Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of JTE-162 in Subjects With Cryopyrin-Associated Periodic Syndrome (CAPS)

Официальное название

A Phase 1b, Open-label, Single-arm Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of JTE-162 in Subjects With Cryopyrin-Associated Periodic Syndrome (CAPS)

Обзор

This study will evaluate the efficacy, safety, tolerability and pharmacokinetics of JTE-162 administered once daily for 2 weeks in subjects with cryopyrin-associated periodic syndrome (CAPS)

Вмешательства

• Препарат JTE-162
  Tablets containing JTE-162

Первичные конечные точки

• Change and percent change from baseline to end of treatment (EOT) in high-sensitivity C-reactive protein (hs-CRP) [Срок оценки: 2 Weeks]
• Change and percent change from baseline to EOT in Serum amyloid A (SAA) [Срок оценки: 2 Weeks]
• Change and percent change from baseline to EOT in Interleukin (IL)-6 [Срок оценки: 2 Weeks]
• Change and percent change from baseline to EOT in Key symptom score (KSS) [Срок оценки: 2 Weeks]
• Change and percent change from baseline to EOT in Individual symptom score on Daily Health Assessment Form, Second Generation (DHAF2) [Срок оценки: 2 Weeks]
• Change and percent change from baseline to EOT in Global assessments of disease activity on DHAF2 [Срок оценки: 2 Weeks]
• Change and percent change from baseline to EOT in Global CAPS symptom assessment by the Investigator [Срок оценки: 2 Weeks]
• Change and percent change from baseline to EOT in Individual CAPS symptom assessment by the Investigator [Срок оценки: 2 Weeks]
• Number of adverse events [Срок оценки: 4 Weeks]
• JTE-162 post-dose plasma concentrations on Day 1 [Срок оценки: 1 Day]

Критерии участия

Критерии включения

• Diagnosed with familial cold autoinflammatory syndrome (FCAS) or Muckle-Wells syndrome (MWS) confirmed by:
• Clinical History: At least 2 typical clinical symptoms (e.g., urticarial skin rash, myalgia, arthralgia, recurrent fever, fatigue/malaise, conjunctivitis or other autoinflammatory symptoms) prior to the Screening Visit; AND
• Genetic Confirmation: Confirmed nucleotide-binding and oligomerization domain (NOD)-like receptor family pyrin domain containing 3 (NLRP3) mutation;
• Willing to discontinue current anti-interleukin (IL)-1 treatment, if applicable;
• Demonstrates de novo flaring of CAPS during the Screening Period.

Критерии исключения

• Has chronic infantile neurologic cutaneous articular syndrome (CINCA)/neonatal-onset multisystem inflammatory disease (NOMID);
• Has a history or presence of amyloidosis, progressive hearing loss, organ damage or any symptom contraindicating anti-IL-1 treatment washout;
• Has active systemic bacterial, fungal or viral infection(s) within 14 days prior to Day 1 or a history of clinically significant recurrent infectious diseases

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Канада · 1 центр

• Gordon Sussman Clinical Research Inc. — North York

Идентификаторы

NCT: NCT07247266 · AE162-X-24-003

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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