Trial · NCT03533257
Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease
NCT03533257, Phase 2, Completed, Amylyx Pharmaceuticals Inc.; results first posted 2025-03-07
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease. Trials, record NCT03533257. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03533257
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease
- NCT number
- NCT03533257
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 95 participants, actual
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Conditions
- Alzheimer Disease
- Interventions
- AMX0035, Placebo
- First posted
- 2018-05-23
- Last updated
- 2025-03-07
- Results first posted
- 2025-03-07
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Event (TEAEs) | From first dose to 24 weeks | Comparison between the AMX0035 Group and Placebo of the number of participants with TEAEs |
| Primary | Effect of Treatment on a Global Composite Statistical Test of Cognition, Function, and Neuroanatomy (GST) | 24 weeks | Change from Baseline in GST (global statistical test combining three measures relevant to disease trajectory (cognition \[MADCOMS: Mild/Moderate Alzheimer's Disease Composite Score\], function \[FAQ: Functional Activities Questionnaire\], and total hippocampal volume on magnetic resonance imaging)) for AMX0035 relative to placebo. For MADCOMS and FAQ, a higher score indicates a worse outcome. A larger hippocampal volume is better, so it was reversed before being normalized. Each of the three were normalized against respective baseline means and standard deviations. The mean of the three normalized scores is the final GST. A higher GST score indicates a worse outcome. Standard deviations above the mean are worse; standard deviations below the mean are better. The expected value of the GST at baseline is 0 because it is the mean of three z-scores whose expected values at baseline are 0. AD is multifaceted and the GST was designed to be sensitive to changes in multiple dimensions. |
| Secondary | Effect of Treatment on Cognition | 24 weeks | Impact of AMX0035 on clinical symptoms as measured by Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-Cog). Scoring is in the range of 0 to 90 with a higher score indicating greater cognitive impairment. |
| Secondary | Effect of Treatment on Functioning | 24 weeks | Impact of AMX0035 on Functional Activities Questionnaire (FAQ) scores. FAQ total score can range from 0 to 30, with higher scores indicating less functional independence. |
| Secondary | Effect of Treatment on Dementia Severity | 24 weeks | Impact of AMX0035 on Dementia Severity Rating Scale (DSRS) scores. Total score can range from 0 to 54, with higher scores indicating greater severity of dementia. |
| Secondary | Effect of Treatment on Cognitive Impairment | 24 weeks | Impact of AMX0035 on the Montreal Cognitive Assessment (MoCA) scores. MoCA scores can range from 0 to 30, with lower scores indicating greater cognitive impairment. |
| Secondary | Effect of Treatment on Neuropsychiatric Symptoms | 24 weeks | Impact of AMX0035 on neuropsychiatric symptoms as assessed by the Neuropsychiatric Inventory (NPI). The total score ranges from 0 to 36, with higher scores indicating a greater severity of symptoms. |
| Secondary | Regional Brain Volume | 24 weeks | Impact of AMX0035 on levels of hippocampal atrophy, as assessed by volumetric Magnetic Resonance Imaging (vMRI) |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Event (TEAEs) | From first dose to 24 weeks | Safety Population | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Effect of Treatment on a Global Composite Statistical Test of Cognition, Function, and Neuroanatomy (GST) | 24 weeks | ITT population | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Effect of Treatment on Cognition | 24 weeks | ITT population | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Effect of Treatment on Functioning | 24 weeks | ITT population | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Effect of Treatment on Dementia Severity | 24 weeks | ITT population | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Effect of Treatment on Cognitive Impairment | 24 weeks | ITT population | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Effect of Treatment on Neuropsychiatric Symptoms | 24 weeks | ITT population | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Regional Brain Volume | 24 weeks | ITT population | mm^3 | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Event (TEAEs) | Active (AMX0035) | 34 | Participants | ||
| Number of Participants With Treatment-Emergent Adverse Event (TEAEs) | Placebo | 26 | Participants | ||
| Effect of Treatment on a Global Composite Statistical Test of Cognition, Function, and Neuroanatomy (GST) | Active (AMX0035) | 0.24 | Standard Error 0.049 | score on a scale | |
| Effect of Treatment on a Global Composite Statistical Test of Cognition, Function, and Neuroanatomy (GST) | Placebo | 0.17 | Standard Error 0.052 | score on a scale | |
| Effect of Treatment on Cognition | Active (AMX0035) | 2.26 | Standard Error 1.402 | score on a scale | |
| Effect of Treatment on Cognition | Placebo | 1.52 | Standard Error 1.496 | score on a scale | |
| Effect of Treatment on Functioning | Active (AMX0035) | 2.61 | Standard Error 0.790 | score on a scale | |
| Effect of Treatment on Functioning | Placebo | 1.59 | Standard Error 0.845 | score on a scale | |
| Effect of Treatment on Dementia Severity | Active (AMX0035) | 2.34 | Standard Error 0.595 | score on a scale | |
| Effect of Treatment on Dementia Severity | Placebo | 1.81 | Standard Error 0.636 | score on a scale |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Active (AMX0035) | 51 | |
| Overall Study | STARTED | Placebo | 44 | |
| Overall Study | COMPLETED | Active (AMX0035) | 41 | |
| Overall Study | COMPLETED | Placebo | 42 | |
| Overall Study | NOT COMPLETED | Active (AMX0035) | 10 | |
| Overall Study | NOT COMPLETED | Placebo | 2 |
Source ClinicalTrials.gov
Adverse events12
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Active (AMX0035) | 8 | 51 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 3 | 44 | |
| other | Headache | Nervous system disorders | Active (AMX0035) | 4 | 51 | |
| other | Headache | Nervous system disorders | Placebo | 5 | 44 | |
| serious | Depression | Psychiatric disorders | Active (AMX0035) | 1 | 51 | 1 |
| serious | Depression | Psychiatric disorders | Placebo | 0 | 44 | 0 |
| serious | Burns third degree | Injury, poisoning and procedural complications | Active (AMX0035) | 1 | 51 | 1 |
| serious | Burns third degree | Injury, poisoning and procedural complications | Placebo | 0 | 44 | 0 |
| serious | Pneumonia viral | Infections and infestations | Active (AMX0035) | 1 | 51 | 1 |
| serious | Pneumonia viral | Infections and infestations | Placebo | 0 | 44 | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Active (AMX0035) | 0 | 51 |
| Total, all-cause mortality | Placebo | 0 | 44 |
| Total, other adverse events | Active (AMX0035) | 12 | 51 |
| Total, other adverse events | Placebo | 6 | 44 |
| Total, serious adverse events | Active (AMX0035) | 3 | 51 |
| Total, serious adverse events | Placebo | 1 | 44 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Active (AMX0035) vs Placebo: deaths from any cause | Trial comparisons |
| Active (AMX0035) vs Placebo: Number of Participants With Treatment-Emergent Adverse Event (TEAEs) | Trial comparisons |
| Active (AMX0035) vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03533257
GET https://conductscience.org/api/trials/v1/NCT03533257/registered