- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT03119649
A Study to Evaluate Multiple Doses of GLPG2222 in Adult Subjects With Cystic Fibrosis
19. oktober 2018 opdateret af: Galapagos NV
A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate Multiple Doses of GLPG2222 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation
This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate 4 different doses of GLPG2222 administered for 4 weeks to adult subjects with a confirmed diagnosis of CF and homozygous for the F508del Cystic Fibrosis Transmembrane conductance Regulator (CFTR) mutation.
Studieoversigt
Status
Afsluttet
Betingelser
Intervention / Behandling
Undersøgelsestype
Interventionel
Tilmelding (Faktiske)
59
Fase
- Fase 2
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiesteder
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Antwerp, Belgien
- UZ Antwerpen
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Brussels, Belgien
- UZ Brussel
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Ghent, Belgien
- UZ Gent
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Leuven, Belgien
- UZ Leuven
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Cambridge, Det Forenede Kongerige
- Papworth Hospital
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Leeds, Det Forenede Kongerige
- St James University Hospital
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Liverpool, Det Forenede Kongerige
- Liverpool Heart and Chest Hospital
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Southampton, Det Forenede Kongerige
- Southampton General Hospital
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Alabama
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Chatom, Alabama, Forenede Stater, 35233
- Child Health Research Unit at UAB
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Arkansas
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Little Rock, Arkansas, Forenede Stater, 72205
- University of Arkansas for Medical Sciences
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Florida
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Orlando, Florida, Forenede Stater, 32803
- Central Florida Pulmonary Group
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Illinois
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Glenview, Illinois, Forenede Stater, 60026
- Cystic Fibrosis Center of Chicago
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Maine
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Portland, Maine, Forenede Stater, 04102
- Maine Medical Center
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Maryland
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Baltimore, Maryland, Forenede Stater, 21205
- John Hopkins University School of Medicine
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South Carolina
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Charleston, South Carolina, Forenede Stater, 29425
- Medical University of South Carolina
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Amsterdam, Holland
- AMC Amsterdam
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Rotterdam, Holland
- Erasmus Medisch Centrum
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The Hague, Holland
- Haga ziekenhuis
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Utrecht, Holland
- UMC Utrecht
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Novi Beograd, Serbien
- Mother and child health institute of Serbia
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Barcelona, Spanien
- Hospital Universitari Vall d'Hebron
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Madrid, Spanien
- Hospital Universitario La Paz
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Valencia, Spanien
- Hospital Universitarii Plitecnic La Fe
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Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
18 år til 99 år (Voksen, Ældre voksen)
Tager imod sunde frivillige
Ingen
Køn, der er berettiget til at studere
Alle
Beskrivelse
Inclusion Criteria:
- Male or female subject ≥ 18 years of age, on the day of signing the Informed Consent Form (ICF).
- A confirmed clinical diagnosis of CF and homozygous for the F508del CFTR mutation
- Weight ≥ 40 kg.
- Stable concomitant treatment for at least 4 weeks (28 days) prior to baseline
- Forced expiratory volume in 1 second (FEV1) ≥ 40% of predicted normal for age, gender and height at screening
Exclusion Criteria:
- History of clinically meaningful unstable or uncontrolled chronic disease that makes the subject unsuitable for inclusion in the study in the opinion of the investigator.
- Unstable pulmonary status or respiratory tract infection requiring a change in therapy within 4 weeks of baseline.
- Need for supplemental oxygen during the day, and >2 liters per minute (LPM) while sleeping.
- Use of CFTR modulator therapy (e.g. lumacaftor or ivacaftor) within 4 weeks prior to the first study drug administration.
- History of hepatic cirrhosis with portal hypertension.
- Abnormal liver function test at screening; defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/ or alkaline phosphatase and/or gamma-glutamyl transferase (GGT) ≥ 3x the upper limit of normal (ULN); and/or total bilirubin (>1.5 times ULN)
- Estimated creatinine clearance < 60 mL/min using the Cockcroft-Gault formula at screening.
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Parallel tildeling
- Maskning: Firedobbelt
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
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Eksperimentel: Cohort A: GLPG2222 50 mg once daily (QD)
Participants received a single GLPG2222 50 mg tablet and two matching placebo tablets orally, QD for 29 days.
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Oral tablet(s) containing GLPG2222
Matching oral tablet(s) containing placebo
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Eksperimentel: Cohort A: GLPG2222 100 mg QD
Participants received a single GLPG2222 100 mg tablet and two matching placebo tablets orally, QD for 29 days.
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Matching oral tablet(s) containing placebo
Oral tablet(s) containing GLPG2222
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Eksperimentel: Cohort B: GLPG2222 200 mg QD
Participants received two GLPG2222 100 mg tablets and one matching placebo tablet orally, QD for 29 days.
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Matching oral tablet(s) containing placebo
Oral tablet(s) containing GLPG2222
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Eksperimentel: Cohort B: GLPG2222 400 mg QD
Participants received two GLPG2222 150 mg tablets and one GLPG2222 100 mg tablet orally, QD for 29 days.
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Matching oral tablet(s) containing placebo
Oral tablet(s) containing GLPG2222
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Placebo komparator: Cohort A Placebo
Participants received three matching placebo tablets, orally, QD for 29 days.
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Matching oral tablet(s) containing placebo
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Placebo komparator: Cohort B Placebo
Participants received three matching placebo tablets, orally, QD for 29 days.
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Matching oral tablet(s) containing placebo
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Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Number of Participants With Treatment-Emergent Adverse Events
Tidsramme: First administration (Day 1) through Follow-up (Day 43)
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Number of participants with any treatment-emergent adverse events (TEAEs) and serious or treatment-related TEAEs, as well as number of patients with TEAEs by worst intensity reported (mild, moderate, or severe).
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First administration (Day 1) through Follow-up (Day 43)
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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Mean Change From Baseline in Sweat Chloride Concentration at Day 29
Tidsramme: Prior to dosing on Days 1 and 29, or at early discontinuation
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Two sweat collections, one from each arm, were obtained.
Mean sweat chloride concentration was determined from both arms and measured as millimoles per liter (mmol/L).
Baseline was defined as the predose value on Day 1 (or the last non-missing predose measurement).
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Prior to dosing on Days 1 and 29, or at early discontinuation
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Mean Change From Baseline in Percent (%) Predicted FEV1 (%FEV1) at Day 29
Tidsramme: Predose and between 1 and 2 hours postdose on Days 1 and 29, or at early discontinuation
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Percent predicted FEV1 for age, gender, and height was determined from standardized spirometry assessments and estimated using the 2012 Global Lungs Initiative equation.
Baseline was defined as the last non-missing predose assessment on Day 1.
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Predose and between 1 and 2 hours postdose on Days 1 and 29, or at early discontinuation
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Mean Change From Baseline in the Respiratory Domain of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at Day 29
Tidsramme: Prior to dosing on Days 1 and 29, or at early discontinuation
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis.
The respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), derived from Questions 40, 41, 42, 45, and 46 if at least 50% of the questions had non-missing data.
The scale score ranged from 0-100; higher scores indicated fewer symptoms and better health-related quality of life with a negative change indicating a worsening of symptoms.
A change of 4 is considered clinically relevant.
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Prior to dosing on Days 1 and 29, or at early discontinuation
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Mean Maximum Observed Plasma Concentration (Cmax; Nanograms Per Milliliter [mg/mL]) of GLPG2222
Tidsramme: Day 15 (predose and 0.5, 1, 2, 3, 4, 6, and 8 hours postdose) and prior to dosing on Day 29
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Maximum concentration of GLPG2222 after multiple dosing (ng/ML), obtained directly from the observed concentration versus time data.
All pharmacokinetic (PK) parameters were determined from Day 15; Day 29 data were determined if the participant was not available for full PK profiling on Day 15.
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Day 15 (predose and 0.5, 1, 2, 3, 4, 6, and 8 hours postdose) and prior to dosing on Day 29
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Mean GLPG2222 Plasma Concentration Observed at Predose (Ctrough; ng/mL)
Tidsramme: Days 15 and 29 (predose)
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Plasma concentration of GLPG2222 observed at pre-dose (ng/mL), obtained directly from the observed concentration versus time data.
Ctrough was calculated using both Day 15 and Day 29 PK data.
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Days 15 and 29 (predose)
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Median Time to Occurrence of GLPG2222 Cmax (Tmax; Hours [h])
Tidsramme: Day 15 (predose and 0.5, 1, 2, 3, 4, 6, and 8 hours postdose) and prior to dosing on Day 29
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Time of occurrence of maximum concentration of GLPG2222 after multiple dosing (h), obtained directly from the observed concentration versus time data.
All PK parameters were determined from Day 15; Day 29 data were determined if the participant was not available for full PK profiling on Day 15.
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Day 15 (predose and 0.5, 1, 2, 3, 4, 6, and 8 hours postdose) and prior to dosing on Day 29
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Mean Area Under the Concentration-Time Curve From Time 0 up to 24 Hours Following Multiple Dosing (AUC[0-t]; ng.h/mL) of GLPG2222
Tidsramme: Day 15 (predose and 0.5, 1, 2, 3, 4, 6, and 8 hours postdose) and prior to dosing on Day 29
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Area under the concentration-time curve from time 0 up to 24 hours following multiple dosing (ng.h/mL), calculated by linear up/log down trapezoidal summation.
All PK parameters were determined from Day 15; Day 29 data were determined if the participant was not available for full PK profiling on Day 15.
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Day 15 (predose and 0.5, 1, 2, 3, 4, 6, and 8 hours postdose) and prior to dosing on Day 29
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Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Publikationer og nyttige links
Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Faktiske)
18. marts 2017
Primær færdiggørelse (Faktiske)
19. oktober 2017
Studieafslutning (Faktiske)
19. oktober 2017
Datoer for studieregistrering
Først indsendt
11. april 2017
Først indsendt, der opfyldte QC-kriterier
17. april 2017
Først opslået (Faktiske)
18. april 2017
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
16. november 2018
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
19. oktober 2018
Sidst verificeret
1. oktober 2018
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- GLPG2222-CL-202
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
Uafklaret
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Ja
Studerer et amerikansk FDA-reguleret enhedsprodukt
Ingen
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .
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Eisai Korea Inc.RekrutteringParkinsons sygdomKorea, Republikken
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Idorsia Pharmaceuticals Ltd.AfsluttetSøvnløshedForenede Stater, Tyskland, Australien, Canada, Danmark, Italien, Polen, Serbien, Spanien, Schweiz
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