JPRN-jRCT2073220094进行中（未招募）3 期
Phase IIIb, open-label, single-arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101 administered intrathecally (1.2 x 10^14 vector genomes) to participants 2 to < 18 years of age with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen (Spinraza) or risdiplam (Evrysdi)
Hirano Takamitsu0 个研究点目标入组 4 人开始时间: 2023年1月22日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 4
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
- 年龄范围
- >= 2age old 至 < 12age old（—）
- 性别
- All
入选标准
- •1. SMA diagnosis
- •2. Aged 2 to < 18 years
- •3. Have had at least four loading doses of nusinersen (Spinraza) or at least 3 months of treatment with risdiplam (Evrysdi) at Screening
- •4. Must have symptoms of SMA as defined in the protocol
排除标准
- •1. Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an immunoassay is reported as elevated
- •2. Clinically significant abnormalities in test results during screening
- •3. Contraindications for lumbar puncture procedure
- •4. At Baseline, participants are excluded if they received:
- •Nusinersen (Spinraza) or risdiplam (Evrysdi) within a defined timeframe
- •5. Vaccinations 2 weeks prior to administration of OAV101
- •6. Hospitalization for a pulmonary event, or for nutritional support within 2 months prior to Screening or inpatient major surgery planned.
- •7. Presence of an infection or febrile illness
- •8. Requiring invasive ventilation
研究者
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