Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis

NCT03451045 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: JBT101-CF-002
• Study Type: interventional
• Target: 447
• Locations: 20 countries
• Sites: 102

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.

Conditions

Cystic Fibrosis

Keywords

Pulmonary exacerbation, lenabasum, JBT-101

Outcome Measures

Primary Outcomes (1)

• Pulmonary Exacerbation (PEx) Rate Over 28 Weeks

  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.

  28 weeks (Baseline Day 0 to Week 28)

Secondary Outcomes (5)

• Pulmonary Exacerbation (PEx) Rate

  28 weeks (Baseline Day 0 to Week 28)

• Time to First New Pulmonary Exacerbation (PEx)

  28 weeks (Baseline Day 0 to Week 28)

• Pulmonary Exacerbation (PEx)

  28 weeks (Baseline Day 0 to Week 28)

• CFQ-R Respiratory Symptom Domain

  28 weeks (Change from Baseline Day 0 to Week 28)

• FEV1 % Predicted

  28 weeks (Change from Baseline Day 0 to Week 28)

Study Arms (3)

Lenabasum 20 mg BID

EXPERIMENTAL

Drug: Lenabasum 20 mg

Lenabasum 5 mg BID

EXPERIMENTAL

Drug: Lenabasum 5 mg

Placebo BID

PLACEBO COMPARATOR

Other: Placebo

Interventions

Lenabasum 20 mg DRUG

Subjects will receive lenabasum 20 mg twice daily.

Lenabasum 20 mg BID

Lenabasum 5 mg DRUG

Subjects will receive lenabasum 5 mg twice daily.

Lenabasum 5 mg BID

Placebo OTHER

Subjects will receive placebo twice daily.

Placebo BID

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥ 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Corbus Pharmaceuticals Inc.: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (105)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

Location

Miller Children's Hospital

Long Beach, California, 90806, United States

Location

National Jewish Health

Denver, Colorado, 80206, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Central Florida Pulmonary Group, PA

Orlando, Florida, 32803, United States

Location

USF Center for Advance Lung Disease

Tampa, Florida, 33606, United States

Location

Emory Children's Center

Atlanta, Georgia, 30322, United States

Location

Northwestern Memorial Hospital

Chicago, Illinois, 60611, United States

Location

The Cystic Fibrosis Institute

Glenview, Illinois, 60025, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Dartmouth-Hitchcock Medical Center (main location)

Lebanon, New Hampshire, 03756, United States

Location

Dartmouth-Hitchcock Manchester (satellite site)

Manchester, New Hampshire, 03104, United States

Location

Atlantic Health Children's Hospital

Morristown, New Jersey, 07960, United States

Location

Rutgers Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

Location

New York Medical College

Hawthorne, New York, 10532, United States

Location

North Shore LIJ Health System

New Hyde Park, New York, 11040, United States

Location

Mount Sinai Beth Israel

New York, New York, 10003, United States

Location

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

Cleveland, Ohio, 44106, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

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

Hershey, Pennsylvania, 17033, United States

Location

Drexel University College of Medicine

Philadelphia, Pennsylvania, 19102, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Sanford Children's Specialty Clinic

Sioux Falls, South Dakota, 57105, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

The University of Texas Health Science Center at Tyler

Tyler, Texas, 75708, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

University Hospital and UW Health Clinics

Madison, Wisconsin, 53792, United States

Location

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

Location

Medical University of Vienna

Vienna, Austria

Location

Universitair Ziekenhuis Brussel

Brussels, Belgium

Location

Medical Center Prolet EOOD

Rousse, Bulgaria

Location

UMHAT Alexandrovska

Sofia, Bulgaria

Location

MHAT Sveta Marina EAD

Varna, Bulgaria

Location

Centre hospitalier de l'Université de Montréal (CHUM)

Montreal, Canada

Location

St. Michael's Hospital

Toronto, Canada

Location

The Hospital for Sick Children

Toronto, Canada

Location

St. Paul's Hospital

Vancouver, Canada

Location

Motol University Hospital

Prague, Czechia

Location

Centre de Référence de la Mucoviscidose

Bron, France

Location

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

Bron, France

Location

Service de Pediatrie Medico-Chirurgicale et Genetique

Dijon, France

Location

CHRU de Montpellier

Montpellier, France

Location

CRCM Enfant de Nancy

Nancy, France

Location

CHU de Nice

Nice, France

Location

Centre de Recherche en Explorations Fonctionnelles (CREF)

Paris, France

Location

CRCM Hôpital Necker

Paris, France

Location

Foundation ILDYS

Roscoff, France

Location

Nouvel Hopital Civil Strasbourg

Strasbourg, France

Location

Charité Universitätsmedzin

Berlin, Germany

Location

Catholic Hospital Bochum - St. Josef-Hospital

Bochum, Germany

Location

University Hospital Essen

Essen, Germany

Location

University Medicine Essen Ruhrlandklinik

Essen, Germany

Location

Goethe University Children´s Hospital

Frankfurt, Germany

Location

Hannover Medical School

Hanover, Germany

Location

University Hospital Jena

Jena, Germany

Location

Klinikum der Ludwig Maximilian Universität München

München, Germany

Location

General Hospital of Thessaloniki Ippokratio

Thessaloniki, Greece

Location

National Koranyi Institute of Pulmonology, Department of Cystic Fibrosis

Budapest, Hungary

Location

University of Debrecen - Kenezy Gyula University Hospital

Debrecen, Hungary

Location

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

Kecskemét, Hungary

Location

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

Mosdós, Hungary

Location

Pediatric Pulmonology, Törökbálint, Hungary

Törökbálint, Hungary

Location

Centro Regionale Toscano di Riferimento per la Fibrosi Cistica

Florence, Italy

Location

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

Genova, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, Italy

Location

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Milan, Italy

Location

Azienda Ospedaliera Universitaria Verona

Verona, Italy

Location

Radbound UMC

Nijmegen, Netherlands

Location

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

Dziekanów Leśny, 05-092, Poland

Location

Oddzial Pediatrii z Pododdzialem Leczenia Mukowiscydozy

Gdansk, Poland

Location

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

Rabka-Zdrój, 34-700, Poland

Location

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

Rzeszów, Poland

Location

Hospital de Santa Maria

Lisbon, Portugal

Location

Medial Center for Ambulatory Diagnosis and Treatment

Brasov, Romania

Location

Scientfic Research Institute of Pulmonology

Moscow, Russia

Location

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

Mytishchi, Russia

Location

Children's City Hospital of Saint Olga

Saint Petersburg, Russia

Location

First St. Petersburg State Pavlov Medical University

Saint Petersburg, Russia

Location

Clinical for Pulmonary Diseases, Clinical Center of Serbia

Belgrade, Serbia

Location

Institute for Pulmonary Disease of Vojvodina

Kamenitz, Serbia

Location

Institute for Child and Youth Health Care of Vojvodina

Novi Sad, Serbia

Location

Children's faculty hospital with polyclinic Banska Bystrica

Banská Bystrica, Slovakia

Location

Children's Faculty Hospital Kosice

Košice, Slovakia

Location

Unidad de Fibrosis Quistica Adultos

Barcelona, Spain

Location

Unidad de Fibrosis Quistica Pediatria

Barcelona, Spain

Location

Unidad de Fibrosis Quistica y Transplante Pulmonar

Valencia, Spain

Location

Skane University Hospital

Lund, Sweden

Location

Karolinska University Hospital

Stockholm, Sweden

Location

Liverpool Heart and Chest Hospital

Liverpool, Merseyside, United Kingdom

Location

Belfast City Hospital

Belfast, Northern Ireland, United Kingdom

Location

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, United Kingdom

Location

University Hospitals Birmingham NHS Foundation Trust

Birmingham, United Kingdom

Location

Royal Papworth Hospital NHS Foundation Trust

Cambridge, United Kingdom

Location

Cardiff and Vale University Health Board

Llandough, United Kingdom

Location

Barts Health NHS Trust

London, United Kingdom

Location

Royal Brompton & Harefield NHS Foundation Trust

London, United Kingdom

Location

Wolfson Cystic Fibrosis Centre City Hospital Campus

Nottingham, United Kingdom

Location

University Hospital Southampton NHS Foundation Trust

Southampton, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

lenabasum

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

Results Point of Contact

• Title: Head of Clinical Operations
• Organization: Corbus Pharmaceuticals, Inc.

info@corbuspharma.com

6179630100

Study Officials

• James Chmiel, MD

  Indiana University School of Medicine/Riley Physicians Pulmonary

  PRINCIPAL INVESTIGATOR

• J. Stuart Elborn, MD

  National Heart and Lung Institute, Imperial College

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

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

Model Details: Multicenter, double-blind, randomized, placebo-controlled, parallel group study.

Study Record Dates

• First Submitted: February 23, 2018
• First Posted: March 1, 2018
• Study Start: December 22, 2017
• Primary Completion: June 17, 2020
• Study Completion: June 17, 2020
• Last Updated: January 18, 2023
• Results First Posted: January 18, 2023

Record last verified: 2022-12

Locations

GB (10); RO (1); SE (2); DE (8); CA (4); HU (5); FR (10); GR (1); BU (3); BE (1); SL (2); IT (5); NL (1); PL (4); AU (2); ES (3); CZ (1); PT (1); US (34); RU (4)