Study of GLPG1837 in Subjects With Cystic Fibrosis (G551D Mutation)

NCT ID: NCT02707562

Last Updated: 2016-12-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

26 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-02-29

Study Completion Date

2016-11-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

32 cystic fibrosis patients with the G551D mutation will be treated for 4 weeks, consisting of three consecutive treatment periods: two 1-week periods followed by one 2-week period, 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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Cystic fibrosis G551D mutation GLPG1837

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

GLPG1837 dose 1, GLPG1837 dose 2, GLPG1837 dose 3

GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks

Group Type EXPERIMENTAL

GLPG1837 dose 1

Intervention Type DRUG

two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for one week

GLPG1837 dose 2

Intervention Type DRUG

two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for one week

GLPG1837 dose 3

Intervention Type DRUG

two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for two weeks

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

GLPG1837 dose 1

two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for one week

Intervention Type DRUG

GLPG1837 dose 2

two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for one week

Intervention Type DRUG

GLPG1837 dose 3

two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for two weeks

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Male or female subjects ≥ 18 years of age, with a confirmed diagnosis of cystic fibrosis
* Subjects with gating G551D 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

Exclusion Criteria

* 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
* 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 halflife
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Galapagos NV

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Olivier Van de Steen, MD, MBA

Role: STUDY_DIRECTOR

Galapagos NV

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Royal Adelaide Hospital

Adelaide, , Australia

Site Status

The Prince Charles Hospital

Chermside, , Australia

Site Status

Monash Medical Centre

Clayton, , Australia

Site Status

Sir Charles Gairdner Hospital

Nedlands, , Australia

Site Status

Mater Adult Hospital

South Brisbane, , Australia

Site Status

Fakultni nemocnice v Motole

Prague, , Czechia

Site Status

Charité Universitätsmedizin Berlin

Berlin, , Germany

Site Status

Universitätsklinkikum Koeln

Cologne, , Germany

Site Status

Uniklinik Carl-Gustav-Carus

Dresden, , Germany

Site Status

Lungenheilkunde München-Pasing

München, , Germany

Site Status

Beamont Hospital

Dublin, , Ireland

Site Status

St. Vincent's University Hospital

Dublin, , Ireland

Site Status

Queen Elizabeth University Hospital

Glasgow, , United Kingdom

Site Status

Liverpool Heart and Chest Hospital

Liverpool, , United Kingdom

Site Status

Royal Brompton Hospital

London, , United Kingdom

Site Status

The Medicines Evaluation Unit Ltd

Manchester, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Australia Czechia Germany Ireland United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

Davies JC, Van de Steen O, van Koningsbruggen-Rietschel S, Drevinek P, Derichs N, McKone EF, Kanters D, Allamassey L, Namour F, de Kock H, Conrath K. GLPG1837, a CFTR potentiator, in p.Gly551Asp (G551D)-CF patients: An open-label, single-arm, phase 2a study (SAPHIRA1). J Cyst Fibros. 2019 Sep;18(5):693-699. doi: 10.1016/j.jcf.2019.05.006. Epub 2019 May 27.

Reference Type DERIVED
PMID: 31147302 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2015-003291-77

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GLPG1837-CL-201

Identifier Type: -

Identifier Source: org_study_id