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Completed

NCT Number: NCT04868968

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

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

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Key information

Conditions

Familial Cold Autoinflammatory Syndrome Chronic Disease Chronic Inducible Urticaria Chronic Urticaria Cold Urticaria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cryopyrin-Associated Periodic Syndromes Disease Attributes Genetic Diseases, Inborn Hereditary Autoinflammatory Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Skin Diseases Skin Diseases, Genetic Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Novartis Investigative Site, Paris, France

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

DFV890

Drug

100 mg of DFV890 film coated tablets twice daily (b.i.d.). for 3 days starting in the morning of Day 1 and 100 mg of DFV890 in the morning on the fourth day.

Primary outcomes

  1. Ratio of Fold Change From Pre-challenge to the Highest Post-challenge Value of White Cell Count (WCC) Between Treatment and Screening Period

    Time frame: 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.

    A cold challenge was performed during the screening period and on Day 4 of the treatment period. Fold change from pre-challenge to highest post-challenge value of WCC was defined as the ratio of the highest post-challenge WCC value to the pre-challenge WCC value. The ratio of fold change was defined as treatment fold change divided by the screen fold change. A value of less than 1 for the ratio of fold change indicates a lower relative increase of WCC in the treatment than in the screening period, which is a favorable outcome. The log-transformed fold change from pre-challenge to the highest post challenge WCC was analyzed using a log-linear mixed effect model. The analysis was carried out considering the data from -2 to 8 hrs post challenge. The unforeseen screen failure rate and recruitment challenges resulted in early closure of the study. Only 4 out of planned 6 participants were enrolled in the study; thus, the results should be interpreted cautiously.

Secondary outcomes

  1. Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: 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

    Number of participants with treatment emergent AEs (any AE regardless of seriousness), AEs led to study treatment discontinuation, SAEs and SAEs led to study treatment discontinuation.

  2. Physician Global Assessment of Autoinflammatory Disease Activity

    Time frame: 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.

    The Physician global assessment of autoinflammatory disease activity is a questionnaire completed by the Investigator. It uses a 5-point scale. Lower scores represent better outcomes.

    0 = Absent

    • = Minimal
    • = Mild
    • = Moderate
    • = Severe

  3. Physician's Severity Assessment of Autoinflammatory Disease Signs and Symptoms

    Time frame: 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.

    The Physician's severity assessment of autoinflammatory disease signs and symptoms is a questionnaire completed by the Investigator. It uses a 5-point scale. Lower scores represent better outcomes.

    0 = Absent

    • = Minimal
    • = Mild
    • = Moderate
    • = Severe

    The following items were assessed:

    • Assessment of skin disease (urticarial skin rash)
    • Assessment of arthralgia
    • Assessment of myalgia
    • Assessment of headache/migraine
    • Assessment of conjunctivitis
    • Assessment of fatigue/malaise

  4. Patient's Global Assessment of Disease Activity

    Time frame: 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.

    Patient's global assessment of disease activity is a questionnaire completed by the patient. It uses a 5-point scale. The patient selected a rating based on the patient's current disease activity at the time of the assessment. Lower scores represent better outcomes.

    0 = Absent

    • = Minimal
    • = Mild
    • = Moderate
    • = Severe

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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)

Acronym: DFV890-FCAS

Important dates

Study start
2021
Primary completion
2022
Study completion
2023
First posted
May 3, 2021
Registry last updated
Oct 9, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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