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

• Register: EUCTR
• Last refreshed on: 22 April 2024
• Main ID: EUCTR2020-005948-33-DE
• Date of registration: 26/02/2021
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma AG
• Public title: Study of safety, tolerability and efficacy of DFV890 in participants with familial cold auto-inflammatory syndrome (FCAS)
• Scientific 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)
• Date of first enrolment: 26/07/2021
• Target sample size: 6
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-005948-33
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other:
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

France, Germany, Italy, United Kingdom, United States

Contacts

• Name: Medizinischer Infoservice (MCC)
• Affiliation: Novartis Pharma GmbH

• Name: Medizinischer Infoservice (MCC)
• Affiliation: Novartis Pharma GmbH

Key inclusion & exclusion 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.
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:
- 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-?
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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Familial Cold Autoinflammatory Syndrome (FCAS)
MedDRA version: 25.1 Level: PT Classification code 10068850 Term: Cryopyrin associated periodic syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Product Name: DFV890
Product Code: DFV890
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Not yet established
Current Sponsor code: DFV890
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 25-

Primary Outcome(s)

Secondary Objective: - To assess safety and tolerability of DFV890.
- To assess the efficacy of DFV890 to improve the signs and symptoms of FCAS.
- To assess the effect of DFV890 on patient reported outcomes.

Main Objective: To assess the efficacy of DFV890 to reduce cold-induced inflammation in participants with FCAS.

Timepoint(s) of evaluation of this end point: Pre-baseline to post-baseline.

Primary end point(s): Change from baseline for white cell count (WCC).

Secondary Outcome(s)

Secondary end point(s): - Number and severity of safety assessments and adverse events.
- Change from baseline in physician assessed disease scores.
- 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.
- Change from baseline in physician assessed disease scores: pre-baseline to post-baseline
- Change from baseline in Patient’s global assessment: pre-baseline to post-baseline.

Secondary ID(s)

CDFV890A12201

Source(s) of Monetary Support

Novartis Pharma AG

Ethics review

Status: Approved
Approval date: 26/07/2021
Contact:

Results

• Results available: Yes
• Date Posted: 27/03/2024
• Date Completed: 05/05/2023
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2020-005948-33/results