Clinical trial NCT03451045: Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis
Official title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cystic Fibrosis
Keywords: Pulmonary exacerbation, lenabasum, JBT-101
Lead sponsor: Corbus Pharmaceuticals Inc.
Sponsor class: INDUSTRY
Collaborators: name: Cystic Fibrosis Foundation
First posted: 2018-03-01
Enrollment: 447
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Multicenter, double-blind, randomized, placebo-controlled, parallel group study.
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

1. ≥ 12 years of age at the time Informed Consent/ Assent is signed.
2. Weight ≥ 40 kg.
3. FEV1 ≥ 40% predicted and \< 100% predicted in the last 12 months.
4. 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.

Exclusion Criteria:

1. Severe or unstable CF at screening or Visit 1.
2. Any of the following values for laboratory tests at screening:

   1. A positive pregnancy test.
   2. Hemoglobin \< 10 g/dL in males and \< 9 g/dL in females.
   3. Neutrophils \< 1.0 x 10\^9 /L.
   4. Platelets \< 75 x 10\^9/L.
   5. Creatinine clearance \< 50 mL/min according to Modification of Diet in Renal Disease (MDRD) Study equation.
   6. Serum transaminases \> 2.5 x upper limit of normal.
3. 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.

Primary outcomes:
measure: Pulmonary Exacerbation (PEx) Rate Over 28 Weeks
timeFrame: 28 weeks (Baseline Day 0 to Week 28)
Secondary outcomes:
measure: Pulmonary Exacerbation (PEx) Rate
timeFrame: 28 weeks (Baseline Day 0 to Week 28)
measure: Time to First New Pulmonary Exacerbation (PEx)
timeFrame: 28 weeks (Baseline Day 0 to Week 28)
measure: Pulmonary Exacerbation (PEx)
timeFrame: 28 weeks (Baseline Day 0 to Week 28)
measure: CFQ-R Respiratory Symptom Domain
timeFrame: 28 weeks (Change from Baseline Day 0 to Week 28)
measure: FEV1 % Predicted
timeFrame: 28 weeks (Change from Baseline Day 0 to Week 28)
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: Long Beach
country: United States
facility: Miller Children's Hospital
state: California
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Orlando
country: United States
facility: Central Florida Pulmonary Group, PA
state: Florida
city: Tampa
country: United States
facility: USF Center for Advance Lung Disease
state: Florida
city: Atlanta
country: United States
facility: Emory Children's Center
state: Georgia
city: Chicago
country: United States
facility: Northwestern Memorial Hospital
state: Illinois
city: Glenview
country: United States
facility: The Cystic Fibrosis Institute
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Lebanon
country: United States
facility: Dartmouth-Hitchcock Medical Center (main location)
state: New Hampshire
city: Manchester
country: United States
facility: Dartmouth-Hitchcock Manchester (satellite site)
state: New Hampshire
city: Morristown
country: United States
facility: Atlantic Health Children's Hospital
state: New Jersey
city: New Brunswick
country: United States
facility: Rutgers Robert Wood Johnson Medical School
state: New Jersey
city: Hawthorne
country: United States
facility: New York Medical College
state: New York
city: New Hyde Park
country: United States
facility: North Shore LIJ Health System
state: New York
city: New York
country: United States
facility: Mount Sinai Beth Israel
state: New York
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Hershey
country: United States
facility: Milton S. Hershey Medical Center / Penn State College of Medicine
state: Pennsylvania
city: Philadelphia
country: United States
facility: Drexel University College of Medicine
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Sioux Falls
country: United States
facility: Sanford Children's Specialty Clinic
state: South Dakota
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Tyler
country: United States
facility: The University of Texas Health Science Center at Tyler
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Madison
country: United States
facility: University Hospital and UW Health Clinics
state: Wisconsin
city: Innsbruck
country: Austria
facility: Cystic Fibrosis Centre Innsbruck Medical University of Innsbruck, Dept. for Child and Adolescent Health, University Clinic for Paediatrics, Cardiology, Pneumology, Allergology, Cystic Fibrosis
city: Vienna
country: Austria
facility: Medical University of Vienna
city: Brussels
country: Belgium
facility: Universitair Ziekenhuis Brussel
city: Ruse
country: Bulgaria
facility: Medical Center Prolet EOOD
city: Sofia
country: Bulgaria
facility: UMHAT Alexandrovska
city: Varna
country: Bulgaria
facility: MHAT Sveta Marina EAD
city: Montréal
country: Canada
facility: Centre hospitalier de l'Université de Montréal (CHUM)
city: Toronto
country: Canada
facility: St. Michael's Hospital
city: Toronto
country: Canada
facility: The Hospital for Sick Children
city: Vancouver
country: Canada
facility: St. Paul's Hospital
city: Praha
country: Czechia
facility: Motol University Hospital
city: Bron
country: France
facility: Centre de Référence de la Mucoviscidose
city: Bron
country: France
facility: Service de Pneumologie, Allergologie, Mucoviscidose; Hôpital Femme-Mère-Enfant
city: Dijon
country: France
facility: Service de Pediatrie Medico-Chirurgicale et Genetique
city: Montpellier
country: France
facility: CHRU de Montpellier
city: Nancy
country: France
facility: CRCM Enfant de Nancy
city: Nice
country: France
facility: CHU de Nice
city: Paris
country: France
facility: Centre de Recherche en Explorations Fonctionnelles (CREF)
city: Paris
country: France
facility: CRCM Hôpital Necker
city: Roscoff
country: France
facility: Foundation ILDYS
city: Strasbourg
country: France
facility: Nouvel Hopital Civil Strasbourg
city: Berlin
country: Germany
facility: Charité Universitätsmedzin
city: Bochum
country: Germany
facility: Catholic Hospital Bochum - St. Josef-Hospital
city: Essen
country: Germany
facility: University Hospital Essen
city: Essen
country: Germany
facility: University Medicine Essen Ruhrlandklinik
city: Frankfurt
country: Germany
facility: Goethe University Children´s Hospital
city: Hanover
country: Germany
facility: Hannover Medical School
city: Jena
country: Germany
facility: University Hospital Jena
city: München
country: Germany
facility: Klinikum der Ludwig Maximilian Universität München
city: Thessaloníki
country: Greece
facility: General Hospital of Thessaloniki Ippokratio
city: Budapest
country: Hungary
facility: National Koranyi Institute of Pulmonology, Department of Cystic Fibrosis
city: Debrecen
country: Hungary
facility: University of Debrecen - Kenezy Gyula University Hospital
city: Kecskemét
country: Hungary
facility: Bács-Kiskun County Hospital, Teaching Hospital of the University of Szeged
city: Mosdós
country: Hungary
facility: Moritz Kaposi General Hospital, Mosdós, Department of Pediatric Pulmonary Rehabilitation
city: Torokbalint
country: Hungary
facility: Pediatric Pulmonology, Törökbálint, Hungary
city: Firenze
country: Italy
facility: Centro Regionale Toscano di Riferimento per la Fibrosi Cistica
city: Genova
country: Italy
facility: U.O.S.D. - Centro fibrosi cistica
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
city: Verona
country: Italy
facility: Azienda Ospedaliera Universitaria Verona
city: Nijmegen
country: Netherlands
facility: Radbound UMC
city: Dziekanow Lesny
country: Poland
facility: Instytut Matki I Dziecka, Centrum Leczenia Mukowiscydozy - Oddzial Chorob Pluc
city: Gdańsk
country: Poland
facility: Oddzial Pediatrii z Pododdzialem Leczenia Mukowiscydozy
city: Rabka-Zdrój
country: Poland
facility: Instytut Gruzlicy I Chorob Pluc Oddzial Terenowy im Jana I Ireny Rudnikow
city: Rzeszów
country: Poland
facility: Institute for Mother and Child, Department of CF for Children's and Youth
city: Lisboa
country: Portugal
facility: Hospital de Santa Maria
city: Braşov
country: Romania
facility: Medial Center for Ambulatory Diagnosis and Treatment
city: Moscow
country: Russian Federation
facility: Scientfic Research Institute of Pulmonology
city: Mytishchi
country: Russian Federation
facility: Diagnostic Children Hospital, Department of Pediatrics and Adolescent Medicine, Department of Pediatric Pulmonary, Allergology and Endocrinology
city: Saint Petersburg
country: Russian Federation
facility: Children's City Hospital of Saint Olga
city: St. Petersburg
country: Russian Federation
facility: First St. Petersburg State Pavlov Medical University
city: Belgrade
country: Serbia
facility: Clinical for Pulmonary Diseases, Clinical Center of Serbia
city: Novi Sad
country: Serbia
facility: Institute for Child and Youth Health Care of Vojvodina
city: Sremska Kamenica
country: Serbia
facility: Institute for Pulmonary Disease of Vojvodina
city: Banská Bystrica
country: Slovakia
facility: Children's faculty hospital with polyclinic Banska Bystrica
city: Košice
country: Slovakia
facility: Children's Faculty Hospital Kosice
city: Barcelona
country: Spain
facility: Unidad de Fibrosis Quistica Adultos
city: Barcelona
country: Spain
facility: Unidad de Fibrosis Quistica Pediatria
city: Valencia
country: Spain
facility: Unidad de Fibrosis Quistica y Transplante Pulmonar
city: Lund
country: Sweden
facility: Skane University Hospital
city: Stockholm
country: Sweden
facility: Karolinska University Hospital
city: Liverpool
country: United Kingdom
facility: Liverpool Heart and Chest Hospital
state: Merseyside
city: Belfast
country: United Kingdom
facility: Belfast City Hospital
state: Northern Ireland
city: Birmingham
country: United Kingdom
facility: Birmingham Women's and Children's NHS Foundation Trust
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust
city: Cambridge
country: United Kingdom
facility: Royal Papworth Hospital NHS Foundation Trust
city: Llandough
country: United Kingdom
facility: Cardiff and Vale University Health Board
city: London
country: United Kingdom
facility: Barts Health NHS Trust
city: London
country: United Kingdom
facility: Royal Brompton & Harefield NHS Foundation Trust
city: Nottingham
country: United Kingdom
facility: Wolfson Cystic Fibrosis Centre City Hospital Campus
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust
Study officials:
affiliation: Indiana University School of Medicine/Riley Physicians Pulmonary
name: James Chmiel, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: National Heart and Lung Institute, Imperial College
name: J. Stuart Elborn, MD
role: PRINCIPAL_INVESTIGATOR
References: