NL-OMON55647已完成不适用
A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults with C9ORF72-Associated Amyotrophic Lateral Sclerosis - 245AS101 - ALS
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Biogen
- 入组人数
- 3
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
- 18 至 99（—）
入选标准
- •- Ability of the participant to understand the purpose and risks of the
- •study, to provide signed and dated informed consent, and to authorize
- •the use of confidential health information in accordance
- •with national and local participant privacy regulations; or, in the event of
- •the participant's physical incapacity to sign, to confirm that
- •understanding and consent orally to a legally authorized representative
- •(LAR) for the express purpose of having said informed consent and
- •authorization signed on his/her behalf.
- •- All participants of childbearing potential must agree to practice highly
- •effective contraception during the study and be willing and able to
- •continue contraception for 5 months after their last dose of study
- •- Must meet the possible, laboratory-supported probable, probable, or
- •definite criteria for diagnosing ALS according to the World Federation of
- •Neurology El Escorial criteria and have documentation of a clinical
- •genetic test demonstrating the presence of a pathogenic mutation in
- •-Slow vital capacity (SVC) * 50% of predicted value as adjusted for sex,
- •age, and height (from the sitting position).
- •- Participants taking concomitant riluzole at study entry must be on a
- •stable dose for * 30 days prior to the first dose of study treatment (Day
- •- Participants taking concomitant edaravone at study entry must be on a
- •stable dose for * 60 days prior to the first dose of study treatment (Day
- •- ALS Cognitive Behavioral Screen (ALS-CBS) score * 11 for the cognitive
- •portion; * 33 for the behavioral portion.
- •- Medically able to undergo the study procedures, and to adhere to the
- •visit schedule at the time of study entry, as determined by the
- •Investigator.
- •- Screening values of coagulation parameters including platelet count,
- •international normalized ratio (INR), prothrombin time (PT), and
- •activated partial thromboplastin time (APTT) should be within normal
- •- Has an informant/caregiver who, in the Investigator's judgment, has
- •frequent and sufficient contact with the participant as to be able to
- •provide accurate information about the participant's cognitive and
- •functional abilities at Screening.
排除标准
- •- History of drug abuse or alcoholism * 6 months of Screening that would limit
- •participation in the study, as determined by the Investigator.
- •- Tracheostomy.
- •- Prescreening ALSFRS-R slope less than 0.4 point/month, where prescreening
- •ALSFRS-R slope is defined as follows: (48-ALSFRS-R score at Screening) /
- •(months from date of symptom onset to date of Screening).
- •- History of or positive test result at Screening for human immunodeficiency
- •- History of, or positive test result at Screening for, hepatitis C virus
- •- Treatment with another investigational drug or biological agent within 1
- •month of Screening or 5 half-lives of study agent, whichever is longer.
- •- Treatment with an antiplatelet or anticoagulant therapy that cannot safely be
- •interrupted for lumbar puncture (LP) according to local standard of care and/or
- •institutional guidelines, in the opinion of the Investigator or Prescriber.
- •- Current or anticipated need, in the opinion of the Investigator, of a
- •diaphragm pacing system during the study period.
- •- Female participants who are pregnant or currently breastfeeding.
研究者
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