Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS (Aspire) (GTX-102-CL301)

Ultragenyx Pharmaceutical Inc.0 个研究点目标入组 6 人开始时间: 2025年5月27日最近更新: 2025年6月9日

适应症

试验速览

暂无简介。

•Signed informed consent from parent(s) or legal guardian(s)
•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 within 1.5x the normal limits at the Screening Visit
•Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, and all study procedures, including LP procedure and tolerating anesthesia without intubation

•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)
•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 that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results
•human immunodeficiency virus (HIV) antibodypositive,hepatitis B (HBs) antigen-positive, HBs or HBc antibody-positive, or TB
•positive by laboratory testing at the Screening Visit
•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
•Concurrent participation in any study, including observational natural history studies

Bayley-4 Cognitive raw score (without caregiver input)

时间窗: baseline to Day 338

the change from baseline to Day 338 for the GTX-102 group compared with the Sham-LP group

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发起方