Study of safety, tolerability and efficacy of DFV890 in participants with familial cold auto-inflammatory syndrome (FCAS)

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)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-005948-33-DE

Enrollment

Registered

2021-02-26

Start date

2021-07-26

Completion date

Unknown

Last updated

2024-04-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Familial Cold Autoinflammatory Syndrome (FCAS) MedDRA version: 25.1 Level: PT Classification code 10068850 Term: Cryopyrin associated periodic syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: DFV890 Product Code: DFV890 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: Not yet established Current Sponsor code: DFV890 Concentration unit: mg milligram(s) Concentratio

Eligibility

Sex/Gender

All

Inclusion criteria

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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 3 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 3

Exclusion criteria

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-? 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.

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the efficacy of DFV890 to reduce cold-induced inflammation in participants with FCAS.;Secondary Objective: - To assess safety and tolerability of DFV890. - To assess the efficacy of DFV890 to improve the signs and symptoms of FCAS. - To assess the effect of DFV890 on patient reported outcomes.;Primary end point(s): Change from baseline for white cell count (WCC).;Timepoint(s) of evaluation of this end point: Pre-baseline to post-baseline.	—

Measure

Time frame

Main Objective: To assess the efficacy of DFV890 to reduce cold-induced inflammation in participants with FCAS.;Secondary Objective: - To assess safety and tolerability of DFV890. - To assess the efficacy of DFV890 to improve the signs and symptoms of FCAS. - To assess the effect of DFV890 on patient reported outcomes.;Primary end point(s): Change from baseline for white cell count (WCC).;Timepoint(s) of evaluation of this end point: Pre-baseline to post-baseline.

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Secondary

Measure	Time frame
Secondary end point(s): - Number and severity of safety assessments and adverse events. - Change from baseline in physician assessed disease scores. - Change from baseline in Patient’s global assessment.;Timepoint(s) of evaluation of this end point: - Number and severity of safety assessments and adverse events: up to End of Study. - Change from baseline in physician assessed disease scores: pre-baseline to post-baseline - Change from baseline in Patient’s global assessment: pre-baseline to post-baseline.	—

Measure

Time frame

Secondary end point(s): - Number and severity of safety assessments and adverse events. - Change from baseline in physician assessed disease scores. - Change from baseline in Patient’s global assessment.;Timepoint(s) of evaluation of this end point: - Number and severity of safety assessments and adverse events: up to End of Study. - Change from baseline in physician assessed disease scores: pre-baseline to post-baseline - Change from baseline in Patient’s global assessment: pre-baseline to post-baseline.

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Countries

France, Germany, Italy, United Kingdom, United States

Contacts

Public ContactMedizinischer Infoservice (MCC)

Novartis Pharma GmbH

infoservice.novartis@novartis.com+4991127312100

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026