EUCTR2021-003474-31-DK进行中（未招募）1 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 = 2 to < 18 years of age, treatment naive, sitting, and never ambulatory - STEER
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 125
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •- Diagnostic confirmation during screening period 5q SMA
- •- The patient must be treatment naive (historical or current use) for all SMN-targeting therapies (e.g., risdiplam (Evrysdi) and nusinersen (Spinraza)).
- •- =2 years and <18 years of age at screening visit 1
- •- 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.
- •- Estimated life expectancy > 2 years from screening in the opinion of the Investigator
- •- Meets age-appropriate institutional criteria for use of anesthesia/sedation as assessed by the physician responsible for
- •administering anesthesia/sedation
- •(Full list of inclusion criteria can be found in the clinical protocol Section 5.1)
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 125
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •- Excluding SMA, any medical condition considered clinically significant
- •by the Investigator, including cardiomyopathy, hepatic dysfunction,
- •kidney disorder, endocrine disorder including diabetes
- •mellitus, gastrointestinal disorders, metabolic disorders, untreated B6
- •deficiency, severe respiratory compromise and significant brain
- •abnormalities at either Screening or Baseline that, in the opinion of the
- •investigator, would interfere with the overall interpretation of safety or
- •efficacy of the study.
- •- Anti-AAV9 antibody titer reported as elevated (reference to >1:50 or
- •validated results consistent with being elevated) at Screening as
- •determined by ligand binding immunoassay.
- •- Clinically significant abnormalities in test results during screening
- •period and/or during baseline period as determined by the Investigator
- •- Participants will be excluded from the trial for inpatient surgery
- •hospitalization, hospitalization for a pulmonary event, or hospitalization
- •for nutritional support within 2 months prior to Screening and up to Day
- •1. In addition, patients will not be eligible for the trial if at Screening,
- •inpatient major surgery is planned at any time during the 64-week
- •study. Any other surgeries must not interfere with the ability of the
- •patient to perform the study assessments.
- •- Prior injury (e.g., upper or lower limb fracture) or surgical procedure
- •which impacts the participant's ability to perform any of the outcome
- •measure testing required in the protocol and from which the participant
- •has not fully recovered or achieved a stable baseline upon entry into
- •screening period
- •- Contraindications for lumbar puncture procedure, (including but not
- •limited to cutaneous infection at the treatment site and signs or
- •symptoms of increased intracranial pressure), active administration of
- •any intrathecal therapy, presence of an implanted shunt for the drainage
- •of CSF, presence of an implanted central nervous system (CNS) catheter,
- •or any impediment to CSF access.
- •(Full list of exclusion criteria can be found in the clinical protocol Section
研究者
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