Cystic FibrosisPhase 1FALCONNCT03540524
A Study Looking at the Safety, Tolerability and Efficacy of the Combination of the Study Drugs GLPG2451 and GLPG2222 With or Without GLPG2737 in Patients With Cystic Fibrosis.
A Phase 1 study testing GLPG2451 dose regimen A, GLPG2451 dose regimen B and GLPG2222 in people with Cystic Fibrosis, run by Lakefront Biotherapeutics NV at 20 sites in 8 countries.
Trial summary
This is a Phase Ib, multi-center, open-label, nonrandomized multiple cohorts study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of a combination treatment of GLPG2451 and GLPG2222, with and without GLPG2737, in adult subjects with Cystic Fibrosis. 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?
- GLPG2451 dose regimen A, GLPG2451 dose regimen B and GLPG2222
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 20 sitesin 8 countries
- Study dates
- 31 May 2018to 11 Mar 2019
- Phase
- Phase 1
- 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
- Female or male subject ≥18 years of age, on the day of signing the Informed Consent Form (ICF)
- Confirmed clinical diagnosis of cystic fibrosis (CF) (documented in the subject's medical record).
- Eligible cystic fibrosis transmembrane conductance regulator (CFTR) genotype at screening:
- Cohort A: Homozygous for the F508del CFTR mutation
- Cohort B: Heterozygous for the F508del CFTR mutation with a potentiator non-responsive mutation on the second allele
- Cohort C: Homozygous for the F508del CFTR mutation
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- A body weight of ≥40 kg at screening.
- Stable concomitant medication for pulmonary health for CF for at least 4 weeks prior to the first study drug administration and planned continuation of the same concomitant medication for the duration of the dosing period of the study. Subjects with diabetes mellitus and/or pancreatic insufficiency are eligible for the study provided they are on stable treatment (e.g. medication, diet, pancreatic enzyme replacement therapy) for at least 4 weeks prior to the first study drug administration in the opinion of the investigator.
- Forced expiratory volume in 1 second (FEV1): 40% ≤ FEV1 ≤ 90% of predicted normal for age, sex, and height at screening (pre- or post bronchodilator) at screening.
- Sweat chloride concentration ≥60 mmol/L at screening.
- Non-smoker and non-user of any nicotine and or cannabis containing products. A non-smoker is defined as an individual who has abstained from smoking for at least 1 year prior to the screening. A non-user is defined as an individual who has abstained from any nicotine containing products for at least 1 year prior to the screening.
Who cannot join
- History of or ongoing allergic bronchopulmonary aspergillosis.
- Medical history of cataract (or lens opacity) and/or glaucoma.
- Cataract (or lens opacity) and/or glaucoma determined by an ophthalmologist during the screening period.
- Unstable pulmonary status or respiratory tract infection (including rhinosinusitis) requiring a change in therapy within 4 weeks prior to the first study drug administration.
- History of clinically meaningful unstable or uncontrolled chronic disease that makes the subject unsuitable for inclusion in the study in the opinion of the investigator.
- Need for supplemental oxygen during the day, and >2 L/minute while sleeping.
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- History of hepatic cirrhosis with portal hypertension (e.g., signs/symptoms of splenomegaly, esophageal varices).
- History of malignancy within the past 5 years (except for basal cell carcinoma of the skin with no evidence of recurrence and/or carcinoma in situ of the cervix that has been treated with no evidence of recurrence).
- Use of any moderate and strong inhibitor(s) or inducer(s) of CYP3A4 within 4 weeks prior to the first study drug administration (e.g., clarithromycin, itraconazole, ketoconazole, telithromycin, rifampin, carbamazepine).
- Use of CFTR modulator therapy (e.g., lumacaftor and/or ivacaftor) within 4 weeks prior to the first study drug administration.
- Use of any oral corticosteroid within 3 months of screening; or history of oral corticosteroid use for ≥30 days (cumulative) within 2 years of screening.
- Abnormal liver function test at screening; defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/or alkaline phosphatase and/or gamma-glutamyl transferase (GGT) ≥3× the upper limit of normal (ULN); and/or total bilirubin ≥1.5× the ULN.
Trial locations and contacts
20 sites in 8 countries.
20 sites in 8 countries
United Kingdom7 sites
Study Site GBR003
Birmingham
Study Site GBR004
Glasgow
Study Site GBR005
Liverpool
Study Site GBR007
London, SW3 6NP
Study Site GBR006
Newcastle
Study Site GBR001
Papworth Everard
Study Site GBR002
Wythenshawe
Belgium4 sites
Study Site BEL004
Antwerp
Study Site BEL003
Brussels
Study Site BEL002
Ghent
Study Site BEL001
Leuven
About this study
Official title: Assessment of Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Oral Doses of the Combination of GLPG2451 and GLPG2222, With or Without GLPG2737, in Adult Subjects With Cystic Fibrosis
This is a Phase Ib, multi-center, open-label, nonrandomized multiple cohorts study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of a combination treatment of GLPG2451 and GLPG2222, with and without GLPG2737, in adult subjects with Cystic Fibrosis.
- Conditions
- Cystic Fibrosis
- Being tested
- GLPG2451 dose regimen A, GLPG2451 dose regimen B, GLPG2222, GLPG2737
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GLPG2451 dose regimen ADrug
GLPG2451 oral suspension, daily.
GLPG2451 dose regimen BDrug
GLPG2451 oral suspension, daily.
GLPG2222Drug
GLPG2222 tablet for oral use, daily.
GLPG2737Drug
GLPG2737 capsules for oral use, daily.
| Study group | Role | What they receive |
|---|---|---|
| Cohort A - F508del homozygous | Experimental | Dual combination (GLPG2451 and GLPG2222) will be administered for 14 days, followed by the triple combination (GLPG2451, GLPG2222 and GLPG2737) for 14 days, without washout in between the sequential treatment periods.( Study Part I) Receives: Drug: GLPG2451 dose regimen A; Drug: GLPG2222; Drug: GLPG2737 |
| Cohort B - F508del heterozygous/potentiator nonresponsive | Experimental | Dual combination (GLPG2451 and GLPG2222) will be administered for 14 days, followed by the triple combination (GLPG2451, GLPG2222 and GLPG2737) for 14 days, without washout in between the sequential treatment periods. (study Part II) Receives: Drug: GLPG2451 dose regimen B; Drug: GLPG2222; Drug: GLPG2737 |
| Cohort C - F508del homozygous | Experimental | Dual combination (GLPG2451 and GLPG2222) will be administered for 14 days, followed by the triple combination (GLPG2451, GLPG2222 and GLPG2737) for 14 days, without washout in between the sequential treatment periods. (Study Part II) Receives: Drug: GLPG2451 dose regimen B; Drug: GLPG2222; Drug: GLPG2737 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with adverse events.
To assess safety and tolerability of doses of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I and Part II).
Measured: Up to 24 weeks after the last dose
Maximum observed plasma concentration (Cmax).
To characterize the pharmacokinetics (PK) of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I).
Measured: Day 14
Maximum observed plasma concentration (Cmax).
To characterize the PK of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I).
Measured: Day 28
Area under the plasma concentration-time curve from time zero until 24 hours (AUC0-24h).
To characterize the PK of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I).
Measured: Day 14
Area under the plasma concentration-time curve from time zero until 24 hours (AUC0-24h).
To characterize the PK of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I).
Measured: Day 28
Trough plasma concentration observed at the end of the dosing interval (24 hours post-dose) (Ctrough).
To characterize the PK of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I and Part II).
Measured: Between Day 2 and Day 28
Change from baseline in sweat chloride concentration.
To assess changes in sweat chloride concentration after administration of the combination of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part II).
Measured: Between Day 1 pre-dose and Day 28
Change from baseline in percent predicted FEV1.
To assess changes in percent predicted FEV1 after administration of the combination of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part II).
Measured: Between Day 1 pre-dose and Day 28
Secondary outcomes (2)
Change from baseline in sweat chloride concentration.
To assess changes in sweat chloride concentration after administration of the combination of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I).
Measured: Between Day 1 pre-dose and Day 28
Change from baseline in percent predicted FEV1.
To assess changes in percent predicted FEV1 after administration of the combination of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I).
Measured: Between Day 1 pre-dose and Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 May 2018
- Study start31 May 2018
- Primary completion11 Mar 2019
- Study completion11 Mar 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.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lakefront Biotherapeutics NV
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Olivier Van de Steen, MD MBA, Study director, Lakefront Biotherapeutics NV
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03540524
- Sponsor's protocol ID
- GLPG2737-CL-105
- Other IDs
- 2017-001067-20, 2017
- Acronym
- FALCON
- Status last verified
- Apr 2018
- First submitted
- 27 Apr 2018
- Last updated
- 8 Apr 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Oral Doses of the Combination of GLPG2451 and GLPG2222, With or Without GLPG2737, in Adult Subjects With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03540524
- Acronym / sponsor ID
- FALCON / GLPG2737-CL-105
- Conditions
- Cystic Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Lakefront Biotherapeutics NVIndustry (pharma, biotech, devices)
- Study start
- 31 May 2018 (actual)
- Primary completion
- 11 Mar 2019 (actual)
- Study completion
- 11 Mar 2019 (actual)
- First posted
- 30 May 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.
This is a Phase Ib, multi-center, open-label, nonrandomized multiple cohorts study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of a combination treatment of GLPG2451 and GLPG2222, with and without GLPG2737, in adult subjects with Cystic Fibrosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort A - F508del homozygous | Experimental | Drug: GLPG2451 dose regimen A, Drug: GLPG2222, Drug: GLPG2737Dual combination (GLPG2451 and GLPG2222) will be administered for 14 days, followed by the triple combination (GLPG2451, GLPG2222 and GLPG2737) for 14 days, without washout in between the sequential treatment periods.( Study Part I) |
| Cohort B - F508del heterozygous/potentiator nonresponsive | Experimental | Drug: GLPG2451 dose regimen B, Drug: GLPG2222, Drug: GLPG2737Dual combination (GLPG2451 and GLPG2222) will be administered for 14 days, followed by the triple combination (GLPG2451, GLPG2222 and GLPG2737) for 14 days, without washout in between the sequential treatment periods. (study Part II) |
| Cohort C - F508del homozygous | Experimental | Drug: GLPG2451 dose regimen B, Drug: GLPG2222, Drug: GLPG2737Dual combination (GLPG2451 and GLPG2222) will be administered for 14 days, followed by the triple combination (GLPG2451, GLPG2222 and GLPG2737) for 14 days, without washout in between the sequential treatment periods. (Study Part II) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Female or male subject ≥18 years of age, on the day of signing the Informed Consent Form (ICF)
- Confirmed clinical diagnosis of cystic fibrosis (CF) (documented in the subject's medical record).
- Eligible cystic fibrosis transmembrane conductance regulator (CFTR) genotype at screening:
- A body weight of ≥40 kg at screening.
- Stable concomitant medication for pulmonary health for CF for at least 4 weeks prior to the first study drug administration and planned continuation of the same concomitant medication for the duration of the dosing period of the study. Subjects with diabetes mellitus and/or pancreatic insufficiency are eligible for the study provided they are on stable treatment (e.g. medication, diet, pancreatic enzyme replacement therapy) for at least 4 weeks prior to the first study drug administration in the opinion of the investigator.
Key exclusion criteria (12 listed)
- History of or ongoing allergic bronchopulmonary aspergillosis.
- Medical history of cataract (or lens opacity) and/or glaucoma.
- Cataract (or lens opacity) and/or glaucoma determined by an ophthalmologist during the screening period.
- Unstable pulmonary status or respiratory tract infection (including rhinosinusitis) requiring a change in therapy within 4 weeks prior to the first study drug administration.
- History of clinically meaningful unstable or uncontrolled chronic disease that makes the subject unsuitable for inclusion in the study in the opinion of the investigator.
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 |
|---|---|---|
| GLPG2451 dose regimen AGLPG2451 oral suspension, daily. | Drug | Cohort A - F508del homozygous |
| GLPG2451 dose regimen BGLPG2451 oral suspension, daily. | Drug | Cohort B - F508del heterozygous/potentiator nonresponsive, Cohort C - F508del homozygous |
| GLPG2222GLPG2222 tablet for oral use, daily. | Drug | Cohort A - F508del homozygous, Cohort B - F508del heterozygous/potentiator nonresponsive, Cohort C - F508del homozygous |
| GLPG2737GLPG2737 capsules for oral use, daily. | Drug | Cohort A - F508del homozygous, Cohort B - F508del heterozygous/potentiator nonresponsive, Cohort C - F508del homozygous |
Primary outcome
Number of subjects with adverse events.
Time frame: Up to 24 weeks after the last dose
To assess safety and tolerability of doses of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I and Part II).
Maximum observed plasma concentration (Cmax).
Time frame: Day 14
To characterize the pharmacokinetics (PK) of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I).
Maximum observed plasma concentration (Cmax).
Time frame: Day 28
To characterize the PK of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I).
Area under the plasma concentration-time curve from time zero until 24 hours (AUC0-24h).
Time frame: Day 14
To characterize the PK of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I).
Area under the plasma concentration-time curve from time zero until 24 hours (AUC0-24h).
Time frame: Day 28
To characterize the PK of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I).
Trough plasma concentration observed at the end of the dosing interval (24 hours post-dose) (Ctrough).
Time frame: Between Day 2 and Day 28
To characterize the PK of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part I and Part II).
Change from baseline in sweat chloride concentration.
Time frame: Between Day 1 pre-dose and Day 28
To assess changes in sweat chloride concentration after administration of the combination of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part II).
Change from baseline in percent predicted FEV1.
Time frame: Between Day 1 pre-dose and Day 28
To assess changes in percent predicted FEV1 after administration of the combination of GLPG2451 and GLPG2222 with or without GLPG2737 (Study Part II).
2 secondary outcomes
Change from baseline in sweat chloride concentration.
Time frame: Between Day 1 pre-dose and Day 28
Change from baseline in percent predicted FEV1.
Time frame: Between Day 1 pre-dose and Day 28
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 8 Apr 2019.
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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.
Olivier Van de Steen, MD MBA study director
Lakefront Biotherapeutics NV
20 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GLPG2451 dose regimen A, GLPG2451 dose regimen B and GLPG2222 in people with Cystic Fibrosis, run by Lakefront Biotherapeutics NV at 20 sites in 8 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 11 inclusion and 12 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 8 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United Kingdom, Belgium, Germany, Netherlands, Sweden and 3 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Number of subjects with adverse events. (time frame: Up to 24 weeks after the last dose). The study lists 8 primary outcomes in all. 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 8 Apr 2019.