Cystic FibrosisPhase 2SAPHIRA2NCT02690519
Study of GLPG1837 in Subjects With Cystic Fibrosis (S1251N Mutation)
A Phase 2 study testing GLPG1837 dose 1 and GLPG1837 dose 2 in people with Cystic Fibrosis, run by Lakefront Biotherapeutics NV at 5 sites in 2 countries.
Trial summary
At least 6 cystic fibrosis patients with the S1251N mutation will be treated for 4 weeks, consisting of two consecutive treatment periods of two weeks evaluating one dose of GLPG1837 each. After the treatment period, there is a 7-10 days follow-up period. 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?
- GLPG1837 dose 1 and GLPG1837 dose 2
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 5 sitesin 2 countries
- Study dates
- Jan 2016to Sep 2016
- 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 subjects ≥ 18 years of age, with a confirmed diagnosis of cystic fibrosis
- Subjects with gating S1251N CFTR mutation on at least one allele in the CFTR gene
- Subjects currently receiving treatment with ivacaftor on a stable regimen or not on a treatment regimen with ivacaftor, for at least 2 weeks prior to screening
- Weight ≥ 40.0 kg
- Subjects on stable concomitant treatment regimen for at least 4 weeks prior to baseline (excluding ivacaftor)
- Pre- or post-bronchodilator FEV1 ≥ 40% of predicted normal
- Subject will have to use highly effective contraceptive methods
Who cannot join
- On an ivacaftor-containing treatment regimen and unable or unwilling to discontinue ivacaftor for the washout and treatment periods of the study
- Concomitant use of antifungal drugs within 4 weeks of baseline
- A history of a clinically meaningful unstable or uncontrolled chronic disease
- Liver cirrhosis and portal hypertension
- Any significant change in the medical regimen for pulmonary health within 4 weeks of baseline
- Unstable pulmonary status or respiratory tract infection or changes in therapy for pulmonary disease within 4 weeks of baseline
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- Abnormal liver function
- Clinically significant abnormalities on ECG
- History of malignancy, solid organ/haematological transplantation
- Abnormal renal function
- Participation in another experimental therapy study within 30 days or 5 times half-life
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
Belgium3 sites
University Hospital Antwerp
Antwerp
University Hospital Ghent
Ghent
University Hospitals Leuven
Leuven
About this study
Official title: A Phase IIa, Open-label Study of Two Doses of GLPG1837 in Subjects With Cystic Fibrosis and the S1251N Mutation
At least 6 cystic fibrosis patients with the S1251N mutation will be treated for 4 weeks, consisting of two consecutive treatment periods of two weeks evaluating one dose of GLPG1837 each. After the treatment period, there is a 7-10 days follow-up period.
During the course of the study, subjects will be examined for any side effects that may occur (safety and tolerability).
Changes in sweat chloride will be assessed as biomarker from baseline onwards, and changes in pulmonary function (efficacy) will be explored throughout the study. The amount of GLPG1837 present in the blood (pharmacokinetics) will also be determined.
- Conditions
- Cystic Fibrosis
- Being tested
- GLPG1837 dose 1, GLPG1837 dose 2
- Keywords
- Cystic fibrosis S1251N mutation, GLPG1837
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GLPG1837 dose 1Drug
one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 weeks
GLPG1837 dose 2Drug
one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| GLPG1837 dose 1 and GLPG1837 dose 2 | Experimental | GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks Receives: Drug: GLPG1837 dose 1; Drug: GLPG1837 dose 2 |
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 GLPG1837 in terms of adverse events at every visit
Measured: Up to 9 weeks
Changes in laboratory parameters
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal laboratory parameters at every visit
Measured: Up to 7 weeks
Changes in vital signs
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal vital signs at every visit
Measured: Up to 9 weeks
Changes in physical examination
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal physical examination at every visit
Measured: Up to 9 weeks
Changes in electrocardiogram
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit
Measured: Up to 7 weeks
Secondary outcomes (3)
Changes in sweat chloride concentration
To evaluate the effect of GLPG1837 in terms of change in sweat chloride concentration, a biomarker to measure cystic fibrosis transmembrane conductance regulator (CFTR) ion channel function at every visit
Measured: Up to 9 weeks
Changes in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry
To explore the effect of GLPG1837 in terms of change in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry at every visit
Measured: Up to 9 weeks
Plasma levels of GLPG1837
To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 1 and Day 29 at every visit
Measured: Up to 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2016 (estimated)
- Study startJan 2016
- Primary completionAug 2016
- Study completionSep 2016
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 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
- NCT02690519
- Sponsor's protocol ID
- GLPG1837-CL-202
- Other IDs
- 2015-003292-30, 2015
- Acronym
- SAPHIRA2
- Status last verified
- Mar 2016
- First submitted
- 16 Feb 2016
- Last updated
- 11 Oct 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIa, Open-label Study of Two Doses of GLPG1837 in Subjects With Cystic Fibrosis and the S1251N Mutation
- ClinicalTrials.gov ID
- NCT02690519
- Acronym / sponsor ID
- SAPHIRA2 / GLPG1837-CL-202
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic fibrosis S1251N mutation, GLPG1837
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Lakefront Biotherapeutics NVIndustry (pharma, biotech, devices)
- Study start
- Jan 2016
- Primary completion
- Aug 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 24 Feb 2016 (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.
At least 6 cystic fibrosis patients with the S1251N mutation will be treated for 4 weeks, consisting of two consecutive treatment periods of two weeks evaluating one dose of GLPG1837 each. After the treatment period, there is a 7-10 days follow-up period.
During the course of the study, subjects will be examined for any side effects that may occur (safety and tolerability).
Changes in sweat chloride will be assessed as biomarker from baseline onwards, and changes in pulmonary function (efficacy) will be explored throughout the study. The amount of GLPG1837 present in the blood (pharmacokinetics) will also be determined.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GLPG1837 dose 1 and GLPG1837 dose 2 | Experimental | Drug: GLPG1837 dose 1, Drug: GLPG1837 dose 2GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male or female subjects ≥ 18 years of age, with a confirmed diagnosis of cystic fibrosis
- Subjects with gating S1251N CFTR mutation on at least one allele in the CFTR gene
- Subjects currently receiving treatment with ivacaftor on a stable regimen or not on a treatment regimen with ivacaftor, for at least 2 weeks prior to screening
- Weight ≥ 40.0 kg
- Subjects on stable concomitant treatment regimen for at least 4 weeks prior to baseline (excluding ivacaftor)
Key exclusion criteria (11 listed)
- On an ivacaftor-containing treatment regimen and unable or unwilling to discontinue ivacaftor for the washout and treatment periods of the study
- Concomitant use of antifungal drugs within 4 weeks of baseline
- A history of a clinically meaningful unstable or uncontrolled chronic disease
- Liver cirrhosis and portal hypertension
- Any significant change in the medical regimen for pulmonary health within 4 weeks of baseline
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 |
|---|---|---|
| GLPG1837 dose 1one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 weeks | Drug | GLPG1837 dose 1 and GLPG1837 dose 2 |
| GLPG1837 dose 2one GLPG1837 tablet in the morning and one GLPG1837 tablet in the evening, for 2 weeks | Drug | GLPG1837 dose 1 and GLPG1837 dose 2 |
Primary outcome
Changes in adverse events
Time frame: Up to 9 weeks
To evaluate the safety and tolerability of GLPG1837 in terms of adverse events at every visit
Changes in laboratory parameters
Time frame: Up to 7 weeks
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal laboratory parameters at every visit
Changes in vital signs
Time frame: Up to 9 weeks
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal vital signs at every visit
Changes in physical examination
Time frame: Up to 9 weeks
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal physical examination at every visit
Changes in electrocardiogram
Time frame: Up to 7 weeks
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit
3 secondary outcomes
Changes in sweat chloride concentration
Time frame: Up to 9 weeks
Changes in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry
Time frame: Up to 9 weeks
Plasma levels of GLPG1837
Time frame: Up to 4 weeks
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 Oct 2016.
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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
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GLPG1837 dose 1 and GLPG1837 dose 2 in people with Cystic Fibrosis, run by Lakefront Biotherapeutics NV at 5 sites in 2 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 7 inclusion and 11 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 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Belgium, Netherlands. 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?
Changes in adverse events (time frame: Up to 9 weeks). The study lists 5 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 11 Oct 2016.