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

• Register: ClinicalTrials.gov
• Last refreshed on: 9 January 2017
• Main ID: NCT02707562
• Date of registration: 22/02/2016
• Prospective Registration: No
• Primary sponsor: Galapagos NV
• Public title: Study of GLPG1837 in Subjects With Cystic Fibrosis (G551D Mutation) SAPHIRA1
• Scientific title: A Phase IIa, Open-label Study of Multiple Doses of GLPG1837 in Subjects With Cystic Fibrosis and the G551D Mutation
• Date of first enrolment: February 2016
• Target sample size: 26
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02707562
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Australia, Czech Republic, Germany, Ireland, United Kingdom

Contacts

• Name: Olivier Van de Steen, MD, MBA
• Affiliation: Galapagos NV

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: GLPG1837 dose 3

Drug: GLPG1837 dose 1

Drug: GLPG1837 dose 2

Primary Outcome(s)

Changes in adverse events [Time Frame: Up to 9 weeks]

Changes in electrocardiogram [Time Frame: Up to 7 weeks]

Changes in laboratory parameters [Time Frame: Up to 7 weeks]

Changes in vital signs - composite outcome measure [Time Frame: Up to 9 weeks]

Changes in physical examination - composite outcome measure [Time Frame: Up to 9 weeks]

Secondary Outcome(s)

Changes in sweat chloride concentration [Time Frame: Up to 9 weeks]

Plasma levels of GLPG1837: Cmax, the maximum observed plasma concentration [Time Frame: Up to 3 weeks]

Plasma levels of GLPG1837: AUC, the area under the plasma concentration-time curve [Time Frame: Up to 3 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: tmax, the time of occurrence of Cmax [Time Frame: Up to 3 weeks]

Secondary ID(s)

2015-003291-77

GLPG1837-CL-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors