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

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)

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).

Study Overview

• Status: Completed
• Conditions: Familial Cold Autoinflammatory Syndrome
• Intervention / Treatment: Drug: DFV890

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 2

Contacts and Locations

Study Locations

• France
  • Paris, France, 75020
    • Novartis Investigative Site
• Germany
  • Tuebingen, Germany, 72076
    • Novartis Investigative Site
• United States
  • California
    • La Jolla, California, United States, 92093
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 78 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: DFV890	Drug: DFV890; DFV890

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ratio of fold change from baseline for white cell count (WCC)	To assess the efficacy of DFV890 in patients with FCAS	At pre-baseline and up to 24 hours post-baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number and severity of safety assessments and adverse events	To evaluate the safety and tolerability of DFV890 in patients with FCAS	Through study completion, approximately 4 months
Change from pre-baseline to post-baseline in Physician global assessment of autoinflammatory disease activity	The Physician global assessment of autoinflammatory disease activity is a questionnaire to be completed by the Investigator. It uses a 5- point rating scale: absent, minimal, mild, moderate, and severe. Lower scores represent better outcomes.	At pre-baseline and up to 24 hours post-baseline
Change from pre-baseline to post-baseline in Physician's severity assessment of autoinflammatory disease signs and symptoms	The Physician's severity assessment of autoinflammatory disease signs and symptoms is a questionnaire to be completed by the Investigator. It uses a 5-point rating scale: absent, minimal, mild, moderate, and severe. Lower scores represent better outcomes.	At pre-baseline and up to 24 hours post-baseline
Change from pre-baseline to post-baseline in Patient's global assessment of disease activity	Patient's global assessment of disease activity is a questionnaire to be completed by the patient. It uses a 5-point rating scale: absent, minimal, mild, moderate, and severe. The patient will select a rating based on the patient's current disease activity at the time of the assessment. Lower scores represent better outcomes.	At pre-baseline and up to 24 hours post-baseline

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): September 20, 2021
• Primary Completion (Actual): December 13, 2022
• Study Completion (Actual): May 5, 2023

Study Registration Dates

• First Submitted: April 22, 2021
• First Submitted That Met QC Criteria: April 28, 2021
• First Posted (Actual): May 3, 2021

Study Record Updates

• Last Update Posted (Actual): May 23, 2023
• Last Update Submitted That Met QC Criteria: May 18, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: CAPS; DFV890; Familial Cold Auto-inflammatory Syndrome; FCAS; NLRP3 inhibitor, inflammasome inhibition; Cryopyrin-associated periodic syndromes
• Additional Relevant MeSH Terms: Pathologic Processes; Skin Diseases; Immune System Diseases; Hypersensitivity, Immediate; Disease Attributes; Disease; Genetic Diseases, Inborn; Hereditary Autoinflammatory Diseases; Skin Diseases, Genetic; Skin Diseases, Vascular; Hypersensitivity; Urticaria; Chronic Disease; Chronic Inducible Urticaria; Chronic Urticaria; Syndrome; Cryopyrin-Associated Periodic Syndromes
• Other Study ID Numbers: CDFV890A12201; 2020-005948-33 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No