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Само по покана Фаза 3 NCT06415344

Long-term Extension of GTX-102 in Angelman Syndrome

Фаза 3 – широко изпитване преди одобрение
Заболявания: Angelman Syndrome

Спонсор: Ultragenyx Pharmaceutical Inc

Налично на: БГ
Обобщение
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Описание
This study is a phase 3, long term extension (LTE) which is open to participants rolling over from a prior GTX-102 clinical study. The LTE study will evaluate the long-term safety and efficacy of GTX-102 in participants with AS.
Кой може да участва
Inclusion Criteria: * Signed informed consent from parent(s) or legal guardian(s). * Completed a final study visit in a prior clinical trial with GTX-102. If the subject has an ongoing adverse event, medical monitor approval is required to rollover. The Screening visit must occur within 6 months of the last visit in the prior GTX-102 trial. * From the time of informed consent through the end of the study and for at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102. Exclusion Criteria: * Discontinued from prior GTX-102 trial due to treatment-emergent adverse event assessed as related to GTX-102 and where the risk of study participation outweighs the benefits as deemed by the clinical judgment of the Investigator. * History or evidence of any other medical, neurological, or psychological condition that would expose the subject to an undue risk of a significant AE or interfere with study assessments during the course of the trial as determined by the clinical judgment of the Investigator. * Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects. * Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
Места на провеждане 8
Австралия (1)
Queensland Children's Hospital
South Brisbane , Queensland
Канада (1)
MAGIC Clinic Ltd
Calgary , Alberta
Франция (1)
AP-HM - Hopital de la Timone
Marseille
Германия (1)
Universitaetsklinikum Hamburg-Eppendorf
Hamburg
Израел (1)
Sheba Medical Center
Ramat Gan
Испания (1)
Hospital Universitario Puerta de Hierro
Majadahonda
Великобритания (1)
University of Cambridge
Cambridge
САЩ (1)
University of California, Los Angeles (UCLA)
Los Angeles , California
Технически детайли
Статус
Само по покана
Фаза
Фаза 3
Вид изследване
INTERVENTIONAL
Пол
Мъже и жени
Здрави доброволци
Не
Начална дата
31.07.2024
Крайна дата
01.02.2029
Регистрационен номер
NCT06415344
Източник
anzctr
Запитване за медицински туризъм

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