Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis

NCT ID: NCT03451045

Last Updated: 2023-01-18

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

447 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-22

Study Completion Date

2020-06-17

Brief Summary

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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

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Multicenter, double-blind, randomized, placebo-controlled, parallel group study.
Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Lenabasum 20 mg BID

Group Type EXPERIMENTAL

Lenabasum 20 mg

Intervention Type DRUG

Subjects will receive lenabasum 20 mg twice daily.

Lenabasum 5 mg BID

Group Type EXPERIMENTAL

Lenabasum 5 mg

Intervention Type DRUG

Subjects will receive lenabasum 5 mg twice daily.

Placebo BID

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Subjects will receive placebo twice daily.

Interventions

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Lenabasum 20 mg

Subjects will receive lenabasum 20 mg twice daily.

Intervention Type DRUG

Lenabasum 5 mg

Subjects will receive lenabasum 5 mg twice daily.

Intervention Type DRUG

Placebo

Subjects will receive placebo twice daily.

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Cystic Fibrosis Foundation

OTHER

Sponsor Role collaborator

Corbus Pharmaceuticals Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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James Chmiel, MD

Role: PRINCIPAL_INVESTIGATOR

Indiana University School of Medicine/Riley Physicians Pulmonary

J. Stuart Elborn, MD

Role: PRINCIPAL_INVESTIGATOR

National Heart and Lung Institute, Imperial College

Locations

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Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Site Status

Miller Children's Hospital

Long Beach, California, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

University of Miami

Miami, Florida, United States

Site Status

Central Florida Pulmonary Group, PA

Orlando, Florida, United States

Site Status

USF Center for Advance Lung Disease

Tampa, Florida, United States

Site Status

Emory Children's Center

Atlanta, Georgia, United States

Site Status

Northwestern Memorial Hospital

Chicago, Illinois, United States

Site Status

The Cystic Fibrosis Institute

Glenview, Illinois, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Dartmouth-Hitchcock Medical Center (main location)

Lebanon, New Hampshire, United States

Site Status

Dartmouth-Hitchcock Manchester (satellite site)

Manchester, New Hampshire, United States

Site Status

Atlantic Health Children's Hospital

Morristown, New Jersey, United States

Site Status

Rutgers Robert Wood Johnson Medical School

New Brunswick, New Jersey, United States

Site Status

New York Medical College

Hawthorne, New York, United States

Site Status

North Shore LIJ Health System

New Hyde Park, New York, United States

Site Status

Mount Sinai Beth Israel

New York, New York, United States

Site Status

Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

Milton S. Hershey Medical Center / Penn State College of Medicine

Hershey, Pennsylvania, United States

Site Status

Drexel University College of Medicine

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Sanford Children's Specialty Clinic

Sioux Falls, South Dakota, United States

Site Status

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status

Cook Children's Medical Center

Fort Worth, Texas, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

The University of Texas Health Science Center at Tyler

Tyler, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

University Hospital and UW Health Clinics

Madison, Wisconsin, United States

Site Status

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

Site Status

Medical University of Vienna

Vienna, , Austria

Site Status

Universitair Ziekenhuis Brussel

Brussels, , Belgium

Site Status

Medical Center Prolet EOOD

Rousse, , Bulgaria

Site Status

UMHAT Alexandrovska

Sofia, , Bulgaria

Site Status

MHAT Sveta Marina EAD

Varna, , Bulgaria

Site Status

Centre hospitalier de l'Université de Montréal (CHUM)

Montreal, , Canada

Site Status

St. Michael's Hospital

Toronto, , Canada

Site Status

The Hospital for Sick Children

Toronto, , Canada

Site Status

St. Paul's Hospital

Vancouver, , Canada

Site Status

Motol University Hospital

Prague, , Czechia

Site Status

Centre de Référence de la Mucoviscidose

Bron, , France

Site Status

Service de Pneumologie, Allergologie, Mucoviscidose; Hôpital Femme-Mère-Enfant

Bron, , France

Site Status

Service de Pediatrie Medico-Chirurgicale et Genetique

Dijon, , France

Site Status

CHRU de Montpellier

Montpellier, , France

Site Status

CRCM Enfant de Nancy

Nancy, , France

Site Status

CHU de Nice

Nice, , France

Site Status

Centre de Recherche en Explorations Fonctionnelles (CREF)

Paris, , France

Site Status

CRCM Hôpital Necker

Paris, , France

Site Status

Foundation ILDYS

Roscoff, , France

Site Status

Nouvel Hopital Civil Strasbourg

Strasbourg, , France

Site Status

Charité Universitätsmedzin

Berlin, , Germany

Site Status

Catholic Hospital Bochum - St. Josef-Hospital

Bochum, , Germany

Site Status

University Hospital Essen

Essen, , Germany

Site Status

University Medicine Essen Ruhrlandklinik

Essen, , Germany

Site Status

Goethe University Children´s Hospital

Frankfurt, , Germany

Site Status

Hannover Medical School

Hanover, , Germany

Site Status

University Hospital Jena

Jena, , Germany

Site Status

Klinikum der Ludwig Maximilian Universität München

München, , Germany

Site Status

General Hospital of Thessaloniki Ippokratio

Thessaloniki, , Greece

Site Status

National Koranyi Institute of Pulmonology, Department of Cystic Fibrosis

Budapest, , Hungary

Site Status

University of Debrecen - Kenezy Gyula University Hospital

Debrecen, , Hungary

Site Status

Bács-Kiskun County Hospital, Teaching Hospital of the University of Szeged

Kecskemét, , Hungary

Site Status

Moritz Kaposi General Hospital, Mosdós, Department of Pediatric Pulmonary Rehabilitation

Mosdós, , Hungary

Site Status

Pediatric Pulmonology, Törökbálint, Hungary

Törökbálint, , Hungary

Site Status

Centro Regionale Toscano di Riferimento per la Fibrosi Cistica

Florence, , Italy

Site Status

U.O.S.D. - Centro fibrosi cistica

Genova, , Italy

Site Status

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

Azienda Ospedaliera Universitaria Verona

Verona, , Italy

Site Status

Radbound UMC

Nijmegen, , Netherlands

Site Status

Instytut Matki I Dziecka, Centrum Leczenia Mukowiscydozy - Oddzial Chorob Pluc

Dziekanów Leśny, , Poland

Site Status

Oddzial Pediatrii z Pododdzialem Leczenia Mukowiscydozy

Gdansk, , Poland

Site Status

Instytut Gruzlicy I Chorob Pluc Oddzial Terenowy im Jana I Ireny Rudnikow

Rabka-Zdrój, , Poland

Site Status

Institute for Mother and Child, Department of CF for Children's and Youth

Rzeszów, , Poland

Site Status

Hospital de Santa Maria

Lisbon, , Portugal

Site Status

Medial Center for Ambulatory Diagnosis and Treatment

Brasov, , Romania

Site Status

Scientfic Research Institute of Pulmonology

Moscow, , Russia

Site Status

Diagnostic Children Hospital, Department of Pediatrics and Adolescent Medicine, Department of Pediatric Pulmonary, Allergology and Endocrinology

Mytishchi, , Russia

Site Status

Children's City Hospital of Saint Olga

Saint Petersburg, , Russia

Site Status

First St. Petersburg State Pavlov Medical University

Saint Petersburg, , Russia

Site Status

Clinical for Pulmonary Diseases, Clinical Center of Serbia

Belgrade, , Serbia

Site Status

Institute for Pulmonary Disease of Vojvodina

Kamenitz, , Serbia

Site Status

Institute for Child and Youth Health Care of Vojvodina

Novi Sad, , Serbia

Site Status

Children's faculty hospital with polyclinic Banska Bystrica

Banská Bystrica, , Slovakia

Site Status

Children's Faculty Hospital Kosice

Košice, , Slovakia

Site Status

Unidad de Fibrosis Quistica Adultos

Barcelona, , Spain

Site Status

Unidad de Fibrosis Quistica Pediatria

Barcelona, , Spain

Site Status

Unidad de Fibrosis Quistica y Transplante Pulmonar

Valencia, , Spain

Site Status

Skane University Hospital

Lund, , Sweden

Site Status

Karolinska University Hospital

Stockholm, , Sweden

Site Status

Liverpool Heart and Chest Hospital

Liverpool, Merseyside, United Kingdom

Site Status

Belfast City Hospital

Belfast, Northern Ireland, United Kingdom

Site Status

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, , United Kingdom

Site Status

University Hospitals Birmingham NHS Foundation Trust

Birmingham, , United Kingdom

Site Status

Royal Papworth Hospital NHS Foundation Trust

Cambridge, , United Kingdom

Site Status

Cardiff and Vale University Health Board

Llandough, , United Kingdom

Site Status

Barts Health NHS Trust

London, , United Kingdom

Site Status

Royal Brompton & Harefield NHS Foundation Trust

London, , United Kingdom

Site Status

Wolfson Cystic Fibrosis Centre City Hospital Campus

Nottingham, , United Kingdom

Site Status

University Hospital Southampton NHS Foundation Trust

Southampton, , United Kingdom

Site Status

Countries

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United States Austria Belgium Bulgaria Canada Czechia France Germany Greece Hungary Italy Netherlands Poland Portugal Romania Russia Serbia Slovakia Spain Sweden United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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JBT101-CF-002

Identifier Type: -

Identifier Source: org_study_id

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