Alzheimer´s DiseasePhase 2AD BiomarkerNCT07741526
Safety, Tolerability and Biomarker-based Efficacy of NPI-001 (AT-001) in Subjects With MCI or Alzheimer's Disease (AD)
A Phase 2 study testing NPI-001 in people with Alzheimer´s Disease, run by Arctic Therapeutics at 4 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 50 to 84 years
- Conditions the study is for
- Alzheimer´s Disease
- What is being tested?
- NPI-001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 33 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 14 Oct 2025to 31 May 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 84 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject has provided informed consent for participation in trial.
- Subject is male or female, aged 50 or older but younger than 85.
- Female Subjects:
- Of child-bearing potential in the age range of 50-60 years who are still menstruating, are willing to perform a pregnancy test at screening visit, V1 (Baseline) and at each subsequent visit and adhere to contraception requirements.
- Of nonchildbearing Potential (WONCBP), women in the following categories are considered WONCBP: Premenopausal female permanently sterile due to one of the following (for the purpose of this study): Women who have documented hysterectomy, documented bilateral salpingectomy, documented bilateral oophorectomy
- Postmenopausal female is defined as no menses for 12 months. If confirmation is needed, a high follicle stimulating hormone (FSH) level in the postmenopausal range will be used to confirm a postmenopausal state in women not using hormonal contraception or hormonal replacement therapy (HRT). Females on HRT and whose menopausal status is in doubt must discontinue HRT to allow confirmation of postmenopausal status before study enrollment.
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- Male subjects:
- Male subjects with female partners of child-bearing potential who are willing or able to adhere to contraception requirements, or male subjects with documented infertility.
- 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:
- PET amyloid brain load >32 centiloids
- MRI <8 micro bleeds
- 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.
Who cannot join
- Subject has MRI evidence evaluated by central read of
- Brain abnormality caused by other neurological disease than AD including but not limited to vascular disease (vascular dementia), acute or subacute cerebral hemorrhage, large-vessel stroke, brain tumors, inflammatory immunological or metabolic disorders.
- More than 7 microbleeds, multiple lacunes or any lacune in strategically important location, Grade 2 and 3 white matter lesions, any focal area of superficial siderosis or medical implants or foreign bodies unsuitable for MRI.
- 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.
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- 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)
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Denmark3 sites
Sanos Clinic Gandrup
Gandrup
Sanos Clinic Herlev
Herlev
Sanos Clinic Vejle
Vejle
About this study
Official title: A Phase 2a Multi-site, Randomized, Double-blind, Dose Escalated, Placebo Controlled Biomarker Study to Determine the Safety, Tolerability and Biomarker-based Efficacy of NPI-001 (AT-001) in Subjects With MCI (Mild Cognitive Impairment) or Mild Dementia Due to Alzheimer's Disease (AD) Aged 50 to 85 Years
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.
- Conditions
- Alzheimer´s Disease
- Being tested
- NPI-001, Placebo
- Keywords
- Alzheimer´s disease, Mild Cognitive Impairment, Dementia
- Medical subject headings
- Cognitive Dysfunction, Dementia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NPI-001Drug
NPI-001 tablets administered orally BID at escalating doses of 250 mg, 500 mg, and 750 mg for period of 12 months
PlaceboDrug
Placebo tablets administered orally BID at escalating doses of 250 mg, 500 mg, and 750 mg for period of 12 months
| Study group | Role | What they receive |
|---|---|---|
| Active treatment + SOC | Experimental | The group receives increasing dose of the active treatment and SOC Receives: Drug: NPI-001 |
| Placebo + SOC | Placebo comparator | The group receives dose of the placebo and SOC Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment -Emergent Adverse Events
Incidence of Treatment -Emergent Adverse Events in response to NPI (AT-001) administered orally in subjects with Alzheimer's disease (AD). Assessment of the number of participants with treatment-related adverse events. Assessment of the amount of mild and severe adverse events related to the treatment.
Measured: Through study completion, approximately up to 14 months
Safety labs results within normal range
Safety labs result not being outside of normal ranges and/or not clinically significant as assessed by the PI.
Measured: Through study completion, approximately up to 14 months
Assessment of ARIA H and ARIA E in the brain with MRI imaging
MRI imaging to assess ARIA H and ARIA E in the brain by ARIA MRI classification.
Measured: Through study completion, approximately up to 14 months
Secondary outcomes (4)
Biomarker 1: Assessment of reduction in toxic amyloid oligomers in plasma samples
Reduction in toxic amyloid oligomers in plasma samples at 3, 6, 9 and 12 months of therapy measured by SOBA method.
Measured: Through study completion, until 12 months
Biomarker 2: Assessment of the effects of NPI-001 on pTau217 levels in plasma
Reduction in pTau217 in plasma samples at 3, 6, 9 and 12 months of therapy measured by Simoa technology.
Measured: Through study completion, until 12 months
Biomarker 3: Assessment of the effects of NPI-001 on NFL in plasma
Reduction in NFL in plasma samples at 3, 6, 9 and 12 months of therapy assessed by ELISA.
Measured: Through study completion, until 12 months
Biomarker 4: Assessment of reduction in amyloid accumulation in the brain following 12 months of therapy (PET)
To determine if any reduction/reversal of amyloid accumulation in the brain of subjects with AD in response to NPI-001, administered orally.
Measured: Through study completion, until 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2026
- Study start14 Oct 2025
- Primary completion30 Apr 2027 (estimated)
- Study completion31 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alzheimer´s DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arctic Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Blueskin AS
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07741526
- Sponsor's protocol ID
- AT-24-03
- Other IDs
- 2024-519497-39-00
- Acronym
- AD Biomarker
- Status last verified
- Jul 2026
- First submitted
- 18 Mar 2026
- Last updated
- 3 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a Multi-site, Randomized, Double-blind, Dose Escalated, Placebo Controlled Biomarker Study to Determine the Safety, Tolerability and Biomarker-based Efficacy of NPI-001 (AT-001) in Subjects With MCI (Mild Cognitive Impairment) or Mild Dementia Due to Alzheimer's Disease (AD) Aged 50 to 85 Years
- ClinicalTrials.gov ID
- NCT07741526
- Acronym / sponsor ID
- AD Biomarker / AT-24-03
- Conditions
- Alzheimer´s Disease
- Keywords
- Alzheimer´s disease, Mild Cognitive Impairment, Dementia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Arctic TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Blueskin AS
- Study start
- 14 Oct 2025 (actual)
- Primary completion
- 30 Apr 2027 (estimated)
- Study completion
- 31 May 2027 (estimated)
- First posted
- 3 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active treatment + SOC | Experimental | Drug: NPI-001The group receives increasing dose of the active treatment and SOC |
| Placebo + SOC | Placebo comparator | Drug: PlaceboThe group receives dose of the placebo and SOC |
Who may qualify
- Ages
- 50 to 84 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Subject has provided informed consent for participation in trial.
- Subject is male or female, aged 50 or older but younger than 85.
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NPI-001NPI-001 tablets administered orally BID at escalating doses of 250 mg, 500 mg, and 750 mg for period of 12 months | Drug | Active treatment + SOC |
| PlaceboPlacebo tablets administered orally BID at escalating doses of 250 mg, 500 mg, and 750 mg for period of 12 months | Drug | Placebo + SOC |
Primary outcome
Incidence of Treatment -Emergent Adverse Events
Time frame: Through study completion, approximately up to 14 months
Incidence of Treatment -Emergent Adverse Events in response to NPI (AT-001) administered orally in subjects with Alzheimer's disease (AD). Assessment of the number of participants with treatment-related adverse events. Assessment of the amount of mild and severe adverse events related to the treatment.
Safety labs results within normal range
Time frame: Through study completion, approximately up to 14 months
Safety labs result not being outside of normal ranges and/or not clinically significant as assessed by the PI.
Assessment of ARIA H and ARIA E in the brain with MRI imaging
Time frame: Through study completion, approximately up to 14 months
MRI imaging to assess ARIA H and ARIA E in the brain by ARIA MRI classification.
4 secondary outcomes
Biomarker 1: Assessment of reduction in toxic amyloid oligomers in plasma samples
Time frame: Through study completion, until 12 months
Biomarker 2: Assessment of the effects of NPI-001 on pTau217 levels in plasma
Time frame: Through study completion, until 12 months
Biomarker 3: Assessment of the effects of NPI-001 on NFL in plasma
Time frame: Through study completion, until 12 months
Biomarker 4: Assessment of reduction in amyloid accumulation in the brain following 12 months of therapy (PET)
Time frame: Through study completion, until 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2026.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NPI-001 in people with Alzheimer´s Disease, run by Arctic Therapeutics at 4 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 3 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Denmark, Iceland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment -Emergent Adverse Events (time frame: Through study completion, approximately up to 14 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Aug 2026.