Clinical trial NCT04882124: Study of Effect of CSJ117 on Symptoms, Pharmacodynamics and Safety in Patients With COPD
Official title: A 12-week Randomized, participant-and Investigator-blinded, Placebo-controlled, Parallel Group Study to Explore the Efficacy, Pharmacodynamics, Safety, and Pharmacokinetics of Two Doses of Inhaled CSJ117 in Adults With Chronic Obstructive Pulmonary Disease (COPD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Pulmonary Disease, Chronic Obstructive
Keywords: Chronic Obstructive Pulmonary Disease
COPD
CSJ117
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2021-05-11
Enrollment: 38
Sex: ALL
Minimum age (years): 40
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a Phase 2 study in patients with chronic obstructive pulmonary disease (COPD) to assess the efficacy, pharmacodynamics (PD), pharmacokinetics (PK), and safety of two dose levels of CSJ117 in comparison to placebo. For this, the impact of CSJ117 on disease symptom burden and lung function will be explored.

Detailed description: This is a randomized, participant- and investigator-blinded, placebo-controlled, parallel group, multicenter study with two fixed-dose levels of CSJ117 (4 mg, 8 mg) administered once-daily via oral inhalation over 12 weeks in participants with COPD, prone for exacerbations and symptomatic at baseline despite background maintenance triple therapy (inhaled corticosteroids/long acting beta-2 agonist/long acting muscarinic antagonist (ICS/LABA/LAMA) combination, standard of care in this population) for at least three months prior to screening.

The study will include:

* Screening period of up to 2 weeks to assess eligibility.
* Run-in period of 4 weeks where participants will be placed on fixed-dose triple background therapy (ICS/LABA/LAMA) that will be used until the patient's last study treatment.
* Treatment period of 12 weeks. Participants will be stratified by eosinophil levels and then randomized 1:1:1 to either 4 mg CSJ117, 8 mg CSJ117, or placebo.
* Follow-up period of 55 days following the last dose of study drug. The primary objective of the study is to assess the effect of CSJ117 on disease/symptom burden after 12 weeks of treatment.

Eligibility criteria: Inclusion Criteria:

* Male and female COPD patients aged ≥40 years, who have signed an Informed Consent Form prior to initiation of any study-related procedure
* Current or ex-smokers who have a smoking history of at least 10 pack years
* Patients who have been treated with a triple combination of LABA/LAMA/ICS for the last 3 months prior to screening
* Patients with a documented diagnosis of COPD for at least 1 year prior to screening visit

Exclusion Criteria:

* Patients with a past or current medical history of asthma
* Patients who have had a COPD exacerbation that required treatment with antibiotics and/or oral corticosteroids and/or hospitalization, or a respiratory tract infection in the 4 weeks prior to screening, or hospitalization between screening and prior to treatment.
* Use of other investigational drugs (approved or unapproved) within 30 days or 5 half-lives prior to screening, or until the expected pharmacodynamic effect has returned to baseline (e.g., biologics), whichever is longer; or longer if required by local regulations
* Pregnant or nursing (lactating) women, and women of childbearing potential not willing to use acceptable effective methods of contraception during study participation
* Patients with a body mass index (BMI) of more than 40 kg/m2 Other protocol-defined inclusion/exclusion criteria may apply

Primary outcomes:
measure: Change from baseline in E-RS score
timeFrame: Baseline, 12 weeks
Secondary outcomes:
measure: Change from baseline in CAT score
timeFrame: Baseline, 12 weeks
measure: Change from baseline in SGRQ-C score
timeFrame: Baseline, 12 weeks
measure: Response in E-RS in total score decrease from baseline
timeFrame: Baseline, 12 weeks
measure: Response in CAT in total score decrease from baseline
timeFrame: Baseline, 12 weeks
measure: Response in SGRQ-C in total score decrease from baseline
timeFrame: Baseline, 12 weeks
measure: Change from baseline in trough FEV1 after 2, 6, and 12 weeks of treatment
timeFrame: Baseline, 2, 6 and 12 weeks
measure: Puffs of rescue medication per day
timeFrame: 12 weeks
measure: Time to COPD exacerbations via EXACT
timeFrame: 12 weeks
measure: Rate and severity of COPD exacerbations via EXACT
timeFrame: 12 weeks
measure: Time to COPD exacerbations via HCRU
timeFrame: 12 weeks
measure: Rate and severity of COPD exacerbations via HCRU
timeFrame: 12 weeks
measure: Pre-dose trough concentration (Ctrough) of CSJ117
timeFrame: 12 weeks
measure: Accumulation ratio (Racc) of CSJ117
timeFrame: 12 weeks
measure: Measuring anti-drug antibodies
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Andalusia
country: United States
facility: Novartis Investigative Site
state: Alabama
city: Crowley
country: United States
facility: Novartis Investigative Site
state: Louisiana
city: New Orleans
country: United States
facility: Novartis Investigative Site
state: Louisiana
city: Saint Charles
country: United States
facility: Novartis Investigative Site
state: Missouri
city: Saint Louis
country: United States
facility: Novartis Investigative Site
state: Missouri
city: Huntersville
country: United States
facility: Novartis Investigative Site
state: North Carolina
city: Shelby
country: United States
facility: Novartis Investigative Site
state: North Carolina
city: South Brisbane
country: Australia
facility: Novartis Investigative Site
state: Queensland
city: Sainte Foy
country: Canada
facility: Novartis Investigative Site
state: Quebec
city: Ostrava Poruba
country: Czechia
facility: Novartis Investigative Site
state: Czech Republic
city: Balassagyarmat
country: Hungary
facility: Novartis Investigative Site
city: Godollo
country: Hungary
facility: Novartis Investigative Site
city: Szeged
country: Hungary
facility: Novartis Investigative Site
city: Nagoya
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Naka-gun
country: Japan
facility: Novartis Investigative Site
state: Ibaraki
Study officials:
References: