Cystic FibrosisBIO-CFTRNCT02965326
Surrogate Markers of Response to New Therapies in Cystic Fibrosis Patients
A study testing Nasal swab; rectal biopsy. in people with Cystic Fibrosis, run by Hôpital Necker-Enfants Malades at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Nasal swab; rectal biopsy.
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 1 siteFrance
- Study dates
- May 2016to Oct 2026
- Phase
- Interventional
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- pregnant or lactating women
- contraindication to nasal swab
- contraindication to rectal biopsy
Trial locations and contacts
1 site in 1 country.
Jean-Louis Pérignon, MD, PhD contact
France1 site, 1 recruiting
- Recruiting
Necker Hospital
Paris, 75014
SERMET Isabelle, Professor, 01 44 49 48 87, isabelle.sermet@nck.aphp.fr
LE Bourgeois Muriel, MD, 01 44 49 48 87, isabelle.sermet@nck.aphp.fr
About this study
Official title: Personalized Therapy of Cystic Fibrosis: Set-up of Response Markers
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.
Read the detailed description
This study is based upon the hypothesis that clinical response of cystic fibrosis patients to CFTR modulators is correlated to in vitro responses to these drugs of epithelial cells derived from the patients, as assessed by CFTR-dependent Chloride secretion. Epithelial cells will be derived either from nasal or rectal epithelia, and consist both of cultured cells and organoids. The drugs tested will be Ivacaftor, or Lumacaftor/Ivacaftor, according to patient's treatment. Results of these assays will be compared with response to treatment at 6 and 12 months, assessed by clinical response and in vivo assay of CFTR function.
- Conditions
- Cystic Fibrosis
- Being tested
- Nasal swab; rectal biopsy.
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nasal swab; rectal biopsy.Procedure or surgery
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.
| Study group | Role | What they receive |
|---|---|---|
| Cystic fibrosis, treated | Other | Cystic fibrosis patients treated either by Ivacaftor or by the association Ivacaftor-Lumacaftor Receives: Procedure: Nasal swab; rectal biopsy. |
| Cystic fibrosis, non treated | Other | Cystic fibrosis patients, non treated by a CFTR modulator Receives: 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 Receives: Procedure: Nasal swab; rectal biopsy. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Forced Expiratory Volume in 1 second
Respiratory Function test
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
Secondary outcomes (43)
Forced Vital Capacity
Respiratory Function test
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
Forced Expiratory Flow 25-75
Respiratory Function test
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
Residual Volume
Respiratory Function test
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
elastase in sputum
elastase activity in UI/g of sputum
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
calprotectin in sputum
calprotectin in µg/g of sputum
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
IL-8 in sputum
IL-8 in µg/g of sputum
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
Calprotectin in blood
calprotectin in µg/ml of blood
Measured: initiation, 1 month, 3 Months, 6 months and every 6 months
IL-8 in blood
IL-8 in µg/ml of blood
Measured: initiation, 1 month, 3 Months, 6 months and every 6 months
tumor necrosis factor in sputum
tumor necrosis factor in µg/g of sputum
Measured: initiation, 1 month, 3 Months, 6 months and every 6 months
liver function test in blood
serum glutamate oxaloacetate transaminase in ui/ml
Measured: initiation, 1 month, 3 Months, 6 months and every 6 months
liver function test/SGPT in blood
serum glutamate pyruvate transaminase in ui/ml
Measured: initiation, 1 month, 3 Months, 6 months and every 6 months
liver function test/bilirubin in blood
Bilirubin in mg/ml
Measured: initiation, 1 month, 3 Months, 6 months and every 6 months
liver function test, gammaGT in blood
GammaGT in UI/ml
Measured: initiation, 1 month, 3 Months, 6 months and every 6 months
creatine phosphokinase in blood
CPK in mg/ml
Measured: initiation, 1 month, 3 Months, 6 months and every 6 months
Amylase in blood
amylase in mg/ml
Measured: initiation, 1 month, 3 Months, 6 months and every 6 months
Impedancemetry
ambulatory measurement of body composition
Measured: initiation , 1 month, 3 Months, 6 months and every 6 months
Dynamometry
ambulatory measurement of quadriceps strength
Measured: initiation , 1 month, 3 Months, 6 months and every 6 months
Sweat test
chloride concentration in sweat
Measured: initiation ,1 month
proteomics of sweat
measurement of proteins in sweat
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
metabolomics of sweat
measurement of métabolites in sweat
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
proteomics of exhalate
measurement of proteins in exhaled air
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
sputum bronchial microorganism colonization
bacterial, fungi and viral colonization
Measured: initiation , 1 month, 3 Months, 6 months and every 6 months
Lung MRI
Lung Imaging evaluation : number of bronchiectasis, number of mucus plugs
Measured: initiation, at 1 year and every year
osteodensitometry
Bone mineralization body composition
Measured: initiation, 1 year, and every year
fecal elastase
elastase feces in µg/g feces
Measured: initiation, at 6 months and every year
fecal calprotectin
calprotectin, concentraion in feces in µg/g
Measured: initiation, at 6 months and every year
Chest CT scan
Lung Imaging: % versuys normal of lung parenchuma with bronchiectasis, airway wall thickening, mucus plugs, air trapping
Measured: initiation, 3 years and 5 years
glycemic Holter
glycemia monitoring
Measured: initiation, 1 year and every year
abdominal ultrasonography
presence of liver hyperechogenicity, fibrosis, as assessed by the radiologist
Measured: treatment initiation, 1 year and every year
proton density fat fraction
magnetic resonance Imaging of the Pancreas
Measured: treatment initiation, 1 year
patient quality of life
Score tolerance of the treatment, perception of respiratory, digestive symptoms, energy, body image as assessed by the "Cystic Fibrosis Questionnaire" score a better quality of life is indicated by an increase in the score value. Minimum value is 0, maximum is 100.
Measured: initiation, 1 month, 6 months, 1 year and every 6 months
CFTR activity in nasal cells/chloride
Chloride transport in primary nasal cell cultures obtained by nasal brushing and study in Ussing chamber (µA/cm2)
Measured: initiation of treatment and repeated if cell culture failure
CFTR activity in nasal cells/bicarbonat
Bicarbonate transport in primary nasal cell cultures obtained by nasal brushing and study in Ussing chamber (µA/cm2)
Measured: initiation of treatment and repeated if cell culture failure
CFTR activity in intestinal epithelium/chloride
Chloride transport in intestinal primary culture and study in Ussing chamber (µA/cm2)
Measured: initiation of treatment and repeated of cell culture failure
CFTR activity in intestinal epithelium/bicarbonate
Bicarbonate transport in intestinal primary culture and study in Ussing chamber (µA/cm2)
Measured: initiation of treatment and repeated of cell culture failure
sweat evaporimetry
quantity of sweat produced afer bet-adrenergic stimulation after subcutaneous injection
Measured: initiation and 1 month
Elasto MRI
measurement of liver fibrosis by MRI
Measured: initiation and 1 year
Lung Clearance Index
Capacity of the lung to washout pure Oxygen,
Measured: initiation, 6 months, 1 year and every 6 months
proteomics in blood
measurement of proteins in blood
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every year
metabolomics in blood
measurement of métabolites in blood
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every year
proteomic in urine
measurement of proteins in blood
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every year
metabolomics in urine
measurement of métabolites in urine
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every year
Exhaled air composition
Volatile organic compounds in exhaled air
Measured: initiation, 7 days, 1 month, 6 months, 1 year and every year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2016 (estimated)
- Study startMay 2016
- Primary completionMar 2026 (estimated)
- Study completionOct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hôpital Necker-Enfants Malades
- Sponsor type
- University, hospital or other
- Collaborators
- Association Mucoviscidose-ABCF2, Vaincre la Mucoviscidose
- Responsible party
- Principal investigator, Isabelle Sermet-Gaudelus, Hôpital Necker-Enfants Malades
- Investigators
- Aleksander Edelman, phD, Study director, APHP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02965326
- Sponsor's protocol ID
- ABCF-2016-01
- Other IDs
- 2016-A00309-42
- Acronym
- BIO-CFTR
- Status last verified
- Mar 2024
- First submitted
- 11 Nov 2016
- Last updated
- 12 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
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.
PubMed 22047557Graeber 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.
PubMed 26568242Quittner 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.
PubMed 26135562De 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.
PubMed 25266159Boyle 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.
PubMed 24973281Wainwright 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.
PubMed 25981758De 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.
PubMed 22878883Dekkers 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.
PubMed 23727931Pranke 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.
PubMed 30326728
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Personalized Therapy of Cystic Fibrosis: Set-up of Response Markers
- ClinicalTrials.gov ID
- NCT02965326
- Acronym / sponsor ID
- BIO-CFTR / ABCF-2016-01
- Conditions
- Cystic Fibrosis
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Hôpital Necker-Enfants MaladesUniversity, hospital or other
- Collaborators
- Association Mucoviscidose-ABCF2, Vaincre la Mucoviscidose
- Study start
- May 2016 (actual)
- Primary completion
- Mar 2026 (estimated)
- Study completion
- Oct 2026 (estimated)
- First posted
- 16 Nov 2016 (estimated)
- 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.
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.
Detailed description
This study is based upon the hypothesis that clinical response of cystic fibrosis patients to CFTR modulators is correlated to in vitro responses to these drugs of epithelial cells derived from the patients, as assessed by CFTR-dependent Chloride secretion. Epithelial cells will be derived either from nasal or rectal epithelia, and consist both of cultured cells and organoids. The drugs tested will be Ivacaftor, or Lumacaftor/Ivacaftor, according to patient's treatment. Results of these assays will be compared with response to treatment at 6 and 12 months, assessed by clinical response and in vivo assay of CFTR function.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cystic fibrosis, treated | Other | Procedure: Nasal swab; rectal biopsy.Cystic fibrosis patients treated either by Ivacaftor or by the association Ivacaftor-Lumacaftor |
| Cystic fibrosis, non treated | Other | Procedure: Nasal swab; rectal biopsy.Cystic fibrosis patients, non treated by a CFTR modulator |
| Non-Cystic fibrosis | Other | Procedure: Nasal swab; rectal biopsy.Patients in whom cystic fibrosis diagnosis has been suspected, but excluded by physiological and genetic investigations |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- pregnant or lactating women
- contraindication to nasal swab
- contraindication to rectal biopsy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nasal swab; rectal biopsy.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. | Procedure or surgery | Cystic fibrosis, non treated, Cystic fibrosis, treated, Non-Cystic fibrosis |
Primary outcome
Forced Expiratory Volume in 1 second
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
Respiratory Function test
43 secondary outcomes
Forced Vital Capacity
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
Forced Expiratory Flow 25-75
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
Residual Volume
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
elastase in sputum
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
calprotectin in sputum
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
IL-8 in sputum
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
Calprotectin in blood
Time frame: initiation, 1 month, 3 Months, 6 months and every 6 months
IL-8 in blood
Time frame: initiation, 1 month, 3 Months, 6 months and every 6 months
tumor necrosis factor in sputum
Time frame: initiation, 1 month, 3 Months, 6 months and every 6 months
liver function test in blood
Time frame: initiation, 1 month, 3 Months, 6 months and every 6 months
liver function test/SGPT in blood
Time frame: initiation, 1 month, 3 Months, 6 months and every 6 months
liver function test/bilirubin in blood
Time frame: initiation, 1 month, 3 Months, 6 months and every 6 months
liver function test, gammaGT in blood
Time frame: initiation, 1 month, 3 Months, 6 months and every 6 months
creatine phosphokinase in blood
Time frame: initiation, 1 month, 3 Months, 6 months and every 6 months
Amylase in blood
Time frame: initiation, 1 month, 3 Months, 6 months and every 6 months
Impedancemetry
Time frame: initiation , 1 month, 3 Months, 6 months and every 6 months
Dynamometry
Time frame: initiation , 1 month, 3 Months, 6 months and every 6 months
Sweat test
Time frame: initiation ,1 month
proteomics of sweat
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
metabolomics of sweat
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
proteomics of exhalate
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months
sputum bronchial microorganism colonization
Time frame: initiation , 1 month, 3 Months, 6 months and every 6 months
Lung MRI
Time frame: initiation, at 1 year and every year
osteodensitometry
Time frame: initiation, 1 year, and every year
fecal elastase
Time frame: initiation, at 6 months and every year
fecal calprotectin
Time frame: initiation, at 6 months and every year
Chest CT scan
Time frame: initiation, 3 years and 5 years
glycemic Holter
Time frame: initiation, 1 year and every year
abdominal ultrasonography
Time frame: treatment initiation, 1 year and every year
proton density fat fraction
Time frame: treatment initiation, 1 year
patient quality of life
Time frame: initiation, 1 month, 6 months, 1 year and every 6 months
CFTR activity in nasal cells/chloride
Time frame: initiation of treatment and repeated if cell culture failure
CFTR activity in nasal cells/bicarbonat
Time frame: initiation of treatment and repeated if cell culture failure
CFTR activity in intestinal epithelium/chloride
Time frame: initiation of treatment and repeated of cell culture failure
CFTR activity in intestinal epithelium/bicarbonate
Time frame: initiation of treatment and repeated of cell culture failure
sweat evaporimetry
Time frame: initiation and 1 month
Elasto MRI
Time frame: initiation and 1 year
Lung Clearance Index
Time frame: initiation, 6 months, 1 year and every 6 months
proteomics in blood
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every year
metabolomics in blood
Time frame: initiation, 7 days, 1 month, 6 months, 1 year and every year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2024.
- Background: PubMed 22047557Ramsey 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.
- Background: PubMed 26568242Graeber 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.
- Background: PubMed 26135562Quittner 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.
- Background: PubMed 25266159De 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.
- Background: PubMed 24973281Boyle 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.
- Background: PubMed 25981758Wainwright 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.
- Background: PubMed 22878883De 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.
- Background: PubMed 23727931Dekkers 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.
- Derived from this study: PubMed 30326728Pranke 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.
- Individual participant dataSharing planned: No
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Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
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.
Jean-Louis Pérignon, MD, PhD contact
Aleksander Edelman, phD study director
APHP
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Nasal swab; rectal biopsy. in people with Cystic Fibrosis, run by Hôpital Necker-Enfants Malades 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 3 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: status not confirmed, as of the sponsor's last update on 12 Mar 2024. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Isabelle Sermet, MD, PhD (33 1 44 49 48 87, isabelle.sermet@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Forced Expiratory Volume in 1 second (time frame: initiation, 7 days, 1 month, 6 months, 1 year and every 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02965326, which also lists 9 linked publications.
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 12 Mar 2024.