A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 2a Study With an Open-Label Extension Evaluating the Efficacy and Safety of VENT-03 in Adult Participants With Active Cutaneous Lupus Erythematosus With or Without Systemic Lupus Erythematosus

Обзор

The goal of this clinical trial is to learn if VENT-03 works to treat patients with cutaneous lupus erythematosus (CLE) who may or may not have systemic lupus erythematosus (SLE). Another goal is to learn about the safety of VENT-03 and how it is processed by the body. The main questions it aims to answer are: * Does VENT-03 affect the activity and severity of CLE? * What side effects do participants have when taking VENT-03? Researchers will compare VENT-03 to a placebo (a look-alike substance that contains no drug) to see if VENT-03 works to treat patients with CLE. Participants will: * Take VENT-03 or a placebo for 4 weeks, then all participants will switch to VENT-03 for another 8 weeks; * Visit the clinic once a month for checkups and tests.

Вмешательства

• Препарат VENT-03
  VENT-03 is a tablet
• Препарат Placebo
  Placebo is a tablet

Первичные конечные точки

• Evaluate the effect of VENT-03 on the interferon gene signature in the skin [Срок оценки: Baseline to End of Double-Blind Treatment (up to Day 28)]

Вторичные конечные точки (8)

• Evaluate the effect of VENT-03 on CLE disease severity [Срок оценки: Baseline to End of Double-Blind Treatment (up to Day 28)]
• Evaluate effect of VENT-03 on CLE disease severity [Срок оценки: Baseline to End of Double-Blind Treatment (up to Day 28)]
• Change from Baseline in Myxovirus-Resistant Protein A (MXA) Immunostaining in Skin Biopsy [Срок оценки: Baseline to End of Treatment (up to Day 84)]
• Number of participants with at least one Treatment Emergent Adverse Event (TEAE) and/or Serious Adverse Event (SAE) [Срок оценки: Baseline to End of Treatment (up to Day 84)]
• Number of participants with Moderate or Severe Treatment Emergent Adverse Events (TEAEs) [Срок оценки: Baseline to End of Treatment (up to Day 84)]
• Percentage of Participants with ≥ 1 Treatment Emergent Adverse Event (AE) leading to Treatment Discontinuation [Срок оценки: Baseline to End of Treatment (up to Day 84)]
• Cmax: Maximum Observed Plasma Concentration for VENT-03 [Срок оценки: Day 1 pre-dose and at multiple time points (up to 6 hours) post-dose; Day 28 pre-dose and post-dose; Day 56 and Day 84 pre-dose]
• AUClast: Area Under the Plasma Concentration-Time Curve from Time 0 to the Time of the Last Quantifiable Concentration for VENT-03 [Срок оценки: Day 1 pre-dose and at multiple time points (up to 6 hours) post-dose; Day 28 pre-dose and post-dose; Day 56 and Day 84 pre-dose]

Критерии участия

Критерии включения

• Cutaneous lupus:

• CLASI-A score ≥8;
• At least 1 active discoid lupus erythematosus (DLE) lesion, OR at least 1 active subacute CLE lesion
• If participant has previous SLE diagnosis:

• Positive antinuclear antibody test at Screening by immunofluorescent assay at the central laboratory with titer ≥ 1:80;
• Meets the American College of Rheumatology/ European Alliance of Associations for Rheumatology 2019 criteria for SLE; and
• Currently receiving at least one of the specified SLE medication treatments, at stable doses.

Критерии исключения

• Meet protocol-specified infection or lab criteria; any other laboratory test results that, in the investigator's opinion, might place participant at unacceptable risk for participating in this study;
• Moderate or severe liver impairment as classified by the Child-Pugh criteria (categories B and C);
• Has drug-induced lupus, rather than 'idiopathic' lupus;
• History of, or current, inflammatory joint or skin disease other than SLE and cutaneous lupus;
• Diagnosis of select potentially confounding autoimmune disorders
• Active severe or unstable neuropsychiatric SLE;
• Hospitalization for a severe lupus flare in the past 3 months, or active severe SLE-driven disease, including lupus nephritis, for which in the opinion of the PI the protocol-specified SOC is insufficient;
• History of or current diagnosis of anti-phospholipid syndrome;
• History of any non-lupus disease that has required treatment with oral or parenteral corticosteroids for more than a total of 2 weeks within the last 24 weeks prior to Day 1;
• Meets protocol specified medical history of infectious diseases and infections and/or opportunistic infection requiring hospitalization or parenteral antimicrobial treatment within specified timeframes;
• Cancer screening results suspicious of malignancy or history of cancer within time specified with exceptions for curative therapy for squamous or basil cell carcinoma and cervical cancer in situ; and
• Meets protocol specified exclusions related to concomitant medications.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 13 центров

• Investigative Site — Beverly Hills
• Investigative Site — Clearwater
• Investigative Site — DeBary
• Investigative Site — Tampa
• Investigative Site — Indianapolis
• Investigative Site — Flint
• Investigative Site — Troy
• Investigative Site — Saint Joseph
• … и ещё 5 центров

Польша · 7 центров

• Investigative Site — Bialystok
• Investigative Site — Oświęcim
• Investigative Site — Poznan
• Investigative Site — Rzeszów
• Investigative Site — Śląskie
• Investigative Site — Warsaw
• Investigative Site — Wroclaw

Болгария · 3 центра

• Investigative Site — Haskovo
• Investigative Site — Plovdiv
• Investigative Site — Sofia

ЮАР · 3 центра

• Investigative Site — Durban
• Investigative Site — Pretoria
• Investigative Site — Somerset West

Франция · 2 центра

• Investigative Site — Paris
• Investigative Site — Toulouse

Испания · 2 центра

• Investigative Site — Badajoz
• Investigative Site — Madrid

Грузия · 1 центр

• Investigative Site — Tbilisi

Венгрия · 1 центр

• Investigative Site — Szeged

Идентификаторы

NCT: NCT07260877 · VENT-03-201 · 2024-520098-12-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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