NCT03754088

Brief Summary

In order to further enable physiopathology studies, the aim of this project is to validate an in vitro model of cystic fibrosis created using induced pluripotent stemcell (iPS) differentiated bronchial epithelium from cystic fibrosis (CF) patients homozygous for the p.Phe508del mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
6

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Nov 2018

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
completed

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 Start

First participant enrolled

November 5, 2018

Completed
17 days until next milestone

First Submitted

Initial submission to the registry

November 22, 2018

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 27, 2018

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 25, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 25, 2019

Completed
Last Updated

September 26, 2022

Status Verified

December 1, 2021

Enrollment Period

6 months

First QC Date

November 22, 2018

Last Update Submit

September 23, 2022

Conditions

Outcome Measures

Primary Outcomes (1)

  • Obtention of induced pluripotent stem cell line (iPS): yes/no

    Was a pluripotent stem cell line obtained? yes/no

    28 days

Secondary Outcomes (2)

  • Functional bronchial epithelium present for the iPS? yes/no

    28 days

  • Cystic fibrosis transmembrane conductance regulator (CFTR) channel function: yes/no

    28 days

Study Arms (2)

Cystic fibrosis

Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation.

Healthy subjects

Three healthy subjects.

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation and 3 healthy subjects without cystic fibrosis.

You may qualify if:

  • Homozygote for the p.Phe508del mutation
  • Signed informed consent given by the subject
  • Signed informed consent given by the subject

You may not qualify if:

  • Pregnancy, breastfeeding
  • Participant under any kind of guardianship
  • Unaffiliated with or not a beneficiary of a social security program (health insurance)
  • Subject deprived of liberty (e.g. prisoners)
  • Subject with positive infectious markers for HIV1, HIV2, HBC or HBV
  • Any pathology requiring a treatment or a pathology not requiring treatment but with clinical significance according to the investigator

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hôpital Arnaud de Villeneuve - CHU de Montpellier

Montpellier, 34295, France

Location

Biospecimen

Retention: SAMPLES WITH DNA

Blood sample, RNA and induced pluripotent stem cell lines.

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

  • Arnaud Bourdin, MD, PhD

    Montpellier University Hospitals

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 22, 2018

First Posted

November 27, 2018

Study Start

November 5, 2018

Primary Completion

April 25, 2019

Study Completion

April 25, 2019

Last Updated

September 26, 2022

Record last verified: 2021-12

Locations