Back to resultsStudy of Safety, Tolerability and Efficacy of DFV890 in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS) (DFV890-FCAS)
Primary Purpose
Familial Cold Autoinflammatory Syndrome
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
DFV890
Sponsored by
About this trial
This is an interventional treatment trial for Familial Cold Autoinflammatory Syndrome focused on measuring Familial Cold Auto-inflammatory Syndrome, FCAS, DFV890, NLRP3 inhibitor, inflammasome inhibition, Cryopyrin-associated periodic syndromes, CAPS
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
Sites / Locations
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
DFV890
Arm Description
DFV890
Outcomes
Primary Outcome Measures
Ratio of fold change from baseline for white cell count (WCC)
To assess the efficacy of DFV890 in patients with FCAS
Secondary Outcome Measures
Number and severity of safety assessments and adverse events
To evaluate the safety and tolerability of DFV890 in patients with FCAS
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.
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04868968
Brief Title
Study of Safety, Tolerability and Efficacy of DFV890 in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)
Acronym
DFV890-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
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Completed
Study Start Date
September 20, 2021 (Actual)
Primary Completion Date
December 13, 2022 (Actual)
Study Completion Date
May 5, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Cold Autoinflammatory Syndrome
Keywords
Familial Cold Auto-inflammatory Syndrome, FCAS, DFV890, NLRP3 inhibitor, inflammasome inhibition, Cryopyrin-associated periodic syndromes, CAPS
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
DFV890
Arm Type
Experimental
Arm Description
DFV890
Intervention Type
Drug
Intervention Name(s)
DFV890
Intervention Description
DFV890
Primary Outcome Measure Information:
Title
Ratio of fold change from baseline for white cell count (WCC)
Description
To assess the efficacy of DFV890 in patients with FCAS
Time Frame
At pre-baseline and up to 24 hours post-baseline
Secondary Outcome Measure Information:
Title
Number and severity of safety assessments and adverse events
Description
To evaluate the safety and tolerability of DFV890 in patients with FCAS
Time Frame
Through study completion, approximately 4 months
Title
Change from pre-baseline to post-baseline in Physician global assessment of autoinflammatory disease activity
Description
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.
Time Frame
At pre-baseline and up to 24 hours post-baseline
Title
Change from pre-baseline to post-baseline in Physician's severity assessment of autoinflammatory disease signs and symptoms
Description
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.
Time Frame
At pre-baseline and up to 24 hours post-baseline
Title
Change from pre-baseline to post-baseline in Patient's global assessment of disease activity
Description
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.
Time Frame
At pre-baseline and up to 24 hours post-baseline
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
La Jolla
State/Province
California
ZIP/Postal Code
92093
Country
United States
Facility Name
Novartis Investigative Site
City
Paris
ZIP/Postal Code
75020
Country
France
Facility Name
Novartis Investigative Site
City
Tuebingen
ZIP/Postal Code
72076
Country
Germany
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing 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
Learn more about this trial
Study of Safety, Tolerability and Efficacy of DFV890 in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)
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