GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis

NCT03474042 | Completed Phase 2 DMC

• 2 other identifiers: GLPG2737-CL-2022017-002181-42
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 9

Brief Summary

This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d. for 28 days to adult male and female subjects with a confirmed diagnosis of cystic fibrosis homozygous for the F508del CFTR mutation and on stable treatment with Orkambi.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Change from baseline in sweat chloride concentration compared to placebo

  To assess Change from baseline in sweat chloride concentration compared to placebo.

  Between day 1 pre-morning dose and Day 28.

Secondary Outcomes (7)

• Change versus placebo in the proportion of subjects with adverse events.

  Between Day 1 and 3 weeks after the last dose.

• Change from baseline in sweat chloride concentration.

  From baseline (pre-morning dose on Day 1) through 28 days.

• Change in percent predicted forced expiratory volume in 1 second (FEV1).

  From baseline (pre-morning dose on Day 1) through 28 days.

• Change in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R).

  From baseline (pre-morning dose on Day 1) through 28 days.

• Maximum observed plasma concentration of GLPG2737 (Cmax)

  Between day 1 pre-dose and day 14.

Study Arms (2)

GLPG2737

EXPERIMENTAL

GLPG2737 will be provided as capsules for oral use.

Drug: GLPG2737

Placebo

PLACEBO COMPARATOR

Placebo will be provided as capsules for oral use.

Drug: Placebo

Interventions

GLPG2737 DRUG

GLPG2737 oral capsules administered twice daily for 28 days on top of Orkambi.

GLPG2737

Placebo DRUG

Placebo oral capsules administered twice daily for 28 days on top of Orkambi.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female subject ≥18 years of age on the day of signing the ICF.
• A confirmed clinical diagnosis of CF and homozygous for the F508del CFTR mutation.
• Stable intake of physician prescribed Orkambi (lumacaftor 400 mg/ivacaftor 250 mg b.i.d.) for at least 12 weeks prior to the first study drug administration, and planned continuation of Orkambi for the duration of the study.
• FEV1 ≥40% of predicted normal for age, gender and height at screening (pre- or postbronchodilator).
• Sweat chloride concentration ≥60 mmol/L at screening.

You may not qualify if:

• History of serious allergic reaction to any drug as determined by the investigator (e.g., anaphylaxis requiring hospitalization) and/or known sensitivity to any component of the study drug.
• Unstable pulmonary status or respiratory tract infection (including rhinosinusitis) requiring a change in therapy within 4 weeks prior to the first study drug administration.
• 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 gammaglutamyl transferase (GGT) ≥3 x the upper limit of normal (ULN), and/or total bilirubin ≥1.5 x the ULN at screening.

Sponsors & Collaborators

• Galapagos NV: lead

Study Sites (9)

Study Site II

Berlin, Germany

Location

Study Site V

Cologne, Germany

Location

Study Site X

Dresden, Germany

Location

Study Site III

Essen, Germany

Location

Study Site IV

Frankfurt, Germany

Location

Study Site I

Heidelberg, Germany

Location

Study Site VI

München, Germany

Location

Study Site IX

Stuttgart, Germany

Location

Study Site VIII

Tübingen, Germany

Location

Related Publications (1)

• van Koningsbruggen-Rietschel S, Conrath K, Fischer R, Sutharsan S, Kempa A, Gleiber W, Schwarz C, Hector A, Van Osselaer N, Pano A, Corveleyn S, Bwirire D, Santermans E, Muller K, Bellaire S, Van de Steen O. GLPG2737 in lumacaftor/ivacaftor-treated CF subjects homozygous for the F508del mutation: A randomized phase 2A trial (PELICAN). J Cyst Fibros. 2020 Mar;19(2):292-298. doi: 10.1016/j.jcf.2019.09.006. Epub 2019 Oct 5.

  PMID: 31594690 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Olivier Van de Steen, MD MBA

  Galapagos NV

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2018
• First Posted: March 22, 2018
• Study Start: November 29, 2017
• Primary Completion: April 10, 2018
• Study Completion: April 10, 2018
• Last Updated: June 11, 2018

Record last verified: 2018-06

Locations

DE (9)