Clinical trial NCT02465450: Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 (Lenabasum) in Cystic Fibrosis
Official title: A Phase 2, Double-blind, Randomized, Placebo-controlled Multicenter Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 in Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cystic Fibrosis
Keywords: JBT-101, Lenabasum, Cystic Fibrosis
Lead sponsor: Corbus Pharmaceuticals Inc.
Sponsor class: INDUSTRY
First posted: 2015-06-08
Enrollment: 85
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events.
timeFrame: 84 days of treatment
Secondary outcomes:
measure: JBT-101 (Lenabasum) Plasma Concentrations on Day 84
timeFrame: Day 84
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: Long Beach
country: United States
facility: Long Beach Memorial Medical Center/Miller Children's and Women's Hospital
state: California
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: New Brunswick
country: United States
facility: Rutgers Robert-Wood Johnson Medical School
state: New Jersey
city: New Hyde Park
country: United States
facility: North Shore LIJ Health System
state: New York
city: Valhalla
country: United States
facility: New York Medical College
state: New York
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Dallas
country: United States
facility: The University of Texas Southwestern Medical Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital Clinical Care Center
state: Texas
city: Brussels
country: Belgium
facility: Hôpital Erasme
city: Montpellier
country: France
facility: Hôpital Arnaud de Villeneuve
city: Nice
country: France
facility: Institution Hôpital Pasteur
city: Roscoff
country: France
facility: Centre de Perharidy
city: Frankfurt am Main
country: Germany
facility: Christiane Herzog CF-Zentrum Frankfurt am Main
state: Hesse
city: Essen
country: Germany
facility: UNI Essen Abt.Pneumologie
state: North Rhine-Westphalia
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
city: Milano
country: Italy
facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
city: Verona
country: Italy
facility: AOUI di Verona - Ospedale Borgo Trento UOC Fibrosi Cistica
city: Gdańsk
country: Poland
facility: Szpital Dziecięcy Polanki im. Macieja Płażyńskiego w Gdańsku Spółka z o.o. Poradnia Leczenia Mukowiscydozy
city: Rabka Zdrój
country: Poland
facility: Sanatorium Cassia-Villa Medica s.c
city: Rzeszów
country: Poland
facility: Podkarpacki Ośrodek Pulmunologii i Alergologii
city: Warszawa
country: Poland
facility: Instytut Gruźlicy i Chorób Płuc I Klinika Chorób Płuc
city: Belfast
country: United Kingdom
facility: Belfast City Hospital
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital
Study officials:
affiliation: University Hospitals Cleveland Medical Center, Cleveland, OH
name: James Chmiel, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Queens University, Belfast, Northern Ireland, United Kingdom
name: J S Elborn, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.