Cystic FibrosisPaCyFICNCT03754088
In Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via iPS Technology
An observational study of Cystic Fibrosis, run by University Hospital, Montpellier at 1 site in France.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 6 participants
- Where
- 1 siteFrance
- Study dates
- 5 Nov 2018to 25 Apr 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation and 3 healthy subjects without cystic fibrosis.
Who may be able to join
- Homozygote for the p.Phe508del mutation
- Signed informed consent given by the subject
- Signed informed consent given by the subject
Who cannot join
- Pregnancy, breastfeeding
- Participant in an exclusion period determined by a previous study
- 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
Trial locations and contacts
1 site in 1 country.
France1 site
Hôpital Arnaud de Villeneuve - CHU de Montpellier
Montpellier, 34295
About this study
Official title: Initiation of an in Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via Induced Pluripotent Stem Cell Technology
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.
Read the detailed description
The investigator's primary objective is to generate iPS lines from 3 CF patients and from 3 healthy subjects.
Secondary objectives include verification that cell lines express the CFTR gene according to their genotype, verification or relative production of the CFTR protein for each iPS line, and amplification of obtain iPS lines for aliquot creation to facilitate sharing.
- Conditions
- Cystic Fibrosis
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cystic fibrosis | Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation. | |
| Healthy subjects | Three healthy subjects. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Obtention of induced pluripotent stem cell line (iPS): yes/no
Was a pluripotent stem cell line obtained? yes/no
Measured: 28 days
Secondary outcomes (2)
Functional bronchial epithelium present for the iPS? yes/no
Was a functional bronchial epithelium present for the iPS? yes/no
Measured: 28 days
Cystic fibrosis transmembrane conductance regulator (CFTR) channel function: yes/no
CFTR channel function demonstrated via response following exposure to modified oligonucleotides targeting the mutated CFTR transcript.
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2018
- Study start5 Nov 2018
- Primary completion25 Apr 2019
- Study completion25 Apr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University Hospital, MontpellierThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Montpellier
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Arnaud Bourdin, MD, PhD, Principal investigator, Montpellier University Hospitals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03754088
- Sponsor's protocol ID
- RECHMPL17_0254
- Other IDs
- 2017-A02341-52
- Acronym
- PaCyFIC
- Status last verified
- Dec 2021
- First submitted
- 22 Nov 2018
- Last updated
- 26 Sep 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Initiation of an in Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via Induced Pluripotent Stem Cell Technology
- ClinicalTrials.gov ID
- NCT03754088
- Acronym / sponsor ID
- PaCyFIC / RECHMPL17_0254
- Conditions
- Cystic Fibrosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- University Hospital, MontpellierUniversity, hospital or other
- Study start
- 5 Nov 2018 (actual)
- Primary completion
- 25 Apr 2019 (actual)
- Study completion
- 25 Apr 2019 (actual)
- First posted
- 27 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The investigator's primary objective is to generate iPS lines from 3 CF patients and from 3 healthy subjects.
Secondary objectives include verification that cell lines express the CFTR gene according to their genotype, verification or relative production of the CFTR protein for each iPS line, and amplification of obtain iPS lines for aliquot creation to facilitate sharing.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cystic fibrosis | – | –Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation. |
| Healthy subjects | – | –Three healthy subjects. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Three cystic fibrosis patients who are homozygous for the p.Phe508del mutation and 3 healthy subjects without cystic fibrosis.
Key inclusion criteria (3 listed)
- Homozygote for the p.Phe508del mutation
- Signed informed consent given by the subject
- Signed informed consent given by the subject
Key exclusion criteria (7 listed)
- Pregnancy, breastfeeding
- Participant in an exclusion period determined by a previous study
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Obtention of induced pluripotent stem cell line (iPS): yes/no
Time frame: 28 days
Was a pluripotent stem cell line obtained? yes/no
2 secondary outcomes
Functional bronchial epithelium present for the iPS? yes/no
Time frame: 28 days
Cystic fibrosis transmembrane conductance regulator (CFTR) channel function: yes/no
Time frame: 28 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Arnaud Bourdin, MD, PhD principal investigator
Montpellier University Hospitals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by University Hospital, Montpellier at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Obtention of induced pluripotent stem cell line (iPS): yes/no (time frame: 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Sep 2022.