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)

The purpose of this phase II study was to assess the safety, tolerability and efficacy of DFV890 in participants with FCAS.

This was an open-label, single-arm, multiple dose, phase II study to assess safety, tolerability and clinical efficacy of DFV890 in participants with FCAS who showed evidence of inflammatory activity after the cold challenge performed during screening.

The study included a screening period, a treatment period and a follow-up period.

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.