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

• Register: ClinicalTrials.gov
• Last refreshed on: 4 March 2024
• Main ID: NCT04064242
• Date of registration: 20/08/2019
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Study of Efficacy, Safety and Tolerability of CMK389 in Patients With Chronic Pulmonary Sarcoidosis
• Scientific title: 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
• Date of first enrolment: September 23, 2020
• Target sample size: 62
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04064242
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Czechia, Denmark, Germany, Poland, United Kingdom, United States

Key inclusion & exclusion criteria

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

Other protocol-defined inclusion/exclusion criteria may apply



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pulmonary Sarcoidosis

Intervention(s)

Drug: Placebo

Drug: CMK389

Primary Outcome(s)

Forced Vital Capacity [Time Frame: Baseline to Week 16]

Secondary Outcome(s)

Pharmacokinetics of CMK389 maximum concentration (Cmax) [Time Frame: Day 1 through Week 28]

Composite index of pulmonary physiology and exercise capacity [Time Frame: Baseline to Week 16]

Exercise capacity [Time Frame: Baseline to Week 16]

Pharmacokinetics of CMK389 trough concentration (Ctrough) [Time Frame: Day 1 through Week 28]

Steroid use (mg days) [Time Frame: Baseline to Week 16]

[18F]-fluorodeoxyglucose positron emission tomography/computed tomography [Time Frame: Baseline to Week 16]

Pulmonary physiology [Time Frame: Baseline to Week 16]

Secondary ID(s)

CCMK389X2201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors