A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
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: Vosoritide Injection, Human Growth Hormone, Placebo.
- Кому может быть актуально
- Состояния в реестре: Idiopathic Short Stature. Базовые параметры: 3 лет — 11 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- США, Австралия, Франция, Германия, Италия +1
- 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
A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
Обзор
The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).
Подробное описание
Following a minimum 6 month observational period in which baseline growth is assessed, participants in the vosoritide and placebo groups will complete a minimum of 6 months of randomized treatment (maximum of 6 months of placebo treatment), followed by open-label treatment with vosoritide until they reach near-final adult height, or at least 16 years of age for females or 18 years of age for males, whichever comes later. Participants randomized to the hGH group will receive open-label hGH for a minimum of 4 years.
A Treatment Completion visit will occur approximately 4 weeks after the last administration of investigational product. Participants who discontinue study treatment after ≥13 weeks of exposure may remain in the study and complete follow-up assessments per the protocol schedule (including annual and periodic assessments through End of Study). If a participant discontinues treatment and declines further participation, they will be asked to return for a final Treatment Completion and End of Study visit approximately 4 weeks after the last injection.
Safety monitoring includes regular clinical and imaging assessments, including hip and lower-extremity evaluations, monitoring for hypotension, fractures, and slipped capital femoral epiphysis, with oversight by an independent Data Monitoring Committee
An independent Data Monitoring Committee will periodically review safety data and may recommend treatment discontinuation based on predefined safety signals
Вмешательства
- Препарат Vosoritide Injection
Experimental Drug Lyophilized powder for reconstitution - Препарат Human Growth Hormone
Commercial product containing somatotropin - Препарат Placebo
Lyophilized powder for reconstitution
Первичные конечные точки
- Change from baseline in Annualized Growth Velocity (AGV) [Срок оценки: At 6 months]
- Change from baseline in height [Срок оценки: At 4 years]
- Change from baseline in height Z-score [Срок оценки: At 4 years]
Вторичные конечные точки (12)
- Incidence of treatment-emergent adverse events [Срок оценки: Until the end of the study, up to 15 years]
- Change from baseline in Height Z-score (average stature reference) [Срок оценки: At 6 months]
- Change from baseline in height [Срок оценки: Every 6 months through the end of study, up to 15 years]
- Change from baseline in height Z score [Срок оценки: Every 6 months through the end of study, up to 15 years]
- Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP) [Срок оценки: Every 6 months through the end of study, up to 15 years]
- Change from baseline at pre-specified timepoints in serum collagen X marker (CXM) [Срок оценки: Every 6 months through the end of study, up to 15 years]
- Change from baseline in bone age minus chronological age at pre-specified timepoints [Срок оценки: Every 6 months through the end of study, up to 15 years]
- Change from baseline in whole body (less head) bone mineral density (BMD) Z-score [Срок оценки: Every 6 months through the end of study, up to 15 years]
- Change from baseline in lumbar spine BMD Z-score [Срок оценки: Every 6 months through the end of study, up to 15 years]
- Change from baseline in total body (less head) bone mineral content (BMC) [Срок оценки: Every 6 months through the end of study, up to 15 years]
- Change from baseline in lumbar spine BMC [Срок оценки: Every 6 months through the end of study, up to 15 years]
- Maximum concentration (Cmax) of vosoritide in plasma [Срок оценки: Every 6 months through the end of study, up to 15 years]
Критерии участия
Критерии включения
- Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts
- If participant is ≥ 5 years at Screening,must be Tanner Stage I to be eligible for enrollment and randomization3. Historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (≥ -1.00 SDs and ≤+2.00 SDs).
Key Exclusions:
- Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome), ACAN deficiency.
- Previous treatment with a growth promoting agent
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 · 35 центров
- Consano Clinical Research, LLC — California City
- Rady Children's Hospital - San Diego — California City
- Children's Hospital of Orange County Main Campus - Orange — California City
- Children's Hospital Los Angeles — Los Angeles
- Children's Hospital Oakland Research Institute — Oakland
- Center Of Excellence in Diabetes and Endocrinology — Sacramento
- Lundquist Institute for Biomedical Innovation (LA BioMed) — Torrance
- The Children's National Research Institute — Washington D.C.
- … и ещё 27 центров
Франция · 4 центра
- Centre Hospitalier Universitaire d'Angers — Angers
- Hôpital de la Timone - Enfants — Marseille
- CHU de Toulouse - Hôpital des Enfants — Toulouse
- Hôpital Bicêtre — Le Kremlin-Bicêtre
Италия · 3 центра
- Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer — Florence
- Ospedale Pediatrico Bambino Gesù — Roma
- IRCCS Istituto Giannina Gaslini — Genova
South Korea · 3 центра
- Seoul National University Bundang Hospital — Seongnam-si
- Inha University Hospital — Incheon
- Chungnam National University Sejong Hospital (CNUSH) — Sejong
Австралия · 1 центр
- Murdoch Children's Research Institute — Parkville
Германия · 1 центр
- Universitätsklinikum des Saarlandes — Homburg
Идентификаторы
NCT: NCT06382155 · 111-210