All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.
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).
Familial Cold Autoinflammatory Syndrome
Sep 20, 2021
Mar 19, 2023
18 Years - 80 Years (Adult, Older Adult)
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
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