Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 (Lenabasum) in Cystic Fibrosis

A Phase 2, Double-blind, Randomized, Placebo-controlled Multicenter Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 in Cystic Fibrosis

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of JBT-101 in adult subjects with cystic fibrosis (CF).

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: JBT-101 (lenabasum); Other: Placebo

Detailed Description

An interventional, double-blind, randomized, placebo-control design will be used to test safety, tolerability, pharmacokinetics, and efficacy of JBT-101 in 70 subjects ≥ 18 and < 65 years of age with documented cystic fibrosis. There will up a screening period of up to 28 days, 84 days treatment period, and 28 days follow-up.

• Study Type: Interventional
• Enrollment (Actual): 85
• Phase: Phase 2

Contacts and Locations

Study Locations

• Belgium
  • Brussels, Belgium
    • Hôpital Erasme
• France
  • Montpellier, France
    • Hopital Arnaud de Villeneuve
  • Nice, France
    • Institution Hôpital Pasteur
  • Roscoff, France
    • Centre de Perharidy
• Germany
  • Hesse
    • Frankfurt am Main, Hesse, Germany, 60590
      • Christiane Herzog CF-Zentrum Frankfurt am Main
  • North Rhine-Westphalia
    • Essen, North Rhine-Westphalia, Germany, 45127
      • UNI Essen Abt.Pneumologie
• Italy
  • Milano, Italy, 20122
    • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
  • Milano, Italy, 20122
    • Fondazione Irccs Ca' Granda - Ospedale Maggiore Policlinico
  • Verona, Italy
    • AOUI di Verona - Ospedale Borgo Trento UOC Fibrosi Cistica
• Poland
  • Gdańsk, Poland, 80-308
    • Szpital Dziecięcy Polanki im. Macieja Płażyńskiego w Gdańsku Spółka z o.o. Poradnia Leczenia Mukowiscydozy
  • Rabka Zdrój, Poland, 34-700
    • Sanatorium Cassia-Villa Medica s.c
  • Rzeszów, Poland, 35-612
    • Podkarpacki Ośrodek Pulmunologii i Alergologii
  • Warszawa, Poland, 01-138
    • Instytut Gruźlicy i Chorób Płuc I Klinika Chorób Płuc
• United Kingdom
  • Belfast, United Kingdom
    • Belfast City Hospital
  • Glasgow, United Kingdom
    • Queen Elizabeth University Hospital
• United States
  • Arkansas
    • Little Rock, Arkansas, United States
      • University of Arkansas for Medical Sciences
  • California
    • Long Beach, California, United States
      • Long Beach Memorial Medical Center/Miller Children's and Women's Hospital
  • Colorado
    • Denver, Colorado, United States
      • National Jewish Health
  • Massachusetts
    • Boston, Massachusetts, United States
      • Boston Children's Hospital
    • Boston, Massachusetts, United States
      • Massachusetts General Hospital
  • New Jersey
    • New Brunswick, New Jersey, United States, 08903
      • Rutgers Robert-Wood Johnson Medical School
  • New York
    • New Hyde Park, New York, United States
      • North Shore Lij Health System
    • Valhalla, New York, United States
      • New York Medical College
  • Ohio
    • Cleveland, Ohio, United States
      • University Hospitals Cleveland Medical Center
  • South Carolina
    • Charleston, South Carolina, United States
      • Medical University of South Carolina
  • Texas
    • Dallas, Texas, United States
      • The University of Texas Southwestern Medical Center
    • Houston, Texas, United States
      • Texas Children's Hospital Clinical Care Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Documentation of a CF diagnosis as evidenced by 1 or more clinical features consistent with the CF phenotype and 1 or more of the following criteria:

  1. Sweat chloride equal to or greater than 60 mEq/L by quantitative pilocarpine iontophoresis test;
  2. Two well-characterized mutations in the CFTR gene
• FEV1 ≥ 40% predicted corrected
• Stable treatment of CF for 14 days before Visit 1

Exclusion Criteria:

• Severe or unstable CF, such as:

  1. Intravenous antibiotic treatment within 14 days before Visit 1
  2. Treatment with any corticosteroids > 10 mg per day or > 20 mg every other day oral prednisone or equivalent within 14 days before Visit 1

• Any one of the following values for laboratory tests at Screening:

  1. A positive pregnancy test (or at Visit 1);
  2. Hemoglobin < 10 g/dL
  3. Neutrophils < 1.0 x 10~9/L
  4. Platelets < 75 x 10~9/L
  5. Creatinine clearance < 50 ml/min according to modified Cockcroft-Gault equation
  6. Serum transaminases > 2.5 x upper normal limit
  7. Total bilirubin ≥ 1.5 x upper limit of normal
• Any other condition that, in the opinion of the Principal Investigator, is clinically significant and may put the subject at greater safety risk, influence response to study product, or interfere with study assessments.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Double

Number of Arms

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: JBT101 (lenabasum) 1 mg; JBT-101 1 mg once a day on Days 1-28	Drug: JBT-101 (lenabasum); Subjects will receive JBT-101 1 mg qd or JBT-101 5 mg qd on Days 1-28. Subjects will receive either JBT-101 20 mg q am (with placebo q pm) or JBT-101 20 mg twice a day on Days 29-84.
Experimental: JBT-101 (lenabasum) 5 mg; JBT-101 5 mg once a day on Days 1-28	Drug: JBT-101 (lenabasum); Subjects will receive JBT-101 1 mg qd or JBT-101 5 mg qd on Days 1-28. Subjects will receive either JBT-101 20 mg q am (with placebo q pm) or JBT-101 20 mg twice a day on Days 29-84.
Placebo Comparator: Placebo; Placebo once a day on Days 1-28.	Other: Placebo; Subjects will receive placebo once a day on Days 1-28, placebo q pm on Days 29-84 (with JBT-101 20 mg q am), or placebo bid on Days 29-84.
Experimental: JBT-101 (lenabasum) 20 mg QD; JBT-101 20 mg once a day on Days 29-84.	Drug: JBT-101 (lenabasum); Subjects will receive JBT-101 1 mg qd or JBT-101 5 mg qd on Days 1-28. Subjects will receive either JBT-101 20 mg q am (with placebo q pm) or JBT-101 20 mg twice a day on Days 29-84.
Experimental: JBT-101 (lenabasum) 20 mg BID; JBT-101 20 mg twice daily on Days 29-84.	Drug: JBT-101 (lenabasum); Subjects will receive JBT-101 1 mg qd or JBT-101 5 mg qd on Days 1-28. Subjects will receive either JBT-101 20 mg q am (with placebo q pm) or JBT-101 20 mg twice a day on Days 29-84.
Placebo Comparator: Placebo BID; Placebo twice daily on Days 29-84.	Other: Placebo; Subjects will receive placebo once a day on Days 1-28, placebo q pm on Days 29-84 (with JBT-101 20 mg q am), or placebo bid on Days 29-84.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants With Treatment Emergent Adverse Events.	84 days of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
JBT-101 (Lenabasum) Plasma Concentrations on Day 84	Plasma concentrations were reported for the lenabasum 20 mg QD, 20 mg BID, and placebo groups only, at Day 84.	Day 84

Collaborators and Investigators

• Sponsor: Corbus Pharmaceuticals Inc.
• Investigators: Principal Investigator: James Chmiel, M.D., University Hospitals Cleveland Medical Center, Cleveland, OH; Principal Investigator: J S Elborn, M.D., Queens University, Belfast, Northern Ireland, United Kingdom

Publications and helpful links

General Publications

• Chmiel JF, Flume P, Downey DG, Dozor AJ, Colombo C, Mazurek H, Sapiejka E, Rachel M, Constantine S, Conley B, Dgetluck N, Dinh Q, White B, Elborn JS; Lenabasum JBT101-CF-001 Study Group. Safety and efficacy of lenabasum in a phase 2 randomized, placebo-controlled trial in adults with cystic fibrosis. J Cyst Fibros. 2021 Jan;20(1):78-85. doi: 10.1016/j.jcf.2020.09.008. Epub 2020 Oct 1.

Study record dates

Study Major Dates

• Study Start (Actual): September 29, 2015
• Primary Completion (Actual): December 28, 2016
• Study Completion (Actual): December 28, 2016

Study Registration Dates

• First Submitted: June 4, 2015
• First Submitted That Met QC Criteria: June 4, 2015
• First Posted (Estimate): June 8, 2015

Study Record Updates

• Last Update Posted (Actual): April 4, 2018
• Last Update Submitted That Met QC Criteria: March 8, 2018
• Last Verified: March 1, 2018

More Information

Terms related to this study

• Keywords: JBT-101, Lenabasum, Cystic Fibrosis
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Cannabinoid Receptor Agonists; Cannabinoid Receptor Modulators; Lenabasum
• Other Study ID Numbers: JBT101-CF-001