal organoids. Nat Med. 2013 Jul;19(7):939-45. doi: 10.1038/nm.3201. Epub 2013 Jun 2.

    PMID: 23727931BACKGROUND

  • Pranke I, Hatton A, Masson A, Flament T, Le Bourgeois M, Chedevergne F, Bailly C, Urbach V, Hinzpeter A, Edelman A, Sermet-Gaudelus I. Might Brushed Nasal Cells Be a Surrogate for CFTR Modulator Clinical Response? Am J Respir Crit Care Med. 2019 Jan 1;199(1):123-126. doi: 10.1164/rccm.201808-1436LE. No abstract available.

    PMID: 30326728DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Officials

  • Aleksander Edelman, phD

    APHP

    STUDY DIRECTOR

Central Study Contacts

Isabelle Sermet, MD, PhD

CONTACT

33 1 44 49 48 87isabelle.sermet@aphp.fr

Jean-Louis Pérignon, MD, PhD

CONTACT

jean-louis.perignon@aphp.fr

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

November 11, 2016

First Posted

November 16, 2016

Study Start

May 1, 2016

Primary Completion

March 1, 2026

Study Completion (Estimated)

October 1, 2026

Last Updated

March 12, 2024

Record last verified: 2024-03

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)