Kiacta in Sarcoidosis

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Why Study Kiacta in Sarcoidosis?

TLS has begun a collaboration with Auven Therapeutics to develop Kiacta in sarcoidosis. Kiacta is a compound developed by Auven Therapeutics for AA amyloidosis and sarcoidosis. TLS is assisting Auven on the sarcoidosis aspect of the program.

  • Sarcoidosis is a disease of unknown origin with autoimmune features that causes granulomatous inflammation. It often affects the lungs and can cause pulmonary scarring.
     
  • The current treatments for pulmonary sarcoidosis include corticosteroids and immunomodulatory medications, which may cause severe side effects.
     
  • Kiacta is a molecule specifically designed to inhibit amyloid deposition into tissues. It presents the opportunity for a safer treatment than corticosteroids.
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An Opportunity for Publication

All participants will be able to review and comment on drafts of the protocol as they are produced, and they will be mailed a TLS Development Team membership certificate to show their support for open innovation.

Participating in the Protocol Builder also enters you into a friendly competition among the research community. When we identify the researchers who have provided the best input for a certain section of the Protocol Builder, we will name up to three winners of the section.

By winning a section, researchers will enter into a discussion with the TLS study team on how to draft the best protocol. When this protocol is published, the top researchers will have co-authorship rights and an ongoing say into the design and implementation of the trial.

Protocol Builder Results

Rationale

Should Kiacta be investigated for its potential ability to improve pulmonary function by inhibiting granuloma formation in sarcoidosis?

We have not yet reached a broad consensus on our rationale. Do you have anything to add?

We want to hear what patients want most out of a new treatment for sarcoidosis.

Please comment on the proposed rationale in the Protocol Builder itself or in the forum.

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Population

Which segment of patients do you think we should include in our study population?

What's your experience having sarcoidosis.

Please respond to specific population criteria in the Protocol Builder.

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Study Design

This study requires steroid run-in and tapering periods. The specifics of which need to be determined through crowd input.

This study requires patients to take an oral steroid, but many questions remain on how best to standardize steroid dosage.

We'd like patients to weigh the pros and cons of a proposed trial design.

Please respond to a specific dosage regimen for tapering patients' steroid levels.

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Primary Endpoint

Our propsed primary endpoint:

  • Absolute improvement in percent predicted FVC and/or FEV1 at 12 months compared to baseline.

What is an appropriate primary endpoint for a clinical trial in patients with pulmonary sarcoidosis?

Which clinical outcomes would make a clinical trial most worthwhile?

Researchers can evaluate specific possible endpoints in the Protocol Builder.

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Secondary Endpoints

What other endpoints should we monitor?

Please provide additional aspects of sarcoidosis that could be meaningful secondary endpoints.

Researchers can respond to specific possible secondary endpoints.

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Statistics

How should we plan to statistically analyze our data?

Patients are being asked about their willingness to enroll in a clinical trial for Kiacta in order to estimate participation and/or dropout rates for the trial.

In this section, you can critique our plans related to statistical measures in this trials, such as the number of patients screened, number of patients enrolled, and estimated dropout rate.

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Safety

Do you have any safety concerns?

Do you have any safety concerns?

 

Please help us review Kiacta's safety profile.

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Telemedicine

We propose using telemonitoring for this study in the following ways:

  • Portable Spirometry – forced vital capacity and forced expiratory volume
  • Vital signs - blood pressure, pulse, respirations
  • Pulse oximetry - O2 saturation
  • Body weight, height
  • Medication monitoring
  • Diffusing capacity of lung for carbon monoxide (DLCO)
  • Six Minute Walk test
  • Video Visits

In what ways can this study be executed remotely?

Here you can share your knowledge of how sarcoidosis can be monitored at home.

How can we best execute this study remotely?

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Data Visualization

Please help us create a data visualization plan.

Please continue the discussion on data transparency by clicking the comments button below.

We would like researchers' opinions on how to share our study data with the research community.

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