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

Novartis

Status and phase

Completed

Phase 2

Conditions

Familial Cold Autoinflammatory Syndrome

Treatments

Drug: DFV890

Study type

Interventional

Funder types

Industry

Identifiers

NCT04868968

CDFV890A12201

2020-005948-33 (EudraCT Number)

Details and patient eligibility

About

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

Full description

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.

Enrollment

4 patients

Sex

All

Ages

18 to 80 years old

Volunteers

No Healthy Volunteers

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.