CTRI/2021/11/037956进行中（未招募）3 期
A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are greater than or equal to 2 years to less than 18 years of age, treatment naive, sitting, and never ambulatory
ovartis Healthcare Pvt Ltd0 个研究点目标入组 0 人开始时间: 待定最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
入选标准
- •Diagnostic confirmation during screening period of SMA caused by biallelic SMN1 pathogenic variants affecting SMN1 and 2-4 copies of SMN2
- •- The patient must be treatment naive for all SMN dependent therapies (e.g., risdiplam (Evrysdi) and nusinersen (Spinraza)).
- •- = 2 years and < 18 years of age at time of screening
- •- Onset of clinical signs and symptoms at = 6 months of age
- •- Patient must have a complete HFMSE assessment, with available total score as administered by qualified clinical evaluator during the screening period for trial eligibility
- •- Able to sit independently at screening, but has never had the ability to walk independently.
- •--Definition of sitting independently: Child sits up straight with the head erect for at least 10 seconds without using arms or hands to balance body or support position (Wijnhoven et al 2004).
- •--Definition of walking independently: The child is able to balance the body and control forward stepping movements without assistance (Wijnhoven et al 2004).
排除标准
- •- Anti-adeno-associated virus serotype 9 (AAV9) antibody titers > 1:50 as determined by enzyme-linked immunosorbent assay (ELISA) binding immunoassay. NOTE: A negative anti-AAV9 antibody titer is defined as = 1:50.
- •- Presence of the following:
- •. An active infectious process requiring systemic antiviral or antimicrobial therapy at any time between onset of screening and dosing of OAV101 or the sham procedure
- •· An active but untreated viral or bacterial infectious process at any time between onset of screening and dosing of OAV101 or the sham procedure
- •· Any febrile illness within two weeks prior to start of screening, during screening period or during baseline period up to OAV101 treatment or sham procedure
- •· Hepatic dysfunction (i.e., aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin, gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH), > upper limit of normal (ULN) (Common Terminology Criteria for Adverse Events (CTCAE) grade1 or greater) at Screening Visit 1. NOTE: In the absence of other liver laboratory abnormalities, isolated AST elevation is not considered exclusionary
- •· Requiring invasive or awake noninvasive ventilation for > 6 hours during a 24-hour period, invasive or noninvasive ventilation for > 12 hours during a 24-hour period, or requiring tracheostomy during the 4 weeks prior to screening or baseline.
- •· Complications at screening, as determined by theInvestigator, that would interfere with motor efficacy assessments
- •. Body mass index (BMI) < 3rd percentile
研究者
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