NCT07832370尚未招募1 期
Phase 1/2 Open-Label Trial With Extended Follow-Up to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Multiple Doses of ALN-6457 in Participants With Symptomatic Prion Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 60
- 主要终点
- Percentage change in concentration of Neurofilament Light chain (NfL) in plasma from baseline
研究概览
简要总结
This study will test a Regeneron drug called ALN-6457 ("study drug") to see if it can help treat Prion Disease (PrD).
In PrD, the prion protein folds into an abnormal shape. These abnormal proteins build up in the brain and cause damage to brain cells, leading to problems with thinking, memory, and movement.
ALN-6457 is designed to reduce the amount of prion protein made in the brain. ALN-6457 works by targeting the genetic "instructions" that cells use to produce this protein, with the goal of slowing or stopping disease progression.
This study is looking at:
- What side effects ALN-6457 might cause
- How well ALN-6457 works
- What is the best dose of ALN-6457 for treating prion disease
- How much ALN-6457 is in the blood and Cerebrospinal Fluid (CSF, fluid that surrounds the brain and spinal cord) at different times
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must meet all of the criteria under either:
- •sporadic CJD as described in the protocol or
- •genetic PrD, as described in the protocol
- •Has a Body Mass Index (BMI) <35 kg/m2 (inclusive) at the screening visit
- •Able to understand and complete trial-related questionnaires at screening
排除标准
- •Clinically significant cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, psychiatric or neurological disease (excluding prion disease) that, as assessed by the investigator, may confound the results of the trial or pose an additional risk to the participant during trial participation
- •Medical history of brain or spinal disease/injury that would interfere with the Lumbar Puncture (LP) procedure or CSF circulation, as described in the protocol
- •Any contraindications to undergo a brain Magnetic Resonance Imaging (MRI)
- •An established allergy or intolerance to local anesthetics
- •Current history of bleeding diatheses that would increase risk of bleeding upon LP
- •Any malignancy that is progressing or requires active treatment, as described in the protocol
- •Attempted suicide, suicidal ideation with a plan that required hospital admission and/or change in level of care within 12 months prior to screening
- •NOTE: Other Protocol-Defined Inclusion/Exclusion Criteria Apply.
研究组 & 干预措施
Dose Escalation
Experimental
干预措施: ALN-6457 (Drug)
Cohort Expansion
Experimental
干预措施: ALN-6457 (Drug)
结局指标
主要结局
Percentage change in concentration of Neurofilament Light chain (NfL) in plasma from baseline
时间窗: Through Week 130
Occurrence of Treatment Emergent Adverse Events (TEAEs)
时间窗: Through Week 156
Severity of TEAEs
时间窗: Through Week 156
次要结局
- Concentration of Prion Protein (PrP) in CSF(Through Week 130)
- Change from baseline in concentration of PrP in CSF(Through Week 130)
- Concentration of ALN-6457 and its major metabolites in plasma(Through Week 130)
- Concentration of ALN-6457 and its major metabolites in CSF(Through Week 130)
- Overall survival rate(At Week 52)
研究者
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