Clinical trial NCT04868968: Study of Safety, Tolerability and Efficacy of DFV890 in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)
Official title: An Open-label, Single Arm Phase II Study of DFV890 to Assess the Safety, Tolerability and Efficacy in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Familial Cold Autoinflammatory Syndrome
Keywords: Familial Cold Auto-inflammatory Syndrome
FCAS
DFV890
NLRP3 inhibitor, inflammasome inhibition
Cryopyrin-associated periodic syndromes
CAPS
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2021-05-03
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Detailed 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.

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.

Primary outcomes:
measure: Ratio of Fold Change From Pre-challenge to the Highest Post-challenge Value of White Cell Count (WCC) Between Treatment and Screening Period
timeFrame: Screening period and treatment period (Day 4): pre cold challenge and up to 8 hours post cold challenge. The duration of the cold challenge was 45 minutes.
Secondary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 34 days
measure: Physician Global Assessment of Autoinflammatory Disease Activity
timeFrame: Screening and Treatment (Day 4): 1 hour pre and 2, 3, 5, 9 and 24 hours post. Scheduled time refers to the time post-meal (screening) and to the time post-dose (treatment). The start of the cold challenge is at 1 hour post and the duration is 45 minutes.
measure: Physician's Severity Assessment of Autoinflammatory Disease Signs and Symptoms
timeFrame: Screening and Treatment (Day 4): 1 hour pre and 2, 3, 5, 9 and 24 hours post. Scheduled time refers to the time post-meal (screening) and to the time post-dose (treatment). The start of the cold challenge is at 1 hour post and the duration is 45 minutes.
measure: Patient's Global Assessment of Disease Activity
timeFrame: Screening and Treatment (Day 4): 1 hour pre and 2, 3, 5, 9 and 24 hours post. Scheduled time refers to the time post-meal (screening) and to the time post-dose (treatment). The start of the cold challenge is at 1 hour post and the duration is 45 minutes.
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: Novartis Investigative Site
state: California
city: Paris
country: France
facility: Novartis Investigative Site
city: Tuebingen
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: