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| ID | Type | Description | Link |
|---|---|---|---|
| 2015-003291-77 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| GLPG1837 dose 1, GLPG1837 dose 2, GLPG1837 dose 3 | Experimental | GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GLPG1837 dose 1 | Drug | two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for one week |
|
| Measure | Description | Time Frame |
|---|---|---|
| Changes in adverse events | To evaluate the safety and tolerability of GLPG1837 in terms of adverse events at every visit | 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 | Up to 7 weeks |
| Changes in vital signs - composite outcome measure | To evaluate the safety and tolerability of GLPG1837 in terms of abnormal vital signs as measured by temperature, blood pressure, heart rate and respiratory rate, at every visit | Up to 9 weeks |
| Changes in physical examination - composite outcome measure | To evaluate the safety and tolerability of GLPG1837 in terms of abnormalities during physical examination at every visit | Up to 9 weeks |
| Changes in electrocardiogram | To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit | Up to 7 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Up to 9 weeks |
| Changes in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Olivier Van de Steen, MD, MBA | Lakefront Biotherapeutics NV | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Royal Adelaide Hospital | Adelaide | Australia | ||||
| The Prince Charles Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31147302 | Derived | 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. |
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| GLPG1837 dose 2 | Drug | two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for one week |
|
| GLPG1837 dose 3 | Drug | two GLPG1837 tablets in the morning and two GLPG1837 tablets in the evening, for two weeks |
|
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 |
| Up to 9 weeks |
| Plasma levels of GLPG1837: Cmax, the maximum observed plasma concentration | To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 8 and Day 29 at every visit; On Day 29, an 8-hour profile will determine the Cmax, the maximum observed plasma concentration | Up to 3 weeks |
| Plasma levels of GLPG1837: tmax, the time of occurrence of Cmax | To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 8 and Day 29 at every visit; On Day 29, an 8-hour profile will determine the tmax, the time of occurrence of Cmax | Up to 3 weeks |
| Plasma levels of GLPG1837: AUC, the area under the plasma concentration-time curve | To characterize the pharmacokinetics (PK) of GLPG1837 by measuring the amount in plasma between Day 8 and Day 29 at every visit; On Day 29, an 8-hour profile will determine the AUC, the area under the plasma concentration-time curve | Up to 3 weeks |
| Chermside |
| Australia |
| Monash Medical Centre | Clayton | Australia |
| Sir Charles Gairdner Hospital | Nedlands | Australia |
| Mater Adult Hospital | South Brisbane | Australia |
| Fakultni nemocnice v Motole | Prague | Czechia |
| Charité Universitätsmedizin Berlin | Berlin | Germany |
| Universitätsklinkikum Koeln | Cologne | Germany |
| Uniklinik Carl-Gustav-Carus | Dresden | Germany |
| Lungenheilkunde München-Pasing | München | Germany |
| Beamont Hospital | Dublin | Ireland |
| St. Vincent's University Hospital | Dublin | Ireland |
| Queen Elizabeth University Hospital | Glasgow | United Kingdom |
| Liverpool Heart and Chest Hospital | Liverpool | United Kingdom |
| Royal Brompton Hospital | London | United Kingdom |
| The Medicines Evaluation Unit Ltd | Manchester | United Kingdom |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
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