A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- В протоколе указаны: GNR-055 1.0-2.0-3.0 mg/kg, GNR-055 2.0 mg/kg, GNR-055 3.0 mg/kg.
- Кому может быть актуально
- Состояния в реестре: Mucopolysaccharidosis Type II, Metabolic Diseases. Базовые параметры: Без ограничений · Мужчины.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- Россия
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
Multicenter, Open-Label, Multi-cohort Study to Evaluate Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Drug Product GNR 055 (JSC "GENERIUM", Russia) in Patients With Mucopolysaccharidosis Type II
Обзор
This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age groups.
Подробное описание
GNR-055 is intended for ERT in patient with Mucopolysaccharidosis type II (MPS II), or Hunter syndrome. MPS II is a recessive X-linked inheritance lysosomal storage disease, which is characterized by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (ID2S), caused by a mutation in the ID2S gene. Enzyme deficiency leads to the accumulation of Glycosaminoglycans (GAG) (mainly of heparan and dermatan sulfates) in lysosomes of almost all types of cells of various tissues and organs. The disease is manifested by growth retardation, damage of many organs and systems, severe deformations of bones and joints, gross facial features, pathology of the respiratory and cardiovascular systems, damage to parenchymal organs (hepatosplenomegaly), and hearing impairment. A severe form of the disease occurs with the involvement of the nervous system in the pathological process, including mental retardation, behavior anomalies, and impaired motor function.
GNR-055 is a recombinant modified ID2S capable to penetrate the blood-brain barrier and thus expected to prevent neurodegenerative consequences and the cognitive deficit and to attain a significant improvement in the life quality and expectancy of patients with MPS II.
Study IDB-MPS-II-III is a multicenter, open-label, multi-cohort study to assess safety, PK and PD, and efficacy of GNR-055 in patients of different age groups with MPS II (Hunter syndrome).
Вмешательства
- Препарат GNR-055 1.0-2.0-3.0 mg/kg
Weekly IV infusion (lyophilized powder) 1.0-2.0-3.0 mg/kg - Препарат GNR-055 2.0 mg/kg
Weekly IV infusion (lyophilized powder) 2.0 mg/kg - Препарат GNR-055 3.0 mg/kg
Weekly IV infusion (lyophilized powder) 3.0 mg/kg
Первичные конечные точки
- Incidence of Adverse events (AEs) and Serious Adverse Events (SAEs) [Срок оценки: Baseline to Week 56]
- Urine GAG excretion [Срок оценки: Baseline to Week 4, 8, 10, 26, and 52]
Вторичные конечные точки (12)
- Serum concentration of the GNR-055 [Срок оценки: Week 52]
- GAG level in CerebroSpinal Fluid (CSF) [Срок оценки: Baseline to Week 6, 10, 26, and 52]
- Serum GAG level [Срок оценки: Baseline to Week 4, 8, 10, 26, and 52]
- Large joint range of motion [Срок оценки: Week 8, 10, 26, and 52]
- Liver and spleen volumes (MRI) [Срок оценки: Baseline to Week 8, 10, 26, and 52]
- 6-minute walk test [Срок оценки: Baseline to Week 8, 10, 26, and 52]
- Left ventricular mass by EchoCG [Срок оценки: Baseline to Week 8, 10, 26, and 52]
- Lung Forced Vital Capacity (FVC) [Срок оценки: Baseline to Week 8, Week 26, and Week 52]
- Neurocognitive functions assessment [Срок оценки: Baseline to Week 12, 26, and 52]
- Brain white/gray matter structures (MRI) [Срок оценки: Baseline to Week 26, and 52]
- Serum neuromarkers [Срок оценки: Baseline to Week 24, and 52]
- CSF neuromarkers [Срок оценки: Baseline to Week 24, and 52]
Критерии участия
Критерии включения
- Signed inform consent;
- Verified diagnosis of MPS II (Hunter syndrome);
- Naïve patients or patients who have received standard ERT whit idursulfase products;
- No contraindications for lumbar puncture as judged by the Investigator;
- Willingness and ability to follow study procedures.
Критерии исключения
- Clinically pronounced hypersensitivity to ID2S or any other component of the drug product;
- History of hematopoietic stem cell transplantation (HSCT) or bone marrow transplantation;
- Implanted or external non-removable metal devices, a cardiac pacemaker, or other objects sensitive to the magnetic field that may pose a danger to both the wearer and the correct operation of magnetic resonance imaging (MRI) equipment;
- Concomitant diseases and conditions that, in the Investigator's opinion, can put at risk the patient's safety during his/her participation in the study, or which will influence the safety data analysis in case of the disease/condition exacerbation during the study.
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
Россия · 5 центров
- Federal State-Funded Healthcare Institution Central Clinical Hospital of the Russian Acade — Moscow
- Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State — Saint Petersburg
- V.I. Vernadsky Crimean Federal University — Simferopol
- State Budgetary Healthcare Institution Republican Medical Genetic Center — Ufa
- State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clini — Yekaterinburg
Идентификаторы
NCT: NCT05208281 · IDB-MPS-II-III