Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis
- Conditions
- Cystic Fibrosis
- Interventions
- Registration Number
- NCT03451045
- Lead Sponsor
- Corbus Pharmaceuticals Inc.
- Brief Summary
This is a Phase 2 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of cystic fibrosis in patients 12 years of age or older. Approximately 415 subjects will be enrolled in this study at about 100 sites in North America, and Europe. The planned duration of treatment with study drug is 28 weeks.
Study drug will be lenabasum 20 mg BID, lenabasum 5 mg BID, and placebo in a 2:1:2 ratio.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 447
- ≥ 12 years of age at the time Informed Consent/ Assent is signed.
- Weight ≥ 40 kg.
- FEV1 ≥ 40% predicted and < 100% predicted in the last 12 months.
- Physician-initiated treatment with an IV antibiotic 2 or 3 times in the last 12 months for a new PEx or physician-initiated treatment with an IV antibiotic 1 time in the last 12 months plus physician-initiated treatment with oral antibiotic(s) 1 or more times in the past 12 months for a new PEX.
-
Severe or unstable CF at screening or Visit 1.
-
Any of the following values for laboratory tests at screening:
- A positive pregnancy test.
- Hemoglobin < 10 g/dL in males and < 9 g/dL in females.
- Neutrophils < 1.0 x 10^9 /L.
- Platelets < 75 x 10^9/L.
- Creatinine clearance < 50 mL/min according to Modification of Diet in Renal Disease (MDRD) Study equation.
- Serum transaminases > 2.5 x upper limit of normal.
-
Any medical condition or concurrent medical therapies at screening or Visit 1 that may put the subject at greater safety risk, influence response to study drug or interfere with study assessments.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Lenabasum 20 mg BID Lenabasum 20 mg - Lenabasum 5 mg BID Lenabasum 5 mg - Placebo BID Placebo -
- Primary Outcome Measures
Name Time Method Pulmonary Exacerbation (PEx) Rate Over 28 Weeks 28 weeks (Baseline Day 0 to Week 28) Rate of PEx using the primary PEx definition with lenabasum 20 mg BID compared to placebo, during the treatment period. An primary PEx is defined on the physician's decision to treat with oral, intraveneous or inhaled antibiotics in the presence 4/12 Fuch's criteria (Change in sputum, new/increased hemoptysis, increased cough, increased dyspnea, malaise, fatigue/lethargy, Temperature greater than 38C, weight loss, sinus pain, change in sinus discharge, change in exam of chest, decrease in FEV1 of more than 10%, radiographic change). This excludes prophylactic antibiotics. A new PEx is a one that occurs at 28 days after the previous PEx.
- Secondary Outcome Measures
Name Time Method Pulmonary Exacerbation (PEx) Rate 28 weeks (Baseline Day 0 to Week 28) Event rate of PEx using the secondary PEx definition with lenabasum 20 mg BID compared to placebo. The secondary definition of a PEx is based on the physician's diagnosis of pulmonary exacerbation and commencement of new oral, intravenous, or inhaled antibiotics. A new PEx is defined one that starts 28 or more days after the previous confirmed PEx. The PEx rate is calculated as the number of PEx/28 weeks
Time to First New Pulmonary Exacerbation (PEx) 28 weeks (Baseline Day 0 to Week 28) Time to first new PEx using the primary PEx definition with lenabasum 20 mg BID compared to placebo. An primary PEx is defined on the physician's decision to treat with oral, intraveneous or inhaled antibiotics in the presence 4/12 Fuch's criteria (Change in sputum, new/increased hemoptysis, increased cough, increased dyspnea, malaise, fatigue/lethargy, Temperature greater than 38C, weight loss, sinus pain, change in sinus discharge, change in exam of chest, decrease in FEV1 of more than 10%, radiographic change). This excludes prophylactic antibiotics. A new PEx is a one that occurs at 28 days after the previous PEx. The rate is calculate over a 28 week period from visit 1 to week 28 visit
Pulmonary Exacerbation (PEx) 28 weeks (Baseline Day 0 to Week 28) Time to first PEx using the secondary PEx definition with lenabasum 20 mg BID compared to placebo. The secondary definition of a PEx is based on the physician's diagnosis of pulmonary exacerbation and commencement of new oral, intravenous, or inhaled antibiotics. A new PEx is defined one that starts 28 or more days after the previous confirmed PEx.
CFQ-R Respiratory Symptom Domain 28 weeks (Change from Baseline Day 0 to Week 28) Cystic Fibrosis Questionnaire - Revised measures change from baseline in CFQ-R respiratory symptom domain with lenabasum compared to placebo. Subjects \>/= 14 years of age. 5 distinct 4-point Likert scales (e.g., always/often/ sometime/never) Scores for each HRQoL domain; after recoding, each item is summed to generate a domain score and standardized. Scores range from 0 to 100, with higher scores indicating better health.
FEV1 % Predicted 28 weeks (Change from Baseline Day 0 to Week 28) Change from baseline to week 28 in Forced Expiratory Volume in 1 second (FEV1) expressed as a percentage of a normal range. A lower percentage FEV1 is indicative of decrease in lung functionality. The changes observed from baseline to week 28 for lenabasum will be compared with those observed for placebo treated participants.
Trial Locations
- Locations (105)
Cystic Fibrosis Centre Innsbruck Medical University of Innsbruck, Dept. for Child and Adolescent Health, University Clinic for Paediatrics, Cardiology, Pneumology, Allergology, Cystic Fibrosis
🇦🇹Innsbruck, Austria
Medical University of Vienna
🇦🇹Vienna, Austria
Universitair Ziekenhuis Brussel
🇧🇪Brussels, Belgium
Goethe University Children´s Hospital
🇩🇪Frankfurt, Germany
The Hospital for Sick Children
🇨🇦Toronto, Canada
Service de Pediatrie Medico-Chirurgicale et Genetique
🇫🇷Dijon, France
CHRU de Montpellier
🇫🇷Montpellier, France
University Medicine Essen Ruhrlandklinik
🇩🇪Essen, Germany
Centre hospitalier de l'Université de Montréal (CHUM)
🇨🇦Montréal, Canada
Dartmouth-Hitchcock Medical Center (main location)
🇺🇸Lebanon, New Hampshire, United States
UT Southwestern Medical Center
🇺🇸Dallas, Texas, United States
The Cystic Fibrosis Institute
🇺🇸Glenview, Illinois, United States
University of Arkansas for Medical Sciences
🇺🇸Little Rock, Arkansas, United States
Rutgers Robert Wood Johnson Medical School
🇺🇸New Brunswick, New Jersey, United States
University Hospital Essen
🇩🇪Essen, Germany
Motol University Hospital
🇨🇿Praha, Czechia
UMHAT Alexandrovska
🇧🇬Sofia, Bulgaria
Centre de Référence de la Mucoviscidose
🇫🇷Bron, France
Foundation ILDYS
🇫🇷Roscoff, France
Atlantic Health Children's Hospital
🇺🇸Morristown, New Jersey, United States
MHAT Sveta Marina EAD
🇧🇬Varna, Bulgaria
St. Michael's Hospital
🇨🇦Toronto, Canada
Milton S. Hershey Medical Center / Penn State College of Medicine
🇺🇸Hershey, Pennsylvania, United States
Medical Center Prolet EOOD
🇧🇬Ruse, Bulgaria
Service de Pneumologie, Allergologie, Mucoviscidose; Hôpital Femme-Mère-Enfant
🇫🇷Bron, France
New York Medical College
🇺🇸Hawthorne, New York, United States
Charité Universitätsmedzin
🇩🇪Berlin, Germany
The University of Texas Health Science Center at Tyler
🇺🇸Tyler, Texas, United States
CRCM Enfant de Nancy
🇫🇷Nancy, France
Nouvel Hopital Civil Strasbourg
🇫🇷Strasbourg, France
Emory Children's Center
🇺🇸Atlanta, Georgia, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
University of Miami
🇺🇸Miami, Florida, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Centre de Recherche en Explorations Fonctionnelles (CREF)
🇫🇷Paris, France
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
Northwestern Memorial Hospital
🇺🇸Chicago, Illinois, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Dartmouth-Hitchcock Manchester (satellite site)
🇺🇸Manchester, New Hampshire, United States
North Shore LIJ Health System
🇺🇸New Hyde Park, New York, United States
Mount Sinai Beth Israel
🇺🇸New York, New York, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Drexel University College of Medicine
🇺🇸Philadelphia, Pennsylvania, United States
Sanford Children's Specialty Clinic
🇺🇸Sioux Falls, South Dakota, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
St. Paul's Hospital
🇨🇦Vancouver, Canada
CHU de Nice
🇫🇷Nice, France
CRCM Hôpital Necker
🇫🇷Paris, France
Catholic Hospital Bochum - St. Josef-Hospital
🇩🇪Bochum, Germany
Hannover Medical School
🇩🇪Hanover, Germany
University Hospital Jena
🇩🇪Jena, Germany
Klinikum der Ludwig Maximilian Universität München
🇩🇪München, Germany
General Hospital of Thessaloniki Ippokratio
🇬🇷Thessaloníki, Greece
National Koranyi Institute of Pulmonology, Department of Cystic Fibrosis
🇭🇺Budapest, Hungary
University of Debrecen - Kenezy Gyula University Hospital
🇭🇺Debrecen, Hungary
Bács-Kiskun County Hospital, Teaching Hospital of the University of Szeged
🇭🇺Kecskemét, Hungary
U.O.S.D. - Centro fibrosi cistica
🇮🇹Genova, Italy
Centro Regionale Toscano di Riferimento per la Fibrosi Cistica
🇮🇹Firenze, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Moritz Kaposi General Hospital, Mosdós, Department of Pediatric Pulmonary Rehabilitation
🇭🇺Mosdós, Hungary
Pediatric Pulmonology, Törökbálint, Hungary
🇭🇺Torokbalint, Hungary
Azienda Ospedaliera Universitaria Verona
🇮🇹Verona, Italy
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Radbound UMC
🇳🇱Nijmegen, Netherlands
Institute for Mother and Child, Department of CF for Children's and Youth
🇵🇱Rzeszów, Poland
Instytut Matki I Dziecka, Centrum Leczenia Mukowiscydozy - Oddzial Chorob Pluc
🇵🇱Dziekanow Lesny, Poland
Oddzial Pediatrii z Pododdzialem Leczenia Mukowiscydozy
🇵🇱Gdańsk, Poland
Instytut Gruzlicy I Chorob Pluc Oddzial Terenowy im Jana I Ireny Rudnikow
🇵🇱Rabka-Zdrój, Poland
Diagnostic Children Hospital, Department of Pediatrics and Adolescent Medicine, Department of Pediatric Pulmonary, Allergology and Endocrinology
🇷🇺Mytishchi, Russian Federation
Hospital de Santa Maria
🇵🇹Lisboa, Portugal
Scientfic Research Institute of Pulmonology
🇷🇺Moscow, Russian Federation
Medial Center for Ambulatory Diagnosis and Treatment
🇷🇴Braşov, Romania
Children's City Hospital of Saint Olga
🇷🇺Saint Petersburg, Russian Federation
First St. Petersburg State Pavlov Medical University
🇷🇺St. Petersburg, Russian Federation
Clinical for Pulmonary Diseases, Clinical Center of Serbia
🇷🇸Belgrade, Serbia
Institute for Child and Youth Health Care of Vojvodina
🇷🇸Novi Sad, Serbia
Institute for Pulmonary Disease of Vojvodina
🇷🇸Sremska Kamenica, Serbia
Children's faculty hospital with polyclinic Banska Bystrica
🇸🇰Banská Bystrica, Slovakia
Children's Faculty Hospital Kosice
🇸🇰Košice, Slovakia
Unidad de Fibrosis Quistica Adultos
🇪🇸Barcelona, Spain
Unidad de Fibrosis Quistica Pediatria
🇪🇸Barcelona, Spain
Skane University Hospital
🇸🇪Lund, Sweden
Unidad de Fibrosis Quistica y Transplante Pulmonar
🇪🇸Valencia, Spain
Karolinska University Hospital
🇸🇪Stockholm, Sweden
Liverpool Heart and Chest Hospital
🇬🇧Liverpool, Merseyside, United Kingdom
University Hospitals Birmingham NHS Foundation Trust
🇬🇧Birmingham, United Kingdom
Birmingham Women's and Children's NHS Foundation Trust
🇬🇧Birmingham, United Kingdom
Belfast City Hospital
🇬🇧Belfast, Northern Ireland, United Kingdom
Royal Papworth Hospital NHS Foundation Trust
🇬🇧Cambridge, United Kingdom
Barts Health NHS Trust
🇬🇧London, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust
🇬🇧London, United Kingdom
Cardiff and Vale University Health Board
🇬🇧Llandough, United Kingdom
Wolfson Cystic Fibrosis Centre City Hospital Campus
🇬🇧Nottingham, United Kingdom
University Hospital Southampton NHS Foundation Trust
🇬🇧Southampton, United Kingdom
Miller Children's Hospital
🇺🇸Long Beach, California, United States
Central Florida Pulmonary Group, PA
🇺🇸Orlando, Florida, United States
USF Center for Advance Lung Disease
🇺🇸Tampa, Florida, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
University Hospital and UW Health Clinics
🇺🇸Madison, Wisconsin, United States