2024-512600-19-00招募中3 期
A Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects 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
- 40
- 试验地点
- 10
- 主要终点
- Change from Baseline in Bayley-4 Cognitive Raw Score Without Caregiver Input at Day 338
研究概览
简要总结
The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Open-label Period
- 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)
- •Males and females aged 4 to < 18 years of age, inclusive, at time of informed consent
- •Confirmed diagnosis of AS with genetic confirmation of full maternal ubiquitin-protein ligase E3A (UBE3A) gene deletion causing AS in the region of 15q11.2 q13
- •Able to ambulate independently, or with assistance at the Screening Visit (note, a child whose primary means of mobility is by wheelchair is excluded from the study)
- •Platelet count, prothrombin time / international normalized ratio, and partial thromboplastin time < 1.5x the upper limit of normal (CCI) at the Screening Visit
- •Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, and all study procedures, including LP procedure. MRI, and tolerating anesthesia without intubation
- •From the time of informed consent through to 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
- •Any change in medications or diet/supplements intended to treat symptoms of AS (eg, sleeping aids, antiseizure medications, supplements, dietary change including ketogenic or low-glycemic index diet, other) within the month prior to the Screening Visit (excluding weight-based adjustments)
- •Concurrent participation in any interventional study
- •Any condition that creates an increased risk of unsuccessful LP
- •Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors)
- •Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
- •Presence or history of any condition, lab abnormality, or infection that, in the judgement of the Investigator, would interfere with participation, pose undue safety risk, or would confound interpretation of results
- •Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
- •Use of any investigational product or investigational medical device within 6 months or 5 half-lives prior to the Screening Visit or any prior use of gene therapy or ASO regardless of duration since last administration
结局指标
主要结局
Change from Baseline in Bayley-4 Cognitive Raw Score Without Caregiver Input at Day 338
Change from Baseline in Bayley-4 Cognitive Raw Score Without Caregiver Input at Day 338
次要结局
- Net Response in MDRI at Day 338
- Change from Baseline in ABC-C Hyperactivity/Noncompliance Subscale Score at Day 338
- Change from Baseline at Day 338 in Bayley-4 Receptive Communication raw score
- Change from Baseline in ASA Sleep Rating Raw Score at Day 338
- Number of Participants with Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs), Severity of AEs and Relationship to Investigational Drug, Procedure, and Premedication
- Changes from Baseline in Vineland Adaptive Behavior Scales-3 (Vineland-3) Receptive Communication raw score at Day 338
- Changes from Baseline in Vineland-3 Expressive Communication raw score at Day 338
- Changes from Baseline in Bayley-4 Gross Motor raw score at Day 338
- Changes from Baseline in ASA Gross Motor rating at day 338
研究者
Medical Information
Scientific
Ultragenyx Pharmaceutical Inc.
研究点 (10)
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