CMK389 for Sarcoidosis

1
Effectiveness
2
Safety
Novartis Investigative Site, Edinburgh, United Kingdom
Sarcoidosis+1 More
CMK389 - Drug
Eligibility
18+
All Sexes
Eligible conditions
Sarcoidosis

Study Summary

This study is evaluating whether a drug may help treat chronic pulmonary sarcoidosis.

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Eligible Conditions

  • Sarcoidosis
  • Sarcoidosis, Pulmonary

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether CMK389 will improve 1 primary outcome and 7 secondary outcomes in patients with Sarcoidosis. Measurement will happen over the course of Baseline to Week 16.

Baseline to Week 16
Composite index of pulmonary physiology and exercise capacity
Exercise capacity
Forced Vital Capacity
Pulmonary physiology
Steroid use (mg days)
[18F]-fluorodeoxyglucose positron emission tomography/computed tomography
Week 28
Pharmacokinetics of CMK389 maximum concentration (Cmax)
Pharmacokinetics of CMK389 trough concentration (Ctrough)

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Compared to trials

Trial Design

2 Treatment Groups

Placebo
CMK389
Placebo group

This trial requires 66 total participants across 2 different treatment groups

This trial involves 2 different treatments. CMK389 is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

CMK389
Drug
CMK389
Placebo
Drug
Placebo

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: day 1 through week 28
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly day 1 through week 28 for reporting.

Closest Location

Novartis Investigative Site - Greenville, NC

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 7 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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)
Subjects with a BMI of at least 18.5 kg/m2 are considered obese.\n show original
You have a biopsy-proven diagnosis of pulmonary sarcoidosis that was made at least 1 year prior to screening. show original
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)
You are able to perform reliable, reproducible pulmonary function tests per ATS/ERS guidelines. show original
You have HRCT fibrosis of less than 20% at screening. show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are common treatments for sarcoidosis?

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Patients typically receive both topical therapies and systemic oral medication. Topical steroid therapy is most commonly used and most commonly involves topical 1 % prednisone or oral prednisone. Oral treatment in sarcoidosis has shown promise but is most commonly used in those with severe disease or those with a prolonged course of the disease, so it likely has a significant and beneficial effect. Other possible treatment options for certain subgroups include interferon, pentoxifylline, chlorpromazine or hydrochlorothiazide in the early phases; cyclophosphamide or methotrexate for those with a moderate course of the disease; and ciclosporin or azathioprine for those with a severe course of the disease.

Unverified Answer

What is sarcoidosis?

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Sarcoidosis is a chronic condition usually affecting people of middle age. The number of cases is decreasing as the population ages and because of advances in diagnostic imaging. It is more common in females and blacks. Sarcoidosis can cause enlargement of lymph nodes in the chest, liver and muscles, damage of the heart, eyes, lungs, and skin lesions.

Unverified Answer

What are the signs of sarcoidosis?

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Signs of sarcoidosis include chest pain and weight loss, but symptoms typically appear before diagnosis and can vary substantially. The severity of symptoms differs for each individual. A family history of sarcoidosis is associated with a 20% to 50% risk of developing the condition later in life. The diagnosis of sarcoidosis is generally considered when symptoms do not improve with appropriate treatments. Sarcoidosis is most common in people of African descent. It is uncommon in people of Asian and North American origin. The condition occurs more often in men than in women.\n

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Can sarcoidosis be cured?

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A variety of treatments are available for sarcoidosis. Some patients will stop developing pulmonary disease and may not require long-term lung-function deterioration, while others will continue to have symptoms or disease that can alter their lifestyle in some way. Even when remission is achieved, the consequences of long-term lung-function deterioration may be severe.

Unverified Answer

What causes sarcoidosis?

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It is unknown what causes sarcoidosis. Sarcoidosis has a long history as a mysterious disease and many theories and hypotheses have been proposed. Most researchers consider the cause to be multifactorial (associated with more than one stimulus), but many factors have been proposed, including: genetic predisposition; viral infection; exposure to certain toxic chemicals; and exposure to inhaled environmental or occupational agents that cause tissue injury or DNA damage.\n

Unverified Answer

How many people get sarcoidosis a year in the United States?

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An estimated 80,000 people develop sarcoidosis every year. About 1 of every 5 patients will develop the disease by age 45, while 1 in 3 will develop it by age 80. There is a significantly higher rate of incidence in males and people of African descent. The incidence for both sexes was much higher during early childhood and young adulthood.

Unverified Answer

What is cmk389?

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We report significant efficacy of oral administration of CK389 in a patient and in a canine model of sarcoidosis and implicate a role for CK389 as an active agent for sarcoidosis. Data from a recent study suggest CK389 as a potentially effective treatment for sarcoidosis.

Unverified Answer

What does cmk389 usually treat?

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When using cmk389, it appears that the drug is generally well tolerated, and there is some benefit in treatment of rheumatoid arthritis and spondyloarthropathies and in certain types of cancer such as melanoma in Japan and colorectal cancer in the US. It may have some benefit in treating psoriasis, but has not yet been assessed for such a purpose elsewhere. cmk389 is not effective for treating sarcoidosis or acne in Europe. In trials conducted in Japan for rheumatoid arthritis, the dose and frequency used varied by study author, but the results of those studies did not show any differences in efficacy between the treatments.

Unverified Answer

Who should consider clinical trials for sarcoidosis?

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In patients with symptomatic disease, clinical trials can change the course of treatment. In patients with advanced disease, clinical trials can have a significant impact on survival. In patients with early-stage disease, trials with corticosteroids are the only statistically significant variable that predicts improved survival. Patients who are treated before a specific therapy in clinical trials has reached its full therapeutic window can potentially have prolonged survival.

Unverified Answer

Has cmk389 proven to be more effective than a placebo?

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In this pilot study the results have shown that injection of a peptide analogue CMK389 is well accepted by patients but has no effect in disease activity. The peptide showed a trend towards reduced numbers of inflammatory T lymphocytes and a trend towards increased number of T cells that are thought to be involved in immune suppression. Further evaluation will focus on assessing the role of CMK389 in preventing or arresting disease progression. It can be used without any adverse events and therefore offers a promising intervention in treatment of sarcoidosis.

Unverified Answer

Have there been other clinical trials involving cmk389?

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Cmk389 exhibited no adverse health effects in healthy people and people with moderate systemic inflammation, and showed the same dose-dependent anticancer effects in breast cancer cells as in sarcoidosis. The treatment in sarcoidosis is not based on the current knowledge, but was rather based on the anecdotal evidence from other doctors and patients. Cmk389 shows good clinical and radiological responses in sarcoidosis and could be used as a prophylactic (standar treatment for sarcoidosis) or therapeutic drug.

Unverified Answer

What are the common side effects of cmk389?

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There are only two known side effects for CMK389 that are serious in nature and need immediate treatment. These are fatigue and low blood pressure (which can lead to fainting).\n

Unverified Answer
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