Cystic FibrosisPhase 2NCT03045523
A Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis
A Phase 2 study testing GLPG2222 150 mg q.d. and GLPG2222 300 mg q.d. in people with Cystic Fibrosis, run by Lakefront Biotherapeutics NV at 22 sites in 6 countries.
Trial summary
This clinical study is a phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel group study to evaluate two doses of orally administered GLPG2222 in adult subjects with a confirmed diagnosis of CF harbouring one F508del CFTR mutation and a second gating (class III) mutation and on stable… Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- GLPG2222 150 mg q.d. and GLPG2222 300 mg q.d.
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 37 participants
- Where
- 22 sitesin 6 countries
- Study dates
- Jan 2017to 11 Aug 2017
- 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 to 99 years
- 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 Informed Consent Form (ICF).
- A confirmed clinical diagnosis of CF.
- One F508del mutation on one allele in the CFTR gene, a gating (class III) mutation (one of the following: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R) on the 2nd allele in the CFTR gene (documented in the subject's medical record or CF registry).
- Weight ≥ 40 kg.
- Stable concomitant treatment for at least 4 weeks (28 days) prior to baseline (including physician prescribed ivacaftor (Kalydeco®) 150 mg b.i.d.).
- Forced expiratory volume in 1 second (FEV1) ≥ 40% of predicted normal for age, gender and height at screening (pre- or postbronchodilator).
Who cannot join
- 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 of baseline.
- Need for supplemental oxygen during the day, and >2 liters per minute (LPM) while sleeping.
- 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 total bilirubin (>1.5 times ULN (CTCAE Grade 2) and/or gamma-glutamyl transferase (GGT) ≥ 3x the upper limit of normal (ULN), and/or total bilirubin (>1.5 times ULN (CTCAE Grade 2).
- Estimated creatinine clearance < 60mL/min using the Cockroft-Gault formula at screening.
Trial locations and contacts
22 sites in 6 countries.
22 sites in 6 countries
United Kingdom8 sites
Birmingham Heartlands
Birmingham
Royal Devon and Exeter
Exeter
St James's University
Leeds
Liverpool Heart and Chest Hospital
Liverpool
Royal Brompton Hospital
London
University Hospital of South Manchester
Manchester
Royal Victoria Infirmary
Newcastle
Southampton General Hospital
Southampton
Australia4 sites
The Prince Charles Hospital
Chermside
The Alfred
Melbourne
Sir Charles Gairdner Hospital
Nedlands
Westmead Hospital
Westmead
Germany3 sites
Universitaetsklinikum Carl Gustav Carus TU Dresden
Dresden
Universitätsklinikum Erlangen
Erlangen
University Children´s Hospital
Tübingen
Ireland3 sites
Cork University Hospital
Cork
Beaumont Hospital
Dublin
St Vincents University Hospital
Dublin
About this study
Official title: A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis Harbouring One F508del CFTR Mutation and a Second Gating (Class III) Mutation
This clinical study is a phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel group study to evaluate two doses of orally administered GLPG2222 in adult subjects with a confirmed diagnosis of CF harbouring one F508del CFTR mutation and a second gating (class III) mutation and on stable treatment with ivacaftor.
Up to 35 evaluable subjects are planned to be included in the study. Eligible subjects must be on stable treatment with physician prescribed ivacaftor (Kalydeco®) for at least 28 days at the baseline visit. They will be randomized in a 2:2:1 ratio to receive one of two active doses of GLPG2222 (150 mg q.d. or 300 mg q.d.) or placebo q.d. administered for 29 days. Subjects will be in the study for a minimum of 6 weeks and a maximum of 10 weeks, from screening until the follow-up visit.
- Conditions
- Cystic Fibrosis
- Being tested
- GLPG2222 150 mg q.d., GLPG2222 300 mg q.d., 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.
GLPG2222 150 mg q.d.Drug
GLPG2222 150 mg administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days
GLPG2222 300 mg q.d.Drug
GLPG2222 300 mg administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days
PlaceboDrug
Placebo administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days
| Study group | Role | What they receive |
|---|---|---|
| GLPG2222 Dose 1 | Experimental | Not described Receives: Drug: GLPG2222 150 mg q.d. |
| GLPG2222 Dose 2 | Experimental | Not described Receives: Drug: GLPG2222 300 mg q.d. |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in adverse events
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of adverse events
Measured: at screening and at each study visit up to day 43 which is the final FU visit
Changes in abnormal laboratory
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of laboratory
Measured: at screening and at each study visit up to day 43 which is the final FU visit
Changes in abnormal vital signs, ECG or physical examination
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of vital signs, ECG or physical examination
Measured: at screening and at each study visit up to day 43 which is the final FU visit
Secondary outcomes (3)
Change from baseline of Sweat chloride concentration
Measured: at screening and at each study visit up to day 43 which is the final FU visit
Change from baseline of FEV1 (L) and percent predicted FEV1 for age, gender and height as assessed by spirometry
Measured: at screening and at each study visit up to day 43 which is the final FU visit
Change from baseline on the respiratory domain of Revised Cystic Fibrosis Questionnaire (CFQ-R)
Measured: at screening and at each study visit up to day 43 which is the final FU visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2017 (estimated)
- Study startJan 2017
- Primary completion11 Aug 2017
- Study completion11 Aug 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)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
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lakefront Biotherapeutics NV
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Olivier Van Steen, MD, MBA, Study director, Lakefront Biotherapeutics NV
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03045523
- Sponsor's protocol ID
- GLPG2222-CL-201
- Status last verified
- Nov 2017
- First submitted
- 26 Jan 2017
- Last updated
- 21 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
PubMed 37983082Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
PubMed 33331662Bell SC, Barry PJ, De Boeck K, Drevinek P, Elborn JS, Plant BJ, Minic P, Van Braeckel E, Verhulst S, Muller K, Kanters D, Bellaire S, de Kock H, Geller DE, Conrath K, Van de Steen O, van der Ent K. CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials. J Cyst Fibros. 2019 Sep;18(5):700-707. doi: 10.1016/j.jcf.2019.04.014. Epub 2019 May 3.
PubMed 31056441
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2017. 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 GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis Harbouring One F508del CFTR Mutation and a Second Gating (Class III) Mutation
- ClinicalTrials.gov ID
- NCT03045523
- Acronym / sponsor ID
- GLPG2222-CL-201
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Lakefront Biotherapeutics NVIndustry (pharma, biotech, devices)
- Study start
- Jan 2017
- Primary completion
- 11 Aug 2017 (actual)
- Study completion
- 11 Aug 2017 (actual)
- First posted
- 7 Feb 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This clinical study is a phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel group study to evaluate two doses of orally administered GLPG2222 in adult subjects with a confirmed diagnosis of CF harbouring one F508del CFTR mutation and a second gating (class III) mutation and on stable treatment with ivacaftor.
Up to 35 evaluable subjects are planned to be included in the study. Eligible subjects must be on stable treatment with physician prescribed ivacaftor (Kalydeco®) for at least 28 days at the baseline visit. They will be randomized in a 2:2:1 ratio to receive one of two active doses of GLPG2222 (150 mg q.d. or 300 mg q.d.) or placebo q.d. administered for 29 days. Subjects will be in the study for a minimum of 6 weeks and a maximum of 10 weeks, from screening until the follow-up visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| GLPG2222 Dose 1 | Experimental | Drug: GLPG2222 150 mg q.d. |
| GLPG2222 Dose 2 | Experimental | Drug: GLPG2222 300 mg q.d. |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female subject ≥ 18 years of age, on the day of signing the Informed Consent Form (ICF).
- A confirmed clinical diagnosis of CF.
- One F508del mutation on one allele in the CFTR gene, a gating (class III) mutation (one of the following: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R) on the 2nd allele in the CFTR gene (documented in the subject's medical record or CF registry).
- Weight ≥ 40 kg.
- Stable concomitant treatment for at least 4 weeks (28 days) prior to baseline (including physician prescribed ivacaftor (Kalydeco®) 150 mg b.i.d.).
Key exclusion criteria (6 listed)
- 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 of baseline.
- Need for supplemental oxygen during the day, and >2 liters per minute (LPM) while sleeping.
- 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 total bilirubin (>1.5 times ULN (CTCAE Grade 2) and/or gamma-glutamyl transferase (GGT) ≥ 3x the upper limit of normal (ULN), and/or total bilirubin (>1.5 times ULN (CTCAE Grade 2).
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 |
|---|---|---|
| GLPG2222 150 mg q.d.GLPG2222 150 mg administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days | Drug | GLPG2222 Dose 1 |
| GLPG2222 300 mg q.d.GLPG2222 300 mg administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days | Drug | GLPG2222 Dose 2 |
| PlaceboPlacebo administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days | Drug | Placebo |
Primary outcome
Changes in adverse events
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of adverse events
Changes in abnormal laboratory
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of laboratory
Changes in abnormal vital signs, ECG or physical examination
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of vital signs, ECG or physical examination
3 secondary outcomes
Change from baseline of Sweat chloride concentration
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
Change from baseline of FEV1 (L) and percent predicted FEV1 for age, gender and height as assessed by spirometry
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
Change from baseline on the respiratory domain of Revised Cystic Fibrosis Questionnaire (CFQ-R)
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
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 21 Nov 2017.
- Derived from this study: PubMed 37983082Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
- Derived from this study: PubMed 33331662Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
- Derived from this study: PubMed 31056441Bell SC, Barry PJ, De Boeck K, Drevinek P, Elborn JS, Plant BJ, Minic P, Van Braeckel E, Verhulst S, Muller K, Kanters D, Bellaire S, de Kock H, Geller DE, Conrath K, Van de Steen O, van der Ent K. CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials. J Cyst Fibros. 2019 Sep;18(5):700-707. doi: 10.1016/j.jcf.2019.04.014. Epub 2019 May 3.
- 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.
Olivier Van Steen, MD, MBA study director
Lakefront Biotherapeutics NV
22 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GLPG2222 150 mg q.d. and GLPG2222 300 mg q.d. in people with Cystic Fibrosis, run by Lakefront Biotherapeutics NV at 22 sites in 6 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 6 inclusion and 6 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 21 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United Kingdom, Australia, Belgium, Germany, Ireland and 1 more country. 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?
Changes in adverse events (time frame: at screening and at each study visit up to day 43 which is the final FU visit). The study lists 3 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 NCT03045523, which also lists 3 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 21 Nov 2017.