The study aims to measure safety, tolerability and biomarker-based efficacy of NPI-001 (AT-001) in Subjects with MCI or Alzheimer's Disease (AD).
In the study participants receive increasing dose of active treatment (250mg vs 500mg vs 750mg) or matched placebo in the form of tablets BID.
Inclusion Criteria:
Subject has provided informed consent for participation in trial.
Subject is male or female, aged 50 or older but younger than 85.
Female Subjects:
Male subjects:
Subject has MCI or mild dementia due to Alzheimer´s disease according to Jack 2024 with a CDR of 0.5 or 1.0 receiving standard AD care (SOC) (65).
Subject has an MMSE score of >21 < 28
Subject is willing to have a baseline and follow up blood tests according to the schedule of assessments, for up to 12 months.
Subject has a pTau 217 value of > 0.35 pg/ml
Subject is willing and able to undergo MRI, and amyloid PET scan evaluations of the brain, which fulfill the following requirements:
Subject has a spouse/close relative/caregiver as study partner who lives with him/her or supervises subject care. The caregiver must be willing to accompany the subject to all study visits and able to give informed consent for their own participation and be able to read and write.
If taking concomitant medications, treated with stable doses of drugs essentially required for chronic medical conditions which do not lead to exclusion, during a period of at least 3 months prior to screening, and dose regimen is expected to remain stable during the conduct of the study.
Subject is able to read, write, speak clearly for the cognitive tests, with eyesight and hearing sufficient to enable completion of the cognitive tests.
Exclusion Criteria:
Subject has MRI evidence evaluated by central read of
Subject has moderate or severe dementia, defined as CDR of ≥ 2.0
Subject has clinically significant illness, mental or physical, that, in the opinion of the investigator, might confound the results of the study, pose additional risk to the Subject by their participation, or prevent/impede the subject from completing the study.
Subject has known sensitivity to NAC/NACA.
Known or recently suspected (3 months) excessive alcohol or drug abuse.
History of liver disease.
There is any concern by the investigator regarding the subject's safety, compliance, or suitability with respect to his/her participation in the study.
Use of NAC or other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment, or within 14 days, whichever is longer.
Subjects who are or have been on AD immunotherapy.
Any suicidal ideation or suicidal behavior in the C-SSRS (C-SSRS score > 0)
The group receives increasing dose of the active treatment and SOC
The group receives dose of the placebo and SOC
Gandrup, Denmark