Study of Safety, Tolerability and Efficacy of DFV890 in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)

Study Overview
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Brief Summary
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This is an open-label, single-arm, multiple dose, phase II study to assess safety, tolerability and clinical efficacy of DFV890 in participants with FCAS. The study includes a screening period, a treatment period and a follow-up period. At most, the study will last up to 7 months (and up to 13 months for participants with a historical screening cold challenge prior to protocol amendment 04).

Official Title
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An Open-label, Single Arm Phase II Study of DFV890 to Assess the Safety, Tolerability and Efficacy in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)

Conditions
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Familial Cold Autoinflammatory Syndrome

Intervention / Treatment
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* Drug: DFV890


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Written informed consent must be obtained before any study-specific assessment is performed Body mass index within the range of 18-35 kg/m2 Patients with a genetic diagnosis of FCAS Patients with a clinical history and investigations consistent with FCAS Exclusion Criteria: Anti-rejection and/or immunomodulatory drugs must be discontinued (please, see protocol for further details) Clinically significant, suspected active or chronic bacterial (including Mycobacterium tuberculosis), viral or fungal infection within 30 days prior to Day 1. Patients with innate (e.g. TLR immunodeficiencies, defects in IFN-γ signaling) or acquired immune deficiencies (e.g. AIDS). Presence of human immunodeficiency virus (HIV) infection, hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (anti-HBc), or hepatitis C antibodies at screening. Live vaccines within 4 weeks of Day 1 Pregnant or nursing (lactating) women. Women of child-bearing potential unless they are using highly effective methods of contraception. Other protocol-defined inclusion/exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: DFV890<br>DFV890 | Drug: DFV890<br>* DFV890<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ratio of fold change from baseline for white cell count (WCC) | To assess the efficacy of DFV890 in patients with FCAS | At pre-baseline and up to 24 hours post-baseline | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number and severity of safety assessments and adverse events | To evaluate the safety and tolerability of DFV890 in patients with FCAS | Through study completion, approximately 4 months | 
| Change from pre-baseline to post-baseline in Physician global assessment of autoinflammatory disease activity | The Physician global assessment of autoinflammatory disease activity is a questionnaire to be completed by the Investigator. It uses a 5- point rating scale: absent, minimal, mild, moderate, and severe. Lower scores represent better outcomes. | At pre-baseline and up to 24 hours post-baseline | 
| Change from pre-baseline to post-baseline in Physician's severity assessment of autoinflammatory disease signs and symptoms | The Physician's severity assessment of autoinflammatory disease signs and symptoms is a questionnaire to be completed by the Investigator. It uses a 5-point rating scale: absent, minimal, mild, moderate, and severe. Lower scores represent better outcomes. | At pre-baseline and up to 24 hours post-baseline | 
| Change from pre-baseline to post-baseline in Patient's global assessment of disease activity | Patient's global assessment of disease activity is a questionnaire to be completed by the patient. It uses a 5-point rating scale: absent, minimal, mild, moderate, and severe. The patient will select a rating based on the patient's current disease activity at the time of the assessment. Lower scores represent better outcomes. | At pre-baseline and up to 24 hours post-baseline | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Familial Cold Auto-inflammatory Syndrome, FCAS, DFV890, NLRP3 inhibitor, inflammasome inhibition, Cryopyrin-associated periodic syndromes, CAPS