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

NCT ID: NCT04064242

Last Updated: 2025-04-13

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

62 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-09-23

Study Completion Date

2023-12-12

Brief Summary

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

Detailed Description

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This was a subject and investigator blinded, randomized, placebo-controlled, parallel-group, repeat-dose, multicenter, non-confirmatory study of CMK389 in chronic pulmonary sarcoidosis. This study investigated the safety and efficacy of 10 mg/kg CMK389 administered intravenously (i.v.) every 4 weeks for a total of 4 doses, versus placebo

Conditions

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Pulmonary Sarcoidosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Participants will be assigned to one of two treatment arms, either CMK389 or placebo.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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CMK389

CMK389 10 mg/kg i.v. every 4 weeks for a total of 4 doses

Group Type EXPERIMENTAL

CMK389

Intervention Type DRUG

single i.v. dose every 4 weeks

Placebo

Placebo i.v. every 4 weeks for a total of 4 doses

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

single i.v. dose every 4 weeks

Interventions

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CMK389

single i.v. dose every 4 weeks

Intervention Type DRUG

Placebo

single i.v. dose every 4 weeks

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Subjects must have a body mass index (BMI) at screening within the range of 18 - 46 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 \<20% (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
* Active cardiac sarcoidosis requiring treatment. Inactive cardiac sarcoidosis or stable cardiac sarcoidosis not requiring treatment are permissible.
* 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 use of any inhaled substance, including but not limited to tobacco, marijuana products and use of electronic cigarette or vaping device, and excluding inhalers or nebulizers prescribed for pulmonary sarcoidosis
* 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 not due to Cardiac Sarcoidosis and indicating significant risk of safety for patients participating in the study
* A diagnosis of Lofgren's syndrome
* A history of pancreatitis
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Novartis Investigative Site

Birmingham, Alabama, United States

Site Status

Univ of Florida College of Medicine x

Gainesville, Florida, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

John Hopkins Asthma And Alrgy Cntr

Baltimore, Maryland, United States

Site Status

Icahn School Of Med At Mount Sinai .

New York, New York, United States

Site Status

East Carolina University .

Greenville, North Carolina, United States

Site Status

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Site Status

Novartis Investigative Site

Brno, , Czechia

Site Status

Novartis Investigative Site

Olomouc, , Czechia

Site Status

Novartis Investigative Site

Aarhus N, , Denmark

Site Status

Novartis Investigative Site

Hellerup, , Denmark

Site Status

Novartis Investigative Site

Odense C, , Denmark

Site Status

Novartis Investigative Site

Heidelberg, Baden-Wurttemberg, Germany

Site Status

Novartis Investigative Site

Essen, , Germany

Site Status

Novartis Investigative Site

Frankfurt, , Germany

Site Status

Novartis Investigative Site

Hamburg, , Germany

Site Status

Novartis Investigative Site

Hanover, , Germany

Site Status

Novartis Investigative Site

Bialystok, , Poland

Site Status

Novartis Investigative Site

Lodz, , Poland

Site Status

Novartis Investigative Site

Warsaw, , Poland

Site Status

Novartis Investigative Site

Edinburgh, , United Kingdom

Site Status

Novartis Investigative Site

London, , United Kingdom

Site Status

Countries

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United States Czechia Denmark Germany Poland United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=2426

A Plain Language Trial Summary is available on www.novctrd.com

Other Identifiers

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CCMK389X2201

Identifier Type: -

Identifier Source: org_study_id

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