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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-09-20

Study Completion Date

2023-05-05

Brief Summary

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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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Detailed Description

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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.

Conditions

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Familial Cold Autoinflammatory Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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DFV890

Group Type EXPERIMENTAL

DFV890

Intervention Type 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.

Interventions

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DFV890

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.

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No