Cystic FibrosisPhase 2CHOICESNCT06468527
Clinical Trial to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Adults With CF
A Phase 2 study testing Diponecaftor in people with Cystic Fibrosis, run by Kors van der Ent at 16 sites in 9 countries.
Trial summary
CF is caused by mutations in the gene that encodes the 'Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)' channel. To re-establish the function of this complex chloride channel, typically two to three drug modes of action are needed. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Diponecaftor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 41 participants
- Where
- 16 sitesin 9 countries
- Study dates
- 3 Jun 2024to 26 Jun 2025
- Phase
- Phase 2
- 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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A subject must meet ALL the following criteria in order to participate:
- Male or female subjects who completed the HIT-CF Organoid Study and are 18 years of age or older on the date of informed consent
- Confirmed diagnosis of CF as follows:
- Sweat chloride value of ≥60 mmol/L based on quantitative pilocarpine iontophoresis (at screening) OR 2 CF-causing mutations AND
- chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
- Clinically stable CF disease in the opinion of the investigator with no significant changes in health status within 28 days prior to Day 1
Show all 14Show fewer
- Forced expiratory volume in one second (FEV1) ≥40% of predicted to ≤90% of predicted at the Screening Visit, based on the Global Lung Function Initiative (GLI) -2012 multi-ethnic all-age reference equations
- Body mass index (BMI) ≥16 kg/m2 and ≤30 kg/m2
- Non-smoker and non-tobacco user (including all inhalational nicotine delivery systems) for a minimum of 30 days prior to screening, and subject agrees not to smoke or use tobacco for the duration of the study
- Subjects of childbearing potential must meet contraception requirements (Section 13.3.1)
- Willing to remain on a stable medication regimen for CF from 28 days before Day 1 through the last study visit
- Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, and other study procedures
- Selected by an unblinded coordinating team based on organoid response or random selection
- Subject will sign and date an informed consent form (ICF
Who cannot join
- A subject who meets ANY of the following criteria will be excluded from participation:
- Subject has at least one of the following CFTR-mutations: F508del, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H, A455E, 3849+10kbC>T OR A combination of any two of the following mutations: any nonsense mutation, 1717-1G>A, 621+1G>T, 3120+1G>A, 1898+1G->A, CFTRdele2,3, and 2183AA->G
- History or current evidence of any clinically significant cardiac (eg, heart failure, left ventricular hypertrophy, myocardial infarction, and arrhythmia), endocrinologic, hematologic, hepatobiliary (eg, clinically significant cirrhosis with or without portal hypertension, hepatitis B or hepatitis C), immunologic, metabolic, urologic, pulmonary (besides CF), neurologic (eg, subarachnoid haemorrhage, intracranial haemorrhage, cerebrovascular accident, intracranial trauma, and autonomic neuropathy), dermatologic, psychiatric, renal, or other major disease, that is unstable or could interfere with the subject's participation in or completion of the study, in the opinion of the investigator
- Clinically significant screening results that would exclude subject from the study (eg, medical history, physical examination, ECG, vital sign, pulse oximetry, and laboratory profiles) or any conditions that, would make the subject unsuitable for enrolment or could interfere with the subject's participation in or completion of the study, in the opinion of the investigator. The medical monitor must be contacted for review of any subjects with screening results or conditions that may make them unsuitable for enrolment or could interfere with participation in or completion of the study.
- Prolonged QTcF >450 msec at screening
- Abnormal liver function as defined by AST, ALT, gamma-glutamyl transferase (GGT), or alkaline phosphatase ≥3 times or total bilirubin ≥2 times upper limit of the normal range
Show all 20Show fewer
- Haemoglobin <10 g/dL
- Platelet count <150,000 cells/mm3
- Abnormal renal function at screening defined as creatinine clearance <60 mL/min using the Modified Diet in Renal Disease (MDRD)
- Hospitalisation, sinopulmonary infection, CF exacerbation, or other clinically significant infection or illness (in the opinion of the investigator) requiring an increase or addition of medication, such as antibiotics or corticosteroids, within 28 days of Day 1
- Lung infection with organisms associated with a more rapid decline in pulmonary status (eg, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus). Subjects who have a current or past history of a positive culture must be reviewed with the medical monitor to confirm clinical stability.
- Subject is currently taking or has taken a CFTR modulator within 28 days prior to Day 1
- Participation in another clinical trial or treatment with an investigational agent within 28 days or 5 half-lives, whichever is longer, prior to screening. The duration of the elapsed time may be longer if required by local regulations
- History of cancer (excluding cervical carcinoma in situ and non-melanoma skin cancer with curative therapy for at least 5 years prior to screening)
- History of organ or hematologic transplantation
- History or current evidence of alcohol or drug abuse or dependence within 12 months of screening, in the opinion of the investigator
- Initiation of any new chronic therapy (eg, ibuprofen, hypertonic saline, azithromycin, dornase alfa, aztreonam for inhalation solution, and tobramycin) or any change in chronic therapy (excluding pancreatic enzyme replacement therapy) within 28 days prior to Day 1
- Known or suspected hypersensitivity or idiosyncratic reaction to the study drugs or any components thereof
- Pregnant or nursing women
- Special or vulnerable status (e.g. institutionalized, or person related to or an employee of the sponsor, FAIR Therapeutics, or investigator)
Trial locations and contacts
16 sites in 9 countries.
16 sites in 9 countries
Italy4 sites
Instituto Giannina Gaslini
Genova
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan
Ospedale Pediatrico Bambino Gesù
Rome
Azienda Ospedaliera Universitaria Integrata
Verona
United Kingdom3 sites
University Hospitals Birmingham NHS Foundation Trust
Birmingham
Royal Brompton Hospital
London
University Hospital Southampton
Southampton
Germany2 sites
Charité Universitätsmedizin Berlin
Berlin
Medizinische Hochschule Hannover
Hanover
About this study
Official title: A Phase IIb, Multicentre, Randomised, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Subjects With Cystic Fibrosis Aged 18 Years or Older
CF is caused by mutations in the gene that encodes the 'Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)' channel. To re-establish the function of this complex chloride channel, typically two to three drug modes of action are needed. To date, clinical studies of CFTR modulators have focused on patients carrying the F508del CFTR mutation, which is present in approximately 80% of CF patients, or gating mutations which are present in 5% of CF patients (gating mutations result in a reduced opening of the CFTR-channel at the cell surface which limits the flow of chloride ions through the CFTR channel). Although CF is a monogenetic disease, the 15% remaining patients represent more than 2000 different rare and mostly uncharacterized CFTR mutations. Multiple pharma companies have one or more CF drugs in their developmental pipeline. However, it is not known which patients may respond to the drugs in the pipeline. It is hypothesized that by using individual patient's intestinal organoids to screen for drug response, a subset of patients with rare CFTR mutations can be identified who will clinically respond to drugs in the developmental pipeline. The Human Individualized Therapy of CF (HIT-CF) project has been designed to further evaluate this hypothesis. The project has received funding from the European Union's Horizon 2020 research and innovation program under grant agreement No 755021. The core of the project consists of a two-step approach to identify patients outside the existing drug label who may also benefit from CFTR-modulator treatment. In the first step of the project (HIT-CF Organoid Study, NTR7520), novel CFTR modulators and their combinations were tested on organoids from over 500 European and Israeli CF patients with rare CFTR mutations to identify patients who are predicted to clinically benefit from these treatments. The second step will evaluate the predicted clinical effect of the CFTR modulators in subjects identified by their organoid response to investigational products. CFTR modulators from the HIT-CF participating pharmaceutical company, FAIR Therapeutics, will be evaluated in the CHOICES clinical study described in this protocol. Data from this clinical study will be compared with the HIT-CF Organoid Study results to validate the organoid model.
- Conditions
- Cystic Fibrosis
- Being tested
- Diponecaftor, Placebo
- Keywords
- Cystic Fibrosis, CFTR modulator, Intestinal organoids
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DiponecaftorDrug
Diponecaftor is a triple combination of DIR/POS/NES. The combination therapy will be administered orally during 8 weeks. The daily dose contains:
- DIR 300 mg/day
- POS 600 mg/day
- NES 10 mg/day
PlaceboDrug
Placebo once daily for 8 weeks. Oral administration.
| Study group | Role | What they receive |
|---|---|---|
| Diponecaftor/Placebo | Other | Diponecaftor once daily for 8 weeks followed by placebo once daily for 8 weeks. In between both periods there will be a washout period to minimize a possible carryover effect. Receives: Drug: Diponecaftor; Drug: Placebo |
| Placebo/Diponecaftor | Other | Placebo once daily for 8 weeks followed by diponecaftor once daily for 8 weeks. In between both periods there will be a washout period to minimize a possible carryover effect. Receives: Drug: Diponecaftor; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean percent predicted forced expiratory volume in 1 second (ppFEV1)
The primary endpoint in both groups is the mean percent predicted forced expiratory volume in 1 second (ppFEV1) of measurements taken after 4, 6 and 8 weeks of treatment. Period baseline values will be corrected for in the analysis.
Measured: Measurements taken at 4, 6 and 8 weeks of treatment
Secondary outcomes (39)
Sweat chloride
The average of the sweat chloride measurements taken after 4, 6 and 8 weeks of treatment.
Measured: Measurements taken at 4, 6 and 8 weeks of treatment
Body weight
The average of the body weight measurements taken after 4, 6 and 8 weeks of treatment
Measured: Measurements taken at 4, 6 and 8 weeks of treatment
Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain
The average of the Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain measurements taken after 4, 6 and 8 weeks of treatment. Scores range from 0 to 100, with higher scores indicating better health.
Measured: Measurements taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Safety and tolerability assessments based on treatment-emergent Adverse Events (AEs)
Measured: Through study completion, an average of 30 weeks
Safety and tolerability assessments
Safety and tolerability assessments based on treatment-emergent Serious Adverse events (SAEs)
Measured: Through study completion, an average of 30 weeks
Safety and tolerability assessments
Platelet count
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Liver function (total bilirubin)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Liver function (alkaline phosphatase (ALP))
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Liver function (alanine transaminase (ALT))
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Liver function (gamma-glutamyl transferase (GGT))
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Anemia (Haemoglobin (Hb))
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Anemia (Haematocrit)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Anemia (Red blood cell count)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
White blood cell count (including differential)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Glucose measured in blood
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Sodium in blood
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Cholesterol in blood
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Total protein in blood
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Potassium in blood
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Chloride in blood
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Bicarbonate in blood
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Lactate dehydrogenase in blood
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Albumine in blood
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Calcium in blood
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
GFR MDRD (equation based on sex, ethnicity, age, blood urea nitrogen (BUN), creatinine)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Ketonuria (ketones measured by urine dipstick)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Proteinuria (protein measured by urine dipstick)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Hematuria (blood measured by urine dipstick)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Glucosuria (glucose measured by urine dipstick)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
pH of urine (pH measured by urine dipstick)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Specific gravitiy of urine (measured by urine dipstick)
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Coagulation (activated partial thromboplastin time (aPTT))
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Coagulation (prothrombin time international normalized ratio (PT-INR))
Measured: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
ECG QT Interval
Measured: Measurement taken at 4 weeks of treatment
Safety and tolerability assessments
Systolic and diastolic blood pressure
Measured: Measurement taken at start of treatment and at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Heart rate
Measured: Measurement taken at start of treatment and at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Respiratory rate
Measured: Measurement taken at start of treatment and at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Body temperature
Measured: Measurement taken at start of treatment and at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Pulse oximetry measurements
Measured: Measurement taken at start of treatment and at 4, 6 and 8 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2024
- Study start3 Jun 2024
- Primary completion26 Jun 2025
- Study completion26 Jun 2025
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.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kors van der Ent
- Sponsor type
- University, hospital or other
- Collaborators
- European Union
- Responsible party
- Sponsor-investigator, Kors van der Ent, UMC Utrecht
- Investigators
- C.K. van der Ent, Principal investigator, UMC Utrecht
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06468527
- Sponsor's protocol ID
- HIT-CF-001
- Acronym
- CHOICES
- Status last verified
- Nov 2025
- First submitted
- 3 Jun 2024
- Last updated
- 24 Nov 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIb, Multicentre, Randomised, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Subjects With Cystic Fibrosis Aged 18 Years or Older
- ClinicalTrials.gov ID
- NCT06468527
- Acronym / sponsor ID
- CHOICES / HIT-CF-001
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, CFTR modulator, Intestinal organoids
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Kors van der EntUniversity, hospital or other
- Collaborators
- European Union
- Study start
- 3 Jun 2024 (actual)
- Primary completion
- 26 Jun 2025 (actual)
- Study completion
- 26 Jun 2025 (actual)
- First posted
- 21 Jun 2024 (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.
CF is caused by mutations in the gene that encodes the 'Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)' channel. To re-establish the function of this complex chloride channel, typically two to three drug modes of action are needed. To date, clinical studies of CFTR modulators have focused on patients carrying the F508del CFTR mutation, which is present in approximately 80% of CF patients, or gating mutations which are present in 5% of CF patients (gating mutations result in a reduced opening of the CFTR-channel at the cell surface which limits the flow of chloride ions through the CFTR channel). Although CF is a monogenetic disease, the 15% remaining patients represent more than 2000 different rare and mostly uncharacterized CFTR mutations. Multiple pharma companies have one or more CF drugs in their developmental pipeline. However, it is not known which patients may respond to the drugs in the pipeline. It is hypothesized that by using individual patient's intestinal organoids to screen for drug response, a subset of patients with rare CFTR mutations can be identified who will clinically respond to drugs in the developmental pipeline. The Human Individualized Therapy of CF (HIT-CF) project has been designed to further evaluate this hypothesis. The project has received funding from the European Union's Horizon 2020 research and innovation program under grant agreement No 755021. The core of the project consists of a two-step approach to identify patients outside the existing drug label who may also benefit from CFTR-modulator treatment. In the first step of the project (HIT-CF Organoid Study, NTR7520), novel CFTR modulators and their combinations were tested on organoids from over 500 European and Israeli CF patients with rare CFTR mutations to identify patients who are predicted to clinically benefit from these treatments. The second step will evaluate the predicted clinical effect of the CFTR modulators in subjects identified by their organoid response to investigational products. CFTR modulators from the HIT-CF participating pharmaceutical company, FAIR Therapeutics, will be evaluated in the CHOICES clinical study described in this protocol. Data from this clinical study will be compared with the HIT-CF Organoid Study results to validate the organoid model.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Diponecaftor/Placebo | Other | Drug: Diponecaftor, Drug: PlaceboDiponecaftor once daily for 8 weeks followed by placebo once daily for 8 weeks. In between both periods there will be a washout period to minimize a possible carryover effect. |
| Placebo/Diponecaftor | Other | Drug: Diponecaftor, Drug: PlaceboPlacebo once daily for 8 weeks followed by diponecaftor once daily for 8 weeks. In between both periods there will be a washout period to minimize a possible carryover effect. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Male or female subjects who completed the HIT-CF Organoid Study and are 18 years of age or older on the date of informed consent
- Confirmed diagnosis of CF as follows:
- Clinically stable CF disease in the opinion of the investigator with no significant changes in health status within 28 days prior to Day 1
- Forced expiratory volume in one second (FEV1) ≥40% of predicted to ≤90% of predicted at the Screening Visit, based on the Global Lung Function Initiative (GLI) -2012 multi-ethnic all-age reference equations
- Body mass index (BMI) ≥16 kg/m2 and ≤30 kg/m2
Key exclusion criteria (20 listed)
- Subject has at least one of the following CFTR-mutations: F508del, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H, A455E, 3849+10kbC>T OR A combination of any two of the following mutations: any nonsense mutation, 1717-1G>A, 621+1G>T, 3120+1G>A, 1898+1G->A, CFTRdele2,3, and 2183AA->G
- History or current evidence of any clinically significant cardiac (eg, heart failure, left ventricular hypertrophy, myocardial infarction, and arrhythmia), endocrinologic, hematologic, hepatobiliary (eg, clinically significant cirrhosis with or without portal hypertension, hepatitis B or hepatitis C), immunologic, metabolic, urologic, pulmonary (besides CF), neurologic (eg, subarachnoid haemorrhage, intracranial haemorrhage, cerebrovascular accident, intracranial trauma, and autonomic neuropathy), dermatologic, psychiatric, renal, or other major disease, that is unstable or could interfere with the subject's participation in or completion of the study, in the opinion of the investigator
- Clinically significant screening results that would exclude subject from the study (eg, medical history, physical examination, ECG, vital sign, pulse oximetry, and laboratory profiles) or any conditions that, would make the subject unsuitable for enrolment or could interfere with the subject's participation in or completion of the study, in the opinion of the investigator. The medical monitor must be contacted for review of any subjects with screening results or conditions that may make them unsuitable for enrolment or could interfere with participation in or completion of the study.
- Prolonged QTcF >450 msec at screening
- Abnormal liver function as defined by AST, ALT, gamma-glutamyl transferase (GGT), or alkaline phosphatase ≥3 times or total bilirubin ≥2 times upper limit of the normal range
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 |
|---|---|---|
| DiponecaftorDiponecaftor is a triple combination of DIR/POS/NES. The combination therapy will be administered orally during 8 weeks. The daily dose contains: * DIR 300 mg/day * POS 600 mg/day * NES 10 mg/day | Drug | Diponecaftor/Placebo, Placebo/Diponecaftor |
| PlaceboPlacebo once daily for 8 weeks. Oral administration. | Drug | Diponecaftor/Placebo, Placebo/Diponecaftor |
Primary outcome
Mean percent predicted forced expiratory volume in 1 second (ppFEV1)
Time frame: Measurements taken at 4, 6 and 8 weeks of treatment
The primary endpoint in both groups is the mean percent predicted forced expiratory volume in 1 second (ppFEV1) of measurements taken after 4, 6 and 8 weeks of treatment. Period baseline values will be corrected for in the analysis.
39 secondary outcomes
Sweat chloride
Time frame: Measurements taken at 4, 6 and 8 weeks of treatment
Body weight
Time frame: Measurements taken at 4, 6 and 8 weeks of treatment
Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain
Time frame: Measurements taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Through study completion, an average of 30 weeks
Safety and tolerability assessments
Time frame: Through study completion, an average of 30 weeks
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at 4 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at start of treatment and at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at start of treatment and at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at start of treatment and at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at start of treatment and at 4, 6 and 8 weeks of treatment
Safety and tolerability assessments
Time frame: Measurement taken at start of treatment and at 4, 6 and 8 weeks of treatment
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 24 Nov 2025.
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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.
C.K. van der Ent principal investigator
UMC Utrecht
16 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Diponecaftor in people with Cystic Fibrosis, run by Kors van der Ent at 16 sites in 9 countries.
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 14 inclusion and 20 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 24 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in Italy, United Kingdom, France, Germany, Belgium and 4 more countries. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Mean percent predicted forced expiratory volume in 1 second (ppFEV1) (time frame: Measurements taken at 4, 6 and 8 weeks of treatment). 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 24 Nov 2025.