2024-510917-14-00招募中3 期
A Long-Term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 15
- 试验地点
- 6
- 主要终点
- Treatment-emergent AEs and SAEs, frequency, severity, and relationship to investigational product throughout the study
研究概览
简要总结
Evaluate the long-term safety profile of GTX-102 in subjects with AS
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Safety follow-up
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Signed informed consent from parent(s) or legal guardian(s).
- •Prior participation in a clinical trial with GTX-102; including completion of the Loading phase/period, with approval from the Ultragenyx Medical Monitor. The Screening visit must occur within 6 months of the last visit in the prior GTX-102 study.
- •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.
排除标准
- •Discontinued from prior GTX-102 study 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 judgement 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.
结局指标
主要结局
Treatment-emergent AEs and SAEs, frequency, severity, and relationship to investigational product throughout the study
Treatment-emergent AEs and SAEs, frequency, severity, and relationship to investigational product throughout the study
次要结局
- Change from LTE Month 0 and pretreatment (when available) throughout the study in: • Bayley-4 Cognition raw score • Bayley-4 Receptive Communication raw score • Bayley-4 Expressive Communication raw score • Bayley-4 Gross Motor raw score • Bayley-4 Fine Motor raw score
研究者
Medical Information
Scientific
Ultragenyx Pharmaceutical Inc.
研究点 (6)
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