Cystic FibrosisPhase 2PELICANNCT03474042
GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis
A Phase 2 study testing GLPG2737 in people with Cystic Fibrosis, run by Lakefront Biotherapeutics NV at 9 sites in Germany.
Trial summary
This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d. for 28 days to adult male and female subjects with a confirmed diagnosis of cystic fibrosis homozygous for the F508del CFTR mutation and on stable treatment with Orkambi. 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?
- GLPG2737
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 22 participants
- Where
- 9 sitesGermany
- Study dates
- 29 Nov 2017to 10 Apr 2018
- 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
- Male or female subject ≥18 years of age on the day of signing the ICF.
- A confirmed clinical diagnosis of CF and homozygous for the F508del CFTR mutation.
- Stable intake of physician prescribed Orkambi (lumacaftor 400 mg/ivacaftor 250 mg b.i.d.) for at least 12 weeks prior to the first study drug administration, and planned continuation of Orkambi for the duration of the study.
- FEV1 ≥40% of predicted normal for age, gender and height at screening (pre- or postbronchodilator).
- Sweat chloride concentration ≥60 mmol/L at screening.
Who cannot join
- History of serious allergic reaction to any drug as determined by the investigator (e.g., anaphylaxis requiring hospitalization) and/or known sensitivity to any component of the study drug.
- 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.
- 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 hepatic cirrhosis with portal hypertension (e.g.,signs/symptoms of splenomegaly, esophageal varices, etc.).
- Abnormal liver function test at screening, defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/or alkaline phosphatase and/or gammaglutamyl transferase (GGT) ≥3 x the upper limit of normal (ULN), and/or total bilirubin ≥1.5 x the ULN at screening.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
Germany9 sites
Study Site II
Berlin
Study Site V
Cologne
Study Site X
Dresden
Study Site III
Essen
Study Site IV
Frankfurt
Study Site I
Heidelberg
Study Site VI
München
Study Site IX
Stuttgart
Study Site VIII
Tübingen
About this study
Official title: A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2737 in Orkambi-treated Subjects With Cystic Fibrosis Homozygous for the F508del Mutation
This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d. for 28 days to adult male and female subjects with a confirmed diagnosis of cystic fibrosis homozygous for the F508del CFTR mutation and on stable treatment with Orkambi.
- Conditions
- Cystic Fibrosis
- Being tested
- GLPG2737, Placebo
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GLPG2737Drug
GLPG2737 oral capsules administered twice daily for 28 days on top of Orkambi.
PlaceboDrug
Placebo oral capsules administered twice daily for 28 days on top of Orkambi.
| Study group | Role | What they receive |
|---|---|---|
| GLPG2737 | Experimental | GLPG2737 will be provided as capsules for oral use. Receives: Drug: GLPG2737 |
| Placebo | Placebo comparator | Placebo will be provided as capsules for oral use. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in sweat chloride concentration compared to placebo
To assess Change from baseline in sweat chloride concentration compared to placebo.
Measured: Between day 1 pre-morning dose and Day 28.
Secondary outcomes (7)
Change versus placebo in the proportion of subjects with adverse events.
To assess safety and tolerability by the number and percentage of subjects with adverse events.
Measured: Between Day 1 and 3 weeks after the last dose.
Change from baseline in sweat chloride concentration.
To assess the change from baseline in sweat chloride concentration.
Measured: From baseline (pre-morning dose on Day 1) through 28 days.
Change in percent predicted forced expiratory volume in 1 second (FEV1).
To assess the change from baseline in percent predicted forced expiratory volume in 1 second (FEV1).
Measured: From baseline (pre-morning dose on Day 1) through 28 days.
Change in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R).
To assess the change from baseline in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R).
Measured: From baseline (pre-morning dose on Day 1) through 28 days.
Maximum observed plasma concentration of GLPG2737 (Cmax)
To characterize the PK of GLPG2737 and its active metabolite, ivacaftor, and lumacaftor.
Measured: Between day 1 pre-dose and day 14.
Area under the plasma concentration-time curve from time zero until 8 hours (AUC0-8h) post-dose calculated by the linear up - logarithmic down trapezoidal rule (on Day 14)
To characterize the PK of GLPG2737 and its active metabolite G1125498 (M4), ivacaftor, and lumacaftor.
Measured: Between day 1 pre-dose and day 14.
Trough plasma concentration observed at the end of the dosing interval (Ctrough).
To characterize the PK of GLPG2737 and its active metabolite G1125498 (M4), ivacaftor, and lumacaftor.
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 posted22 Mar 2018
- Study start29 Nov 2017
- Primary completion10 Apr 2018
- Study completion10 Apr 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 22 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
- NCT03474042
- Sponsor's protocol ID
- GLPG2737-CL-202
- Other IDs
- 2017-002181-42, 2017
- Acronym
- PELICAN
- Status last verified
- Jun 2018
- First submitted
- 15 Mar 2018
- Last updated
- 11 Jun 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
van Koningsbruggen-Rietschel S, Conrath K, Fischer R, Sutharsan S, Kempa A, Gleiber W, Schwarz C, Hector A, Van Osselaer N, Pano A, Corveleyn S, Bwirire D, Santermans E, Muller K, Bellaire S, Van de Steen O. GLPG2737 in lumacaftor/ivacaftor-treated CF subjects homozygous for the F508del mutation: A randomized phase 2A trial (PELICAN). J Cyst Fibros. 2020 Mar;19(2):292-298. doi: 10.1016/j.jcf.2019.09.006. Epub 2019 Oct 5.
PubMed 31594690
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2737 in Orkambi-treated Subjects With Cystic Fibrosis Homozygous for the F508del Mutation
- ClinicalTrials.gov ID
- NCT03474042
- Acronym / sponsor ID
- PELICAN / GLPG2737-CL-202
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Lakefront Biotherapeutics NVIndustry (pharma, biotech, devices)
- Study start
- 29 Nov 2017 (actual)
- Primary completion
- 10 Apr 2018 (actual)
- Study completion
- 10 Apr 2018 (actual)
- First posted
- 22 Mar 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 IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d. for 28 days to adult male and female subjects with a confirmed diagnosis of cystic fibrosis homozygous for the F508del CFTR mutation and on stable treatment with Orkambi.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GLPG2737 | Experimental | Drug: GLPG2737GLPG2737 will be provided as capsules for oral use. |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo will be provided as capsules for oral use. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female subject ≥18 years of age on the day of signing the ICF.
- A confirmed clinical diagnosis of CF and homozygous for the F508del CFTR mutation.
- Stable intake of physician prescribed Orkambi (lumacaftor 400 mg/ivacaftor 250 mg b.i.d.) for at least 12 weeks prior to the first study drug administration, and planned continuation of Orkambi for the duration of the study.
- FEV1 ≥40% of predicted normal for age, gender and height at screening (pre- or postbronchodilator).
- Sweat chloride concentration ≥60 mmol/L at screening.
Key exclusion criteria (5 listed)
- History of serious allergic reaction to any drug as determined by the investigator (e.g., anaphylaxis requiring hospitalization) and/or known sensitivity to any component of the study drug.
- 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.
- 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 hepatic cirrhosis with portal hypertension (e.g.,signs/symptoms of splenomegaly, esophageal varices, etc.).
- Abnormal liver function test at screening, defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/or alkaline phosphatase and/or gammaglutamyl transferase (GGT) ≥3 x the upper limit of normal (ULN), and/or total bilirubin ≥1.5 x the ULN at screening.
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 |
|---|---|---|
| GLPG2737GLPG2737 oral capsules administered twice daily for 28 days on top of Orkambi. | Drug | GLPG2737 |
| PlaceboPlacebo oral capsules administered twice daily for 28 days on top of Orkambi. | Drug | Placebo |
Primary outcome
Change from baseline in sweat chloride concentration compared to placebo
Time frame: Between day 1 pre-morning dose and Day 28.
To assess Change from baseline in sweat chloride concentration compared to placebo.
7 secondary outcomes
Change versus placebo in the proportion of subjects with adverse events.
Time frame: Between Day 1 and 3 weeks after the last dose.
Change from baseline in sweat chloride concentration.
Time frame: From baseline (pre-morning dose on Day 1) through 28 days.
Change in percent predicted forced expiratory volume in 1 second (FEV1).
Time frame: From baseline (pre-morning dose on Day 1) through 28 days.
Change in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R).
Time frame: From baseline (pre-morning dose on Day 1) through 28 days.
Maximum observed plasma concentration of GLPG2737 (Cmax)
Time frame: Between day 1 pre-dose and day 14.
Area under the plasma concentration-time curve from time zero until 8 hours (AUC0-8h) post-dose calculated by the linear up - logarithmic down trapezoidal rule (on Day 14)
Time frame: Between day 1 pre-dose and day 14.
Trough plasma concentration observed at the end of the dosing interval (Ctrough).
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 11 Jun 2018.
- Derived from this study: PubMed 31594690van Koningsbruggen-Rietschel S, Conrath K, Fischer R, Sutharsan S, Kempa A, Gleiber W, Schwarz C, Hector A, Van Osselaer N, Pano A, Corveleyn S, Bwirire D, Santermans E, Muller K, Bellaire S, Van de Steen O. GLPG2737 in lumacaftor/ivacaftor-treated CF subjects homozygous for the F508del mutation: A randomized phase 2A trial (PELICAN). J Cyst Fibros. 2020 Mar;19(2):292-298. doi: 10.1016/j.jcf.2019.09.006. Epub 2019 Oct 5.
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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
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GLPG2737 in people with Cystic Fibrosis, run by Lakefront Biotherapeutics NV at 9 sites 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 5 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 11 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites 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. Some participants in this study may receive a placebo. 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?
Change from baseline in sweat chloride concentration compared to placebo (time frame: Between day 1 pre-morning dose and Day 28.). 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 NCT03474042, which also lists 1 linked publication.
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 11 Jun 2018; AzTrials last refreshed its copy on 8 Nov 2026.