NCT05930223Available不适用
Intravenous VAL-1221 Lafora Expanded Access Protocol (LEAP)
Parasail, LLC0 个研究点开始时间: 2023年7月5日最近更新:
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- Available
- 发起方
研究概览
简要总结
Only qualified physicians treating a Lafora Disease patient may request VAL-1221 Expanded Access treatment under the Parasail Lafora Expanded Access Protocol (LEAP). According to FDA guidance, this protocol can enroll up to 10 patients. However, individual patient inclusion will ultimately be dependent on protocol eligibility, site geography, treatment requirements, and available supply of the investigational therapy. Inquiring physicians can submit a request by contacting the Central Contact personnel listed below in Contacts/Locations.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 12 Years 至 28 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented genetic diagnosis of Lafora Disease (LD) based on likely pathogenic or pathogenic variants in both alleles of either the EPM2A or the EPM2B gene
- •Mid-stage in evolution of LD between 12 and 28 years of age
- •Able and willing to comply with the protocol, including travel to Protocol Center, procedures, measurements and visits, including:
- •Adequately supportive psychosocial circumstances, in the opinion of the Investigator
- •Caregiver/trial partner committed to facilitate patient's involvement in the study who is reliable, competent, at least 18 years of age
排除标准
- •Have any other conditions, which, in the opinion of the Investigator or Sponsor would make the participant unsafe for inclusion or could interfere with the participant participating in or completing the protocol
研究者
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