EUCTR2020-004590-51-DE进行中（未招募）1 期
A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation - ATLAS
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 150
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
- •Key Part A Inclusion Criteria:
- •- Participants should have a protocol-defined rapidly progressive SOD1 mutation, confirmed by a central reader, or a SOD1 mutation that is approved for inclusion by an external mutation adjudication committee.
- •- Participants with plasma NfL level less than the protocol-defined threshold.
- •- Participants who are clinically presymptomatic for ALS (i.e., must not have clinically manifest ALS).
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 105
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 45
排除标准
- •Key Part A Exclusion Criteria:
- •- History or positive test result at screening for human immunodeficiency
- •virus (HIV). The requirement for testing at Screening may be omitted if
- •it is not permitted by local regulations.
- •- Current hepatitis C infection (defined as positive Hepatitis C Virus
- •(HCV) antibody and detectable HCV RNA). Participants with positive HCV
- •antibody and undetectable HCV Ribonucleic Acid (RNA) are eligible to
- •participate in the study (United States Centers for Disease Control and
- •Prevention).
- •- Current hepatitis B infection (defined as positive for hepatitis B surface
- •antigen (HBsAg) and/or anti-Hepatitis B Core antibody (HBc)).
- •Participants with immunity to hepatitis B from previous natural infection
- •(defined as negative HBsAg, positive anti-HBc, and positive antihepatitis
- •B surface antibody (HBs) or vaccination (defined as negative
- •HBsAg, negative anti-HBc, and positive anti- HBs) are eligible to
- •participate in the study.
- •- History of systemic hypersensitivity reaction to tofersen, the excipients
- •contained in the formulation, and if appropriate, any diagnostic agents to
- •be administered during the study.
- •- History of confounding neuromuscular or neurological disorder that is
- •expected to have a progressive (i.e., worsening) course during the
- •study, and/or is expected to be associated with elevations in NF, in the
- •opinion of the Investigator.
- •- Presence of risk for increased or uncontrolled bleeding and/or risk of
- •bleeding that if not managed optimally could place a participant at an
- •increased risk for intraoperative or postoperative bleeding.
- •- Significant cognitive impairment, clinical dementia, or unstable
- •psychiatric illness, including psychosis, suicidal ideation, suicide
- •attempt, or untreated major depression = 90 days of Screening, which in
- •the opinion of the Investigator would interfere with the study
- •procedures.
- •- Treatment with riluzole, edaravone and/or sodium phenylbutyrate/taurursodiol (also known as ursodoxicoltaurine). If the participant has been
- •on riluzole, edaravone and/or sodium phenylbutyrate/taurursodiol, the medication(s) must be discontinued for at least 5 half-lives prior to screening.
- •- Use of-label treatments for ALS.
- •- Treatment with another investigational drug (including investigational
- •drugs for ALS through compassionate use programs), biological agent, or
- •device within 1 month or 5 half-lives of study agent, whichever is longer.
- •Specifically, no prior treatment with small interfering RNA, stem cell
- •therapy, or gene therapy is allowed.
- •- Anticipated need, in the opinion of the Investigator, for administration
- •of any antiplatelet or anticoagulant medication (e.g., clopidogrel) that
- •cannot be safely continued or held for an LP procedure, if necessary,
- •according to local or institutional guidelines and/or Investigator
- •determination.
- •- Current enrollment or a plan to enroll in any interventional clinical
- •study in which an investigational treatment, biological agent, device, or
- •approved therapy for investigational use. Participation in a
- •noninterventional study focused on ALS natural history may be allowed
- •at the discretion of the Investigator.
- •NOTE: Other protocol defined Inclusion/Exclusion criteria will apply.
研究者
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