Phase 2
N=4
Study of Safety, Tolerability and Efficacy of DFV890 in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)
Familial Cold Autoinflammatory Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT04868968 ↗Enrolled (actual)
4
Serious AEs
0.0%
Results posted
May 2024
Primary outcome: Primary: Ratio of Fold Change From Pre-challenge to the Highest Post-challenge Value of White Cell Count (WCC) Between Treatment and Screening Period — 0.82 ratio of fold change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- DFV890 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Dec 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Ratio of Fold Change From Pre-challenge to the Highest Post-challenge Value of White Cell Count (WCC) Between Treatment and Screening Period |
0.82 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
4; 0; 0; 0 | — |
| SECONDARY Physician Global Assessment of Autoinflammatory Disease Activity |
0; 2; 3; 2; 1; 0 | — |
| SECONDARY Physician's Severity Assessment of Autoinflammatory Disease Signs and Symptoms |
3; 3; 1; 1; 0; 0 | — |
| SECONDARY Patient's Global Assessment of Disease Activity |
2; 3; 2; 1; 0; 0 | — |
Summary
The purpose of this phase II study was to assess the safety, tolerability and efficacy of DFV890 in participants with FCAS.
Eligibility 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
- 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.
Data sourced from ClinicalTrials.gov (NCT04868968). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.