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

Phase 1

• Conditions: Familial Cold Autoinflammatory Syndrome (FCAS).; MedDRA version: 20.0Level: PTClassification code 10068850Term: Cryopyrin associated periodic syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2020-005948-33-IT
• Lead Sponsor: OVARTIS PHARMA AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-08
• Last Update Posted: 11 September 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 6

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

- 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-¿ 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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;<br>- To assess the efficacy of DFV890 to improve the signs and symptoms of FCAS;<br>- 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.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Number and severity of safety assessments and adverse events.<br>- Change from baseline in physician assessed disease scores.<br>- 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.<br>- Change from baseline in physician assessed disease scores: prebaseline to post-baseline<br>- Change from baseline in Patient's global assessment: pre-baseline to post-baseline.