Study of Efficacy, Safety and Tolerability of CMK389 in Patients With Chronic Pulmonary Sarcoidosis

A Subject and Investigator Blinded, Randomized, Placebo-controlled, Repeat-dose, Multicenter Study to Investigate Efficacy, Safety, and Tolerability of CMK389 in Patients With Chronic Pulmonary Sarcoidosis

ClinicalTrials.gov Identifier: NCT04064242

Novartis Reference Number: CCMK389X2201

Last Update: Nov 03, 2021

All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

The purpose of this proof of concept study is to determine whether CMK389 displays the safety and efficacy profile to support further development in chronic pulmonary sarcoidosis.

Condition

Pulmonary Sarcoidosis

Phase

Phase 2

Overall status

Recruiting

Enrollment count

66 participants

Start date

Sep 23, 2020

Completion date

Mar 13, 2023

Gender

All

Age(s)

18 Years - 75 Years (Adult, Older Adult)

Interventions

Drug

CMK389

single i.v. dose every 4 weeks

Drug

Placebo

single i.v. dose every 4 weeks

Eligibility Criteria

Inclusion Criteria:

Subjects must have a body mass index (BMI) at screening within the range of 18 - 43 kg/m2. BMI = Body weight (kg) / [Height (m)]2
Biopsy proven pulmonary sarcoidosis diagnosed > 1 year prior to screening
Scadding stage II, III or IV as determined by the most recent chest x-ray obtained within 12 months prior to screening or at screening (confirmed by the Investigator)
HRCT extent of fibrosis <15% (confirmed by the central imaging reader) at screening
Treatment with 5-15 mg/day prednisone (or prednisone oral equivalents) for ≥ 6 months prior to screening.
Co-medication with methotrexate or azathioprine for ≥ 6 months prior to screening (Note: hydroxychloroquine is allowed as background therapy but not required)
Able to perform reliable, reproducible pulmonary function test maneuvers per American Thoracic Society/European Respiratory Society (ATS/ERS) guidelines

Exclusion Criteria:

Diagnosis of significant pulmonary hypertension (WHO group 5) requiring pharmacological treatment
A known diagnosis of cardiac sarcoidosis
A known diagnosis of neurosarcoidosis
Forced vital capacity (FVC) <50% of predicted at screening (central read)
Modified British Medical Research Council (mMRC) dyspnea scale ≥ 3 at screening
Concomitant treatment with leflunomide, cyclophosphamide, mycophenolate, infliximab, etanercept, adalimumab, golimumab, ustekinumab, roflumilast, pentoxifylline, and abatacept within 12 weeks of screening
Prior treatment with rituximab, canakinumab, anakinra, and tocilizumab
Current smokers, defined as inhaled use of tobacco products
Any conditions or significant medical problems which in the opinion of the investigator and in consultation with the sponsor, immunocompromises the patient and/or places the patient at unacceptable risk for immunomodulatory therapy
Contraindication to FDG-PET scan investigations such as severe claustrophobia or uncontrolled diabetes
History or current diagnosis of ECG abnormalities indicating significant risk of safety for patients participating in the study
A diagnosis of Lofgren's syndrome
A history of pancreatitis
Other protocol-defined inclusion/exclusion criteria may apply