Cystic FibrosisNCT05576324
Effect of the CFTR-modulating Triple Therapy Elexacaftor - Tezacaftor - Ivacaftor
An observational study of Cystic Fibrosis, run by University of Erlangen-Nürnberg Medical School at 1 site in Germany.
Trial summary
The aim of this study is to investigate the frequency distribution, cytokine profile and function of peripheral, mononuclear leukocyte populations (monocytes, NK cells, T/B lymphocytes) and their correlation to clinical and biochemical parameters in patients with cystic fibrosis receiving CFTR modulatory triple… Read more about this study
- Who is it for?
- Ages 6 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Elexacaftor / Ivacaftor / Tezacaftor
- How many people
- 130 participants
- Where
- 1 siteGermany
- Study dates
- 30 Dec 2020to 18 Oct 2023
- 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 6 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: All patients who are under the care of the CF Center Erlangen at the time of the study and who meet the inclusion criteria will be considered for the patient collective. Recruitment of healthy subjects will be performed during routine blood sampling in patients with pulmonary disease during outpatient appointments as well as during inpatient stays after prior informed consent has been obtained.
Who may be able to join
- Patients (m/f/d) with molecularly genetically confirmed cystic fibrosis aged 6 years and older.
- Do not meet any of the exclusion criteria
- Written informed consent
- For study arm "Kaftrio® ongoing": Kaftrio® therapy for at least 6 months
- For study arm "Kaftrio® longitudinal": no Kaftrio® therapy started yet
Who cannot join
- Use of inhaled or systemic glucocorticoids as part of a permanent medication regimen
- Pregnancy
Trial locations and contacts
1 site in 1 country.
Germany1 site, 1 recruiting
- Recruiting
University Hospital Erlange, Department of Pediatrics
Erlangen, Bavaria, 91054
Alexander Schnell, Dr. med., +4991318533118, alexander.schnell@uk-erlangen.de
André Hörning, PD Dr. med., +4991318533118, andre.hoerning@uk-erlangen.de
About this study
Official title: Effect of the CFTR-modulating Triple Therapy Elexacaftor - Tezacaftor - Ivacaftor on Numerical Distribution in Peripheral Mononuclear Immune Cells Derived From Patients With Cystic Fibrosis
The aim of this study is to investigate the frequency distribution, cytokine profile and function of peripheral, mononuclear leukocyte populations (monocytes, NK cells, T/B lymphocytes) and their correlation to clinical and biochemical parameters in patients with cystic fibrosis receiving CFTR modulatory triple therapy consisting of elexacaftor, tezacaftor and ivacaftor and to compare it with patients without CFTR modulatory therapy and healthy control subjects.
Read the detailed description
The therapy of cystic fibrosis usually consists of an inhalative therapy with hy-pertonic saline and other mucolytics (e.g. dornase alpha) for secretolysis as well as a pancreatic enzyme replacement therapy. In recent years, however, the introduction of novel drugs, the so-called CFTR modulators, has revolutionized the previous treatment concept of a symptom-oriented therapy. Ivacaftor, which was approved by the FDA in 2012 for the treatment of patients with G551D mutation, causes a prolongation of the opening probability of the CFTR channel (CFTR potentiator) and was able to show a significant improvement in lung function in studies. By combining ivacaftor with the CFTR corrector lumacaftor, which improves the processing of the CFTR channel in the endoplasmic reticulum as well as its incorporation into the cell membrane, this therapeutic strategy has also been successfully tested for use in patients with F508del homozygous mutation. Also, the combination of ivacaftor with another CFTR corrector, tezacaftor, was approved for the treatment of patients with F508del heterozygous mutations in which the second mutation was classified as a mutation with residual activity and was able to show an increase in FEV1. The efficacy of this therapeutic approach was further enhanced by the combination of ivacaftor as a CFTR potentiator with tezacaftor and a next-generation CFTR corrector, elexacaftor; in the pivotal study, an improvement in FEV1 of an average of 14 points in untreated patients and 11 points in ivacaftor/tezacaftor-pretreated patients was demonstrated, as well as a significant decrease in hospitalizations due to pulmonary exacerbation. Since September 2020 in the European Union, this combination has been approved under the trade name Kaftrio® for the treatment of patients with F508del homozygous mutation or F508del heterozygous mutation and minimal function mutation. This form of therapy is based on a concept that comes closest to a causal therapy. In April 2021, the EMA granted approval for the drug for all patients older than 12 years and with evidence of at least one F508del mutation. In addition, the manufacturer applied for an extension of the approval in the EU for children aged 6-11 years based on the also very positive study results and received a positive decision from the European Medicines Agency (EMA) in November 2021. However, in addition to the clear role of the CFTR channel in epithelial tissues, it has been increasingly shown in recent years that the CFTR channel is also expressed by a variety of immune cells of the innate as well as the acquired immune system, such as neutrophils, macrophages, monocytes, and B and T lymphocytes. Its absence or dysfunction in cystic fibrosis seems to trigger a disturbed regulation or an exaggerated reaction of various immune responses.
- Conditions
- Cystic Fibrosis
- Being tested
- Elexacaftor / Ivacaftor / Tezacaftor
- Keywords
- Cystic fibrosis, Clinical parameters, Immune cell function
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Elexacaftor / Ivacaftor / TezacaftorDrug
Also called ETI, Kaftrio®
Elexacaftor / Ivacaftor / Tezacaftor is a triple drug therapy that modulates CFTR availability at (apical) cell membranes and increases opening probability.
| Study group | Role | What they receive |
|---|---|---|
| longitudinal | Inclusion of patients with diagnosed CF prior ETI therapy, follow-up visit after 6 months Receives: Drug: Elexacaftor / Ivacaftor / Tezacaftor | |
| under ETI | Patients with diagnosed CF already receiving ETI therapy for 6 months Receives: Drug: Elexacaftor / Ivacaftor / Tezacaftor | |
| no ETI | Patients with diagnosed CF that have refused an ETI treatment or are not eligible for ETI therapy | |
| Healthy Individuals | Healthy, age- and gender-matched probands |
What is being measured
The primary outcome is the main question the study is designed to answer.
Peripheral Blood Immunograms
Relative and absolute peripheral blood immune cell count as determined by multicolor flow cytometry
Measured: prior ETI
Peripheral Blood Immunograms
Relative and absolute peripheral blood immune cell count as determined by multicolor flow cytometry
Measured: 6 months ETI
Secondary outcomes (45)
Shear Wave Velocity (SWV)
Difference of mean/median SWV in treated vs. untreated patients as measured by Acoustic Radiation Force Impulse Imaging (ARFI)
Measured: prior ETI
Shear Wave Velocity (SWV)
Difference of mean/median SWV in treated vs. untreated patients as measured by Acoustic Radiation Force Impulse Imaging (ARFI)
Measured: 6 months ETI
Attenuation Coefficient (AC)
Difference of mean/median AC in treated vs. untreated pediatric patients (6-11 yrs) as measured by Ultrasound-guided attenuation parameter (UGAP)
Measured: prior ETI
Attenuation Coefficient (AC)
Difference of mean/median AC in treated vs. untreated pediatric patients (6-11 yrs) as measured by Ultrasound-guided attenuation parameter (UGAP)
Measured: 6 months ETI
Serum bile acids
Level of serum bile acids as measured by mass spectrometry
Measured: prior ETI
Serum bile acids
Level of serum bile acids as measured by mass spectrometry
Measured: 6 months ETI
Respiratory function test (FEV1, FVC)
Respiratory function test (FEV1, FVC) as measured by bodyplethysmography
Measured: prior ETI
Respiratory function test (FEV1, FVC)
Respiratory function test (FEV1, FVC) as measured by bodyplethysmography
Measured: 6 months ETI
Blood cell count
Blood cell count as defined in x10^3/µl
Measured: prior ETI
Blood cell count
Blood cell count as defined in x10^3/µl
Measured: 6 months ETI
Erythrocytoid hemoglobin A1c
Erythrocytoid hemoglobin A1c in %
Measured: prior ETI
Erythrocytoid hemoglobin A1c
Erythrocytoid hemoglobin A1c in %
Measured: 6 months ETI
Plasma electrolytes
Plasma electrolytes (Na, Cl) as defined by mmol/l
Measured: 6 months ETI
Liver transaminases
Aspartate/Alanine aminotransferase (AST, ALt) as defined in U/l
Measured: prior ETI
Liver transaminases
Aspartate/Alanine aminotransferase (AST, ALt) as defined in U/l
Measured: 6 months ETI
Plasmatic bilirubin
Total and direct plasmatic bilirubin as defined in mg/dl
Measured: prior ETI
Plasmatic bilirubin
Total and direct plasmatic bilirubin as defined in mg/dl
Measured: 6 months ETI
Prothrombin time
Prothrombin time as defined by Quick percent
Measured: prior ETI
Prothrombin time
Prothrombin time as defined by Quick percent
Measured: 6 months ETI
Coagulation factors
Vitamin K- dependent coagulation factors (II, VII, IX, X) as measured in %
Measured: prior ETI
Coagulation factors
Vitamin K- dependent coagulation factors (II, VII, IX, X) as measured in %
Measured: 6 months ETI
Plasmatic albumine
Plasma levels of albumine (defined in g/dl)
Measured: prior ETI
Plasmatic albumine
Plasma levels of albumine (defined in g/dl)
Measured: 6 months ETI
Plasmatic C-reactive proteine
Plasmatic C-reactive proteine (defined in mg/l)
Measured: prior ETI
Plasmatic C-reactive proteine
Plasmatic C-reactive proteine (defined in mg/l)
Measured: 6 months ETI
Plasmatic cholinesterase
Plasmatic cholinesterase (defined in U/l)
Measured: prior ETI
Plasmatic cholinesterase
Plasmatic cholinesterase (defined in U/l)
Measured: 6 months ETI
Plasmatic glutamate dehydrogenase
Plasmatic glutamate dehydrogenase (defined in U/l)
Measured: 6 months ETI
Plasmatic creatinin
Plasmatic creatinin (defined in mg/dl)
Measured: prior ETI
Plasmatic creatinin
Plasmatic creatinin (defined in mg/dl)
Measured: 6 months ETI
Serum immunoglobulins
Serum immunoglobulins G, A, M, E (defined in g/l)
Measured: prior ETI
Serum immunoglobulins
Serum immunoglobulins G, A, M, E (defined in g/l)
Measured: 6 months ETI
Sweat chloride
Sweat chloride level (defined in mmol/l)
Measured: prior ETI
Sweat chloride
Sweat chloride level (defined in mmol/l)
Measured: 6 months ETI
BMI
BMI in kg/m^2
Measured: prior ETI
BMI
BMI in kg/m^2
Measured: 6 months ETI
Age
Patients' age in years
Measured: prior ETI
Microbial colonization status
Microbial colonization status as defined by microbiological reports
Measured: prior ETI
Microbial colonization status
Microbial colonization status as defined by microbiological reports
Measured: 6 months ETI
Individual concomitant medication regime
Individual concomitant medication regime
Measured: prior ETI
Individual concomitant medication regime
Individual concomitant medication regime
Measured: 6 months ETI
Functional pulmonary magnetic resonance imaging
Ventilation defect, perfusion defect, combined defects in longitudinal pediatric cohort (6-11 yrs)
Measured: prior ETI
Functional pulmonary magnetic resonance imaging
Ventilation defect, perfusion defect, combined defects in longitudinal pediatric cohort (6-11 yrs)
Measured: 6 months ETI
Neutrophilic dihydrorhodamine assay
Assay for determination of neutrophilic reactive oxygen species (measured as stimulation index)
Measured: prior ETI
Neutrophilic dihydrorhodamine assay
Assay for determination of neutrophilic reactive oxygen species (measured as stimulation index)
Measured: 6 months ETI
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2022
- Study start30 Dec 2020
- Primary completion18 Oct 2023 (estimated)
- Study completion18 Oct 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University of Erlangen-Nürnberg Medical SchoolThe 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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 130 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Erlangen-Nürnberg Medical School
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Alexander Schnell, University of Erlangen-Nürnberg Medical School
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05576324
- Sponsor's protocol ID
- CF-Immuno
- Status last verified
- Oct 2022
- First submitted
- 27 Sep 2022
- Last updated
- 12 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Keating D, Marigowda G, Burr L, Daines C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Sass LA, Tullis E, McKee CM, Moskowitz SM, Robertson S, Savage J, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Taylor-Cousar JL; VX16-445-001 Study Group. VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1612-1620. doi: 10.1056/NEJMoa1807120. Epub 2018 Oct 18.
PubMed 30334692Zemanick ET, Taylor-Cousar JL, Davies J, Gibson RL, Mall MA, McKone EF, McNally P, Ramsey BW, Rayment JH, Rowe SM, Tullis E, Ahluwalia N, Chu C, Ho T, Moskowitz SM, Noel S, Tian S, Waltz D, Weinstock TG, Xuan F, Wainwright CE, McColley SA. A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele. Am J Respir Crit Care Med. 2021 Jun 15;203(12):1522-1532. doi: 10.1164/rccm.202102-0509OC.
PubMed 33734030McDonald TV, Nghiem PT, Gardner P, Martens CL. Human lymphocytes transcribe the cystic fibrosis transmembrane conductance regulator gene and exhibit CF-defective cAMP-regulated chloride current. J Biol Chem. 1992 Feb 15;267(5):3242-8.
PubMed 1371114Schnell A, Jungert J, Klett D, Hober H, Kaiser N, Ruppel R, Geppert A, Tremel C, Sobel J, Plattner E, Schmitt-Grohe S, Zirlik S, Strobel D, Neurath MF, Knieling F, Rauh M, Woelfle J, Hoerning A, Regensburger AP. Increase of liver stiffness and altered bile acid metabolism after triple CFTR modulator initiation in children and young adults with cystic fibrosis. Liver Int. 2023 Apr;43(4):878-887. doi: 10.1111/liv.15544. Epub 2023 Feb 28.
PubMed 36797990
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of the CFTR-modulating Triple Therapy Elexacaftor - Tezacaftor - Ivacaftor on Numerical Distribution in Peripheral Mononuclear Immune Cells Derived From Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT05576324
- Acronym / sponsor ID
- CF-Immuno
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic fibrosis, Clinical parameters, Immune cell function
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 130 participants (estimated)
- Lead sponsor
- University of Erlangen-Nürnberg Medical SchoolUniversity, hospital or other
- Study start
- 30 Dec 2020 (actual)
- Primary completion
- 18 Oct 2023 (estimated)
- Study completion
- 18 Oct 2023 (estimated)
- First posted
- 12 Oct 2022 (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.
The aim of this study is to investigate the frequency distribution, cytokine profile and function of peripheral, mononuclear leukocyte populations (monocytes, NK cells, T/B lymphocytes) and their correlation to clinical and biochemical parameters in patients with cystic fibrosis receiving CFTR modulatory triple therapy consisting of elexacaftor, tezacaftor and ivacaftor and to compare it with patients without CFTR modulatory therapy and healthy control subjects.
Detailed description
The therapy of cystic fibrosis usually consists of an inhalative therapy with hy-pertonic saline and other mucolytics (e.g. dornase alpha) for secretolysis as well as a pancreatic enzyme replacement therapy. In recent years, however, the introduction of novel drugs, the so-called CFTR modulators, has revolutionized the previous treatment concept of a symptom-oriented therapy. Ivacaftor, which was approved by the FDA in 2012 for the treatment of patients with G551D mutation, causes a prolongation of the opening probability of the CFTR channel (CFTR potentiator) and was able to show a significant improvement in lung function in studies. By combining ivacaftor with the CFTR corrector lumacaftor, which improves the processing of the CFTR channel in the endoplasmic reticulum as well as its incorporation into the cell membrane, this therapeutic strategy has also been successfully tested for use in patients with F508del homozygous mutation. Also, the combination of ivacaftor with another CFTR corrector, tezacaftor, was approved for the treatment of patients with F508del heterozygous mutations in which the second mutation was classified as a mutation with residual activity and was able to show an increase in FEV1. The efficacy of this therapeutic approach was further enhanced by the combination of ivacaftor as a CFTR potentiator with tezacaftor and a next-generation CFTR corrector, elexacaftor; in the pivotal study, an improvement in FEV1 of an average of 14 points in untreated patients and 11 points in ivacaftor/tezacaftor-pretreated patients was demonstrated, as well as a significant decrease in hospitalizations due to pulmonary exacerbation. Since September 2020 in the European Union, this combination has been approved under the trade name Kaftrio® for the treatment of patients with F508del homozygous mutation or F508del heterozygous mutation and minimal function mutation. This form of therapy is based on a concept that comes closest to a causal therapy. In April 2021, the EMA granted approval for the drug for all patients older than 12 years and with evidence of at least one F508del mutation. In addition, the manufacturer applied for an extension of the approval in the EU for children aged 6-11 years based on the also very positive study results and received a positive decision from the European Medicines Agency (EMA) in November 2021. However, in addition to the clear role of the CFTR channel in epithelial tissues, it has been increasingly shown in recent years that the CFTR channel is also expressed by a variety of immune cells of the innate as well as the acquired immune system, such as neutrophils, macrophages, monocytes, and B and T lymphocytes. Its absence or dysfunction in cystic fibrosis seems to trigger a disturbed regulation or an exaggerated reaction of various immune responses.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| longitudinal | – | Drug: Elexacaftor / Ivacaftor / TezacaftorInclusion of patients with diagnosed CF prior ETI therapy, follow-up visit after 6 months |
| under ETI | – | Drug: Elexacaftor / Ivacaftor / TezacaftorPatients with diagnosed CF already receiving ETI therapy for 6 months |
| no ETI | – | –Patients with diagnosed CF that have refused an ETI treatment or are not eligible for ETI therapy |
| Healthy Individuals | – | –Healthy, age- and gender-matched probands |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All patients who are under the care of the CF Center Erlangen at the time of the study and who meet the inclusion criteria will be considered for the patient collective. Recruitment of healthy subjects will be performed during routine blood sampling in patients with pulmonary disease during outpatient appointments as well as during inpatient stays after prior informed consent has been obtained.
Key inclusion criteria (5 listed)
- Patients (m/f/d) with molecularly genetically confirmed cystic fibrosis aged 6 years and older.
- Do not meet any of the exclusion criteria
- Written informed consent
- For study arm "Kaftrio® ongoing": Kaftrio® therapy for at least 6 months
- For study arm "Kaftrio® longitudinal": no Kaftrio® therapy started yet
Key exclusion criteria (2 listed)
- Use of inhaled or systemic glucocorticoids as part of a permanent medication regimen
- Pregnancy
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 |
|---|---|---|
| Elexacaftor / Ivacaftor / TezacaftorAlso: ETI, Kaftrio®Elexacaftor / Ivacaftor / Tezacaftor is a triple drug therapy that modulates CFTR availability at (apical) cell membranes and increases opening probability. | Drug | longitudinal, under ETI |
Primary outcome
Peripheral Blood Immunograms
Time frame: prior ETI
Relative and absolute peripheral blood immune cell count as determined by multicolor flow cytometry
Peripheral Blood Immunograms
Time frame: 6 months ETI
Relative and absolute peripheral blood immune cell count as determined by multicolor flow cytometry
45 secondary outcomes
Shear Wave Velocity (SWV)
Time frame: prior ETI
Shear Wave Velocity (SWV)
Time frame: 6 months ETI
Attenuation Coefficient (AC)
Time frame: prior ETI
Attenuation Coefficient (AC)
Time frame: 6 months ETI
Serum bile acids
Time frame: prior ETI
Serum bile acids
Time frame: 6 months ETI
Respiratory function test (FEV1, FVC)
Time frame: prior ETI
Respiratory function test (FEV1, FVC)
Time frame: 6 months ETI
Blood cell count
Time frame: prior ETI
Blood cell count
Time frame: 6 months ETI
Erythrocytoid hemoglobin A1c
Time frame: prior ETI
Erythrocytoid hemoglobin A1c
Time frame: 6 months ETI
Plasma electrolytes
Time frame: 6 months ETI
Liver transaminases
Time frame: prior ETI
Liver transaminases
Time frame: 6 months ETI
Plasmatic bilirubin
Time frame: prior ETI
Plasmatic bilirubin
Time frame: 6 months ETI
Prothrombin time
Time frame: prior ETI
Prothrombin time
Time frame: 6 months ETI
Coagulation factors
Time frame: prior ETI
Coagulation factors
Time frame: 6 months ETI
Plasmatic albumine
Time frame: prior ETI
Plasmatic albumine
Time frame: 6 months ETI
Plasmatic C-reactive proteine
Time frame: prior ETI
Plasmatic C-reactive proteine
Time frame: 6 months ETI
Plasmatic cholinesterase
Time frame: prior ETI
Plasmatic cholinesterase
Time frame: 6 months ETI
Plasmatic glutamate dehydrogenase
Time frame: 6 months ETI
Plasmatic creatinin
Time frame: prior ETI
Plasmatic creatinin
Time frame: 6 months ETI
Serum immunoglobulins
Time frame: prior ETI
Serum immunoglobulins
Time frame: 6 months ETI
Sweat chloride
Time frame: prior ETI
Sweat chloride
Time frame: 6 months ETI
BMI
Time frame: prior ETI
BMI
Time frame: 6 months ETI
Age
Time frame: prior ETI
Microbial colonization status
Time frame: prior ETI
Microbial colonization status
Time frame: 6 months ETI
Individual concomitant medication regime
Time frame: prior ETI
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2022.
- Background: PubMed 30334692Keating D, Marigowda G, Burr L, Daines C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Sass LA, Tullis E, McKee CM, Moskowitz SM, Robertson S, Savage J, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Taylor-Cousar JL; VX16-445-001 Study Group. VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1612-1620. doi: 10.1056/NEJMoa1807120. Epub 2018 Oct 18.
- Background: PubMed 33734030Zemanick ET, Taylor-Cousar JL, Davies J, Gibson RL, Mall MA, McKone EF, McNally P, Ramsey BW, Rayment JH, Rowe SM, Tullis E, Ahluwalia N, Chu C, Ho T, Moskowitz SM, Noel S, Tian S, Waltz D, Weinstock TG, Xuan F, Wainwright CE, McColley SA. A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele. Am J Respir Crit Care Med. 2021 Jun 15;203(12):1522-1532. doi: 10.1164/rccm.202102-0509OC.
- Background: PubMed 1371114McDonald TV, Nghiem PT, Gardner P, Martens CL. Human lymphocytes transcribe the cystic fibrosis transmembrane conductance regulator gene and exhibit CF-defective cAMP-regulated chloride current. J Biol Chem. 1992 Feb 15;267(5):3242-8.
- Derived from this study: PubMed 36797990Schnell A, Jungert J, Klett D, Hober H, Kaiser N, Ruppel R, Geppert A, Tremel C, Sobel J, Plattner E, Schmitt-Grohe S, Zirlik S, Strobel D, Neurath MF, Knieling F, Rauh M, Woelfle J, Hoerning A, Regensburger AP. Increase of liver stiffness and altered bile acid metabolism after triple CFTR modulator initiation in children and young adults with cystic fibrosis. Liver Int. 2023 Apr;43(4):878-887. doi: 10.1111/liv.15544. Epub 2023 Feb 28.
- Individual participant dataSharing planned: No
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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.
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 of Erlangen-Nürnberg Medical School at 1 site in Germany.
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 5 inclusion and 2 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 Oct 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 Germany. 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 Alexander Schnell, Dr. med. (+499131/8533118, alexander.schnell@uk-erlangen.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Peripheral Blood Immunograms (time frame: prior ETI). The study lists 2 primary outcomes in all. 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 NCT05576324, which also lists 4 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 Oct 2022.