A Study of IN026 in Participants With Refractory Gout

Официальное название

A Clinical Study on the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of IN026 in the Treatment of Refractory Gout

Обзор

The goal of this clinical study is to learn if IN026 Injection is safe and works to lower uric acid levels in adults with refractory gout (gout that does not respond well to standard treatments). The main questions it aims to answer are: * What medical problems do participants have when taking IN026, such as changes in vital signs, blood tests, or heart rhythm? * How does the body absorb, process, and respond to IN026, and does it trigger an immune reaction? * Does IN026 lower uric acid levels in the blood and reduce tophi? Investigator will start with lower doses of IN026 and slowly increase the dose to find the well-tolerated dose. Participants will: * Receive IN026 through an intravenous (IV) drip into a vein at a set dose. * Complete a screening period of up to 4 weeks, followed by treatment and check-ups for up to 20 weeks. * Have blood and urine samples taken at set times to check safety and how the body responds to IN026.

Вмешательства

• Препарат IN026 Injection
  IN026 Injection is a lipid nanoparticle (LNP)-formulated mRNA therapeutic administered as an intravenous (IV) infusion

Первичные конечные точки

• Incidence of Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), and Serious Adverse Events (SAEs) [Срок оценки: From first dose (Week 1 Day 1) through end of study (Week 21)]

Вторичные конечные точки (8)

• Change from Baseline in Serum Uric Acid Concentration [Срок оценки: From baseline (Week 1 Day 1) through Week 21]
• Change in Tophi from Baseline [Срок оценки: From baseline (Week 1 Day 1) through Week 21]
• Plasma Concentration of IN026 mRNA Over Time [Срок оценки: At pre-specified timepoints from first dose (Week 1 Day 1) through Week 21]
• Plasma Concentration of Ionizable Lipid SX-66 Over Time [Срок оценки: At pre-specified timepoints from first dose (Week 1 Day 1) through Week 21]
• Serum Uricase Level Over Time [Срок оценки: At pre-specified timepoints from first dose (Week 1 Day 1) through Week 21]
• Serum Uric Acid Level Over Time [Срок оценки: At pre-specified timepoints from first dose (Week 1 Day 1) through Week 21]
• Serum Allantoin Level Over Time [Срок оценки: At pre-specified timepoints from first dose (Week 1 Day 1) through Week 21]
• Titer of Anti-Drug Antibodies (ADAs) Over Time [Срок оценки: At pre-specified timepoints from first dose (Week 1 Day 1) through Week 21]

Критерии участия

Критерии включения

• Can voluntarily sign the informed consent form (ICF) and comply with ICF and study protocol requirements.
• Male or female, 18-75 years old (inclusive) at screening.
• Meet 2015 ACR/EULAR gout classification criteria, in the intercritical phase of gout or acute flare resolved ≥2 weeks at screening.
• Serum uric acid ≥420 μmol/L (7 mg/dl) at screening.
• Meet the definition of refractory gout (poor uric acid control accompanied by severe gout symptoms)

Критерии исключения

• Gout secondary to radiotherapy/chemotherapy, lead poisoning, organ transplantation, tumor, etc. at screening/baseline.
• Rheumatoid arthritis, infectious/septic arthritis, or other acute inflammatory arthritis at screening/baseline.
• Glucose-6-phosphate dehydrogenase (G6PD) deficiency history, or G6PD level below normal lower limit.
• Positive HBsAg; HCV antibody positive is excluded except those with sustained HCV-RNA negativity after standard treatment; HIV antibody positive; active syphilis.
• Presence of chronic liver diseases including active hepatitis, cirrhosis and alcoholic liver disease.
• Participants with a history of any of the following: serious cardiovascular diseases within 6 months prior to screening; or serious diseases of the digestive, respiratory, urinary, musculoskeletal, neuropsychiatric, hematological, or immune systems within 3 months prior to screening.
• Prolonged QTcF at screening.
• Uncontrolled or untreated hypertension at screening.
• Participants who have received medications that may affect endpoint assessment, such as other urate-lowering therapies, mRNA-LNP vaccine, PEGylated drugs and uricase agents.
• History of severe allergy, or known allergy to IN026 or its components.
• Participation in other clinical trials within 30 days prior to screening.
• Participants with poor compliance, or those deemed otherwise unsuitable for this study by the investigator.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology — Ухань

Идентификаторы

NCT: NCT07587684 · IN026-001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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