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

• Status: Recruiting
• Trial end date: 2022-02-23
• Target enrollment: 0
• Participant gender: All

Summary

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 approximately 4 months.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals

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

Exclusion Criteria:

• Participants currently being treated with anti-rejection and/or immunomodulatory drugs and the treatment cannot be discontinued or switched to a different medication

• Clinically significant, suspected active or chronic bacterial (including Mycobacterium tuberculosis), viral or fungal infection within 30 days prior to dosing.

• 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 dosing

• 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