NCT06415344进行中（未招募）3 期
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 255
- 试验地点
- 37
- 主要终点
- Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent from parent(s) or legal guardian(s).
- •Completed a final study visit in a prior clinical trial with GTX-
- •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.
排除标准
- •Discontinued from study or withdrew from study treatment in a prior GTX-102 trial.
- •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
研究组 & 干预措施
GTX-102
Experimental
Participants will receive GTX-102 via intrathecal lumbar puncture (IT LP) on a intrapatient flexible dosing schedule.
干预措施: GTX-102 (Drug)
结局指标
主要结局
Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: 5 Years
Incidence of Treatment Emergent Adverse Events (TEAEs)
时间窗: Up to 5 Years
次要结局
- Change from LTE Month 0 and Pretreatment in the Bayley-4 Raw Score(Month 0, 5 Years)
- Change from Baseline in the Bayley-4 Raw Score(Baseline, Up to 5 Years)
- Net Response in Multidomain Responder Index (MDRI)(Month 12, Month 24)
研究者
研究点 (37)
Loading locations...
相似试验
Enrolling By Invitation
2 期
ORACLE: A Long-term Follow-up Study to Evaluate the Safety of GT005 in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration Treated in a Gyroscope-sponsored Antecedent StudyAge Related Macular Degeneration (AMD)NCT05481827Gyroscope Therapeutics Limited225
Enrolling By Invitation
不适用
Long Term Follow-up for RGX-202Duchenne Muscular DystrophyNCT06491927REGENXBIO Inc.66
终止
2 期
Long-Term Safety Study of GS-6624 in Adults With Idiopathic Pulmonary Fibrosis (IPF)Idiopathic Pulmonary FibrosisNCT01759511Gilead Sciences34
终止
2 期
Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)Glucose Transporter Type 1 Deficiency SyndromeNCT02599961Ultragenyx Pharmaceutical Inc15
已完成
2 期
A Phase 2 Extension Study of Study GCS-100-CS-4002Chronic Kidney DiseaseNCT02155673La Jolla Pharmaceutical Company92