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| ID | Type | Description | Link |
|---|---|---|---|
| 1R01AG061146-01 | U.S. NIH Grant/Contract | View source |
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It has been made the strategic decision to terminate the study. This decision to terminate the study early is not being made for safety reasons.
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| Name | Class |
|---|---|
| Alzheimer's Disease Cooperative Study (ADCS) | OTHER |
| National Institute on Aging (NIA) | NIH |
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This is a Phase 2A multi-center, randomized, double blind, placebo-controlled, parallel group study of varoglutamstat, with a stage gate to Phase 2B.
In Phase 2A there will be adaptive dosing evaluation of three dose levels with exposure to varoglutamstat or placebo for a minimum of 24 weeks, with preliminary evaluation of both cognitive function and pharmacodynamic changes on EEG spectral analysis in approximately 180 participants.
In the event that the stage gate for Phase 2B is reached, then Phase 2B will assesses efficacy and longer-term safety in a larger study group, i.e., 414.
The goal of this study is to advance a first-in-class, new small molecule treatment for early Alzheimer's disease (AD). Varoglutamstat (PQ912) is an oral, twice daily medication that addresses a novel and significantly differentiated amyloid target: N-terminal post-translationally modified Ab (pGlu-Ab), a particularly toxic subspecies of amyloid beta (Ab).
The study is a Phase 2A multi-center, randomized, double-blind, parallel group trial, with a stage gate to Phase 2B.
This study is conducted to further evaluate whether varoglutamstat's mechanism of action can result in a measurable therapeutic effect on cognition, function and relevant pharmacodynamic (PD) and biological markers in early AD.
Phase 2A is designed to determine the highest dose that is both safe and well tolerated using a predefined Pocock safety stopping boundary based on the rate of adverse events of special interest (AESIs).
During this phase there is an adaptive dosing evaluation with exposure to varoglutamstat or placebo for a minimum of 24 weeks (Phase 2A). Participants are randomized 1:1 to varoglutamstat or placebo, and randomization is stratified between mild AD and MCI, as well as by site.
During Phase 2A participants are to be enrolled sequentially into one of three dose cohorts (labelled Cohort A, B and C, with 60 participants per cohort (n=30 active, n=30 placebo)) and treated at the originally assigned full dose until the Data Safety Monitoring Board (DSMB) provides a protocol-specified dose decision:
Cohort A (600 mg): First 4 weeks 150 mg BID, week 5-8 300 mg BID, week 9-up to 72 600 mg BID;
Cohort B (300 mg): First 4 weeks 150 mg BID, week 5-up to 72 300 mg BID;
Cohort C (150 mg): up to 72 weeks on 150 mg BID.
In addition, at the end of Phase 2A (after 24 weeks) an interim analysis for futility will be conducted evaluating both cognitive function (ADNI Battery Composite score) and pharmacodynamics changes on electroencephalogram (EEG) spectral analysis (EEG theta power) to inform a stage-gate decision on whether to proceed with Phase 2B (72 weeks). If the Phase 2A study meets the predefined criteria to proceed to Phase 2B, then Phase 2B will assess the longer-term efficacy and safety of varoglutamstat in a larger study group, using the dose level determined during Phase 2A. In Phase 2B, a composite cognitive and functional measure as well as PD biomarkers will be used to evaluate efficacy during the extended treatment period.
According to the protocol, during Phase 2A the DSMB performs a continuous review of the safety data using a predefined Pocock safety stopping boundary to provide a dose decision.
If Cohort A does not meet the safety stopping boundary until the last participant of the Cohort A reaches 8 weeks on full dose (600 mg BID) the DSMB will provide a dose decision as per protocol for the dose of Cohort A to be carried forward for all participants in the active arm for the remainder of the study. In case Cohort A meets the stopping boundary, all participants will be down-titrated to the next lower planned dose level (Cohort B) and the same review process by the DSMB will continue until either dose-selection, down-titration to the lowest dose level (Cohort C) or stage-gate decision. As pre-specified, the Pocock safety stopping boundary applies only until dose selection. If the first cohort (Cohort A) with the highest dose of PQ912 (600 mg BID) is selected to be carried forward, evaluation of other dose levels is no longer applicable and all data will be collected as one single active Arm/Group.
All participants randomized to PQ912 will start at 150 mg BID and will be titrated to the dose level selected per protocol; all participants in the placebo group will receive matching placebo. Participants enrolled in Phase 2A remain in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PQ912 | Experimental | All participants started at 150 mg BID and were up-titrated to 600 mg BID |
|
| Placebo | Placebo Comparator | Matching placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PQ912 | Drug | PQ912 150 mg tablets |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Phase 2A: Number of Participants With Any Adverse Event of Special Interest (AESI) | The proportion of participants, who experience any AESI during the safety evaluation period, which is from first dose to completion of 8 weeks at the full originally assigned dose. Per protocol, if the first cohort (Cohort A) does not meet the pre-specified Pocock safety stopping boundary within the active arm, that dose will be selected as safe. The dose selected as safe will be the dose carried forward for all participants in the active arm for the remainder of the study. The Pocock boundary is only valid until dose selection. If a higher dose is selected as safe, assignment to lower doses will stop, and all current participants on a lower dose will be titrated up to the selected dose according to uptitration schedule. If Cohort A does not meet the Pocock boundary only evaluation of Cohort A is required for this endpoint. | From first dose to completion of 8 weeks on the full dose (Week 16) |
| Phase 2A: Median Estimated Target Occupancy (TO) of PQ912 in Cerebrospinal Fluid (CSF) at Week 24 | The pharmacokinetics (PK) endpoints in Phase 2A are the derived median values of PQ912 levels in plasma and the corresponding calculated target occupancy (TO) in CSF, following at least 8 weeks of treatment at the dose level being tested. TO in CSF was estimated from plasma PQ912 concentrations. | Week 24, pre-dose (trough level) and 2-3h post-dose |
| Phase 2A: Median Plasma Concentrations of PQ912 at Week 24 | The pharmacokinetics (PK) endpoints in Phase 2A are the derived median values of PQ912 levels in plasma and the corresponding calculated target occupancy (TO) in CSF, following at least 8 weeks of treatment at the dose level being tested. Blood samples were obtained and PQ912 plasma concentrations were determined using a validated high-pressure liquid chromatography hyphenated with tandem mass spectrometry (LC-MS/MS) method. | Week 24, pre-dose (trough level) and 2-3h post-dose |
| Phase 2A: Change From Baseline in Alzheimer's Disease Neuroimaging Initiative (ADNI) Battery Composite (ABC) Score |
| Measure | Description | Time Frame |
|---|---|---|
| Phase 2B: Key Secondary Efficacy: Change From Baseline in Cognitive Functional Composite-2 (CFC2) Score, a Cognitive Functional Composite | The within-participant change from baseline in CFC2 scores. The CFC2 is a novel composite outcome measure of cognition and everyday function that has been derived from measures used in Alzheimer's Disease Neuroimaging Initiative (ADNI) and optimized for detecting change in early Alzheimer's disease (doi.org/10.1016/j.jalz.2012.05.2187). CFC2 is comprised of selected subtests from the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) (Word Recall, Delayed Word Recall, and Orientation), Clinical Dementia Rating (CDR) sum of the Cognitive Boxes (Memory, Orientation, Judgement, and Problem Solving), and the Functional Activities Questionnaire (FAQ). The total score for each component subtest was multiplied by -1 so that higher scores indicate better performance. The CFC2 score was then calculated as the sum of the transformed raw scores for the component subtests. Range: -67 to 0; higher scores indicate less impairment. |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Howard Feldman | Alzheimer's Disease Cooperative Study (ADCS) | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Barrow Neurological Institute | Phoenix | Arizona | 85013 | United States | ||
| Banner Sun Health Research Institute |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39422941 | Background | Feldman HH, Messer K, Qiu Y, Sabbagh M, Galasko D, Turner RS, Lopez O, Smith A, Durant J, Lupo JL, Revta C, Balasubramanian A, Kuehn-Wache K, Wassmann T, Schell-Mader S, Jacobs DM, Salmon DP, Leger G, DeMarco ML, Weber F; ADCS VIVA-MIND Study Group. Varoglutamstat: Inhibiting Glutaminyl Cyclase as a Novel Target of Therapy in Early Alzheimer's Disease. J Alzheimers Dis. 2024;101(s1):S79-S93. doi: 10.3233/JAD-231126. | |
| Background | Messer K, Jacobs DM, Salmon DP, Qiu Y, Revta C, Weber F, Kuhn-Wache K, Leger GC, Feldman HH. (2022), A novel, efficient and seamless Phase 2A-2B design to test varoglutamstat in early AD: the VIVAMIND study. Alzheimer's Dement., 18(S10): e065197. | ||
| 29854937 |
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Data sharing is integral to the ADCS's (Alzheimer's Disease Cooperative Study) mission to develop and execute innovative clinical trials focused on interventions that may prevent, delay, or treat the expression of Alzheimer's disease and related dementias. The ADCS is committed to sharing resources and tools, including data, biospecimens, trial designs, outcome and analysis measures following NIH guidelines.
DATA SHARING: The ADCS Data and Sample Sharing Committee (DSSC) grants access to de-identified data to individuals who complete the request process and agree to the conditions in an ADCS/UCSD Data Use Agreement (DUA). After approval and receipt of the fully executed DUA, applicants are authorized to acquire data. Non-compliance with the DUA, including the requirement to provide requested updates will jeopardize further access to data.
January 2026
Data requestors must complete an ADCS data and sample sharing request form. Upon approval, requestors must complete a data use agreement prior to accessing the data.
Participants were randomized 1:1 to PQ912 or matching placebo. All participants randomized to PQ912 started at 150 mg BID and were up-titrated to 600 mg BID. Therefore, all data from the participants randomized to PQ912 were collected as one single active Arm/Group and separation of doses is not applicable. Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B.
Participants were enrolled at 22 study centers in the US between 2021 and 2024. The first participant was screened on November 10, 2021 and the last participant had his last visit on August 12, 2024. Of 253 screened participants, 112 met inclusion criteria and were randomized to treatment.
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| ID | Title | Description |
|---|---|---|
| FG000 | PQ912 | All participants started at 150 mg BID and were up-titrated to 600 mg BID administered orally for up to 72 weeks and therefore, all data from these participants were collected as one single active Arm/Group. Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B. PQ912: 150 mg tablets |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 17, 2023 | Jul 11, 2025 |
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| Placebo | Other | Placebo tablets to mimic PQ912 150 mg tablets |
|
The within-participant change from baseline in the composite mean of standardized scores from a set of ADNI neuropsychological test measures, the ADNI Battery Composite (ABC, 9-item). The ABC score was calculated from the following 9 measures from the ADNI-1 Neuropsychological Test Battery: Auditory Verbal Learning Test (AVLT)-Immediate Recall, AVLT-Delayed Recall, Number Span Forward, Number Span Backward, Category Fluency, Trail Making Test A, Trail Making Test B, Digit Symbol Substitution, Boston Naming Test. Standardized scores (Z-scores) were calculated using the overall mean and standard deviation of all participants in the modified Intent-to-Treat (mITT) population at baseline as reference. The ABC score for each participant was the mean of the standardized subtest values. Range: -3 to 3; higher scores indicate less impairment. |
| 24 weeks |
| Phase 2A: Change From Baseline in Quantitative Electroencephalogram (qEEG) | The within-participant change from baseline of global relative theta wave power (4-8 Hz) in qEEG. qEEG was used to assess resting-state brain activity, including global relative theta power (4-8 Hz), based on spectral analysis of signals from 21 electrode positions. Higher theta power indicates greater impairment. | 24 weeks |
| Phase 2B: Change From Baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) Score | The within-participant change from baseline in CDR-SB scores. The CDR-SB is a composite rating of cognition and everyday function which incorporates both informant input and direct assessment of performance. It assesses through semi-structured interview three cognitive domains (memory, orientation, and judgement/problem solving) and three everyday functional domains (community affairs, home and hobbies, and personal care). Level of impairment in each of the six domains is rated from none (score=0) to severe (score=3). The six domain scores are then summed to create the CDR-SB. Range: 0-18; higher scores indicate greater impairment. | 72 weeks |
| 72 weeks |
| Phase 2B: Change From Baseline in Alzheimer's Disease Neuroimaging Initiative (ADNI) Battery Composite (ABC, 9-item) Score | The within-participant change from baseline in the composite sum of standardized scores from a set of ADNI neuropsychological test measures, the ADNI Battery Composite (ABC, 9-item). The ABC score is calculated from the following 9 measures from the ADNI-1 Neuropsychological Test Battery: Auditory Verbal Learning Test (AVLT)-Immediate Recall, AVLT-Delayed Recall, Number Span Forward, Number Span Backward, Category Fluency, Trail Making Test A, Trail Making Test B, Digit Symbol Substitution, Boston Naming Test. Standardized scores (Z-scores) were calculated using the overall mean and standard deviation of all participants in the modified intent-to-treat (mITT) population at baseline as reference. The ABC score for each participant was the mean of the standardized subtest values. Range: -3 to 3; higher scores indicate less impairment. | 72 weeks |
| Phase 2B: Change From Baseline in Quantitative Electroencephalogram (qEEG) | The within-participant change from baseline of global relative theta wave power (4-8 Hz) in qEEG. qEEG was used to assess resting-state brain activity, including global relative theta power (4-8 Hz), based on spectral analysis of signals from 21 electrode positions. Higher theta power indicates greater impairment. | 72 weeks |
| Phase 2B: Change From Baseline in Functional Activities Questionnaire (FAQ) | The within-participant change from baseline in FAQ.The FAQ is a validated 10-item questionnaire, completed as a structured interview with the study partner, which rates the study participant's ability to carry out ten complex activities of daily living (doi.org/10.1093/geronj/37.3.323). Each item is rated on a scale from 0 (no impairment) to 3 (dependent). Scores are summed across items to provide a total score. Range: 0 to 30; higher scores indicate greater impairment. | 72 weeks |
| Phase 2B: Change From Baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-Cog-13) | The within-participant change from baseline in ADAS-Cog-13. The ADAS-Cog-13 is a structured scale that evaluates memory (immediate and delayed word recall; immediate word recognition), receptive and expressive language, orientation, ideational praxis (preparing a letter for mailing), constructional praxis (copying figures), and attention (number cancellation). Ratings of spoken language, language comprehension, word finding difficulty, and ability to remember test instructions also are obtained. The ADAS-Cog total score is calculated by summing the scores of the 13 component subtests. Range: 0-85; higher scores indicate greater impairment. | 72 weeks |
| Phase 2B: Change From Baseline in Neuropsychiatric Inventory (NPI) | The within-participant change from baseline in NPI. The NPI is a well-validated, reliable, multi-item instrument to assess psychopathology in Alzheimer's disease dementia based on the results of an interview with the study partner. Frequency and severity of 12 neuropsychiatric symptoms are assessed. Range: 0 to 144; higher scores indicate greater impairment. | 72 weeks |
| Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Rates of All Treatment-emergent Serious Adverse Events (SAEs) | Longer-term safety and tolerability of PQ912 compared between the active and placebo arm as measured by rates of all treatment-emergent SAEs, presented as numbers of participants with at least one SAE and percentages | 76 weeks |
| Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Suicidality on the Columbia-Suicide Severity Rating Scale (C-SSRS) | Assessment of the longer-term safety and tolerability of PQ912 via the C-SSRS. The C-SSRS is an instrument designed to assess the occurrence, severity, and frequency of suicide-related thoughts and behaviors during the corresponding assessment period. The scale includes suggested questions to elicit the type of information needed to determine if a suicide-related thought or behavior occurred. In this study it was used to 'detect' the appearance of new suicidal ideation or behavior as either "present" or "not present". The tables reflect the total number of participants (and percentage) experiencing Any suicidality, Any suicidal ideation, Any suicidal behavior, and Any self-injurious behavior at screening and subsequent time points. | Up to 72 weeks |
| Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Mortality Rates | Longer-term safety and tolerability of PQ912 compared between the active and placebo arm as measured by rates of fatal treatment-emergent serious adverse events (SAEs), presented as numbers of participants with a treatment emergent SAE leading to death and percentages. | 76 weeks |
| Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Rates of All Treatment-emergent Adverse Events (TEAEs) | Longer-term safety and tolerability of PQ912 compared between the active and placebo arm as measured by rates of all TEAEs, presented as numbers of participants with at least one TEAE and percentages | 76 weeks |
| Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Rates of All Adverse Events of Special Interest (AESIs) | Longer-term safety and tolerability of PQ912 compared between the active and placebo arm as measured by rates of all AESIs presented as numbers of participants with at least one AESI and percentages . | 76 weeks |
| Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Drug Discontinuation Rates | Longer-term safety and tolerability of PQ912 compared between the active and placebo arm as measured by rates of drug discontinuation due to treatment-emergent adverse events (TEAEs), presented as numbers of participants with a TEAE leading to drug discontinuation and percentages. | 72 weeks |
| Sun City |
| Arizona |
| 85351 |
| United States |
| The Neuron Clinic | Chula Vista | California | 91910 | United States |
| University of California | Irvine | California | 92868 | United States |
| UCSD Alzheimer's Disease Research Center | La Jolla | California | 92037 | United States |
| Cedars-Sinai Center | Los Angeles | California | 90048 | United States |
| PCND Neurology | Poway | California | 92064 | United States |
| Georgetown University Medical Center | Washington D.C. | District of Columbia | 20057 | United States |
| USF Health Byrd Alzheimer's Center and Research Institute | Tampa | Florida | 33613 | United States |
| Emory University | Atlanta | Georgia | 30329 | United States |
| Northwestern University Feinberg School of Medicine | Chicago | Illinois | 60611 | United States |
| The University of Iowa Carver College of Medicine | Iowa City | Iowa | 52242 | United States |
| The University of Kentucky Sanders-Brown Center on Aging | Lexington | Kentucky | 40504 | United States |
| Northern Light Acadia Hospital | Bangor | Maine | 04401 | United States |
| NYU Langone Health Tisch Hospital | New York | New York | 10016 | United States |
| SUNY Upstate Medical University | Syracuse | New York | 13210 | United States |
| Ohio State University | Columbus | Ohio | 43210 | United States |
| OHSU Neurology Clinic | Portland | Oregon | 97239 | United States |
| Abington Neurological Associates | Abington | Pennsylvania | 19001 | United States |
| Rhode Island Hospital | Providence | Rhode Island | 02903 | United States |
| Lowcountry Center for Veterans Research (LCVR) | Charleston | South Carolina | 29403 | United States |
| UT Health San Antonio | San Antonio | Texas | 78229 | United States |
| Background |
| Lues I, Weber F, Meyer A, Buhring U, Hoffmann T, Kuhn-Wache K, Manhart S, Heiser U, Pokorny R, Chiesa J, Glund K. A phase 1 study to evaluate the safety and pharmacokinetics of PQ912, a glutaminyl cyclase inhibitor, in healthy subjects. Alzheimers Dement (N Y). 2015 Oct 3;1(3):182-195. doi: 10.1016/j.trci.2015.08.002. eCollection 2015 Nov. |
| 30309389 | Background | Scheltens P, Hallikainen M, Grimmer T, Duning T, Gouw AA, Teunissen CE, Wink AM, Maruff P, Harrison J, van Baal CM, Bruins S, Lues I, Prins ND. Safety, tolerability and efficacy of the glutaminyl cyclase inhibitor PQ912 in Alzheimer's disease: results of a randomized, double-blind, placebo-controlled phase 2a study. Alzheimers Res Ther. 2018 Oct 12;10(1):107. doi: 10.1186/s13195-018-0431-6. |
| Result | Feldman HH, Messer K, Zhang J, Quach NE, Léger GC, Jacobs DM, Edland SD, Duehring J, MacKelfresh A, Pol A, Revta C, Lupo JL, Balasubramanian A, Lama N, Wassmann T, Schaeffer M, Hoffmann T, Meyer A, Schell-Mader S, Wenzkowski C, Weber F, for the ADCS VIVA-MIND Study Group. The VIVA-MIND study: Topline Results from Phase 2 RCT of Varoglutamstat in Early AD. In press, abstract at 2025 AAIC, Alzheimer's and Dementia |
| FG001 | Placebo | All participants received matching placebo BID administered orally for up to 72 weeks Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B. Placebo: PQ912 matching placebo tablet |
| Treated |
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| COMPLETED |
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| NOT COMPLETED |
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The modified intent-to-treat (mITT) population, including all randomized participants who took at least one dose of the study medication and who have a baseline assessment of the primary endpoint for phase 2B (CDR-SB)
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| ID | Title | Description |
|---|---|---|
| BG000 | PQ912 | All participants started at 150 mg BID and were up-titrated to 600 mg BID administered orally for up to 72 weeks and therefore, all data from these participants were collected as one single active Arm/Group. Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B. PQ912: 150 mg tablets |
| BG001 | Placebo | All participants received matching placebo BID administered orally for up to 72 weeks Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B. Placebo: PQ912 matching placebo tablet |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||
| Alzheimer's Disease Neuroimaging Initiative (ADNI) Battery Composite (ABC) score | The ABC score was calculated from 9 measures from the ADNI-1 Neuropsychological Test Battery: Auditory Verbal Learning Test (AVLT)-Immediate Recall, AVLT-Delayed Recall, Number Span Forward, Number Span Backward, Category Fluency, Trail Making Test A and, Trail Making Test B, Digit Symbol Substitution, Boston Naming Test. Standardized scores (Z-scores) were calculated using the overall mean and standard deviation of all participants in the mITT at Baseline as reference. The ABC score for each participant was the mean of the standardized subtest values. Range: -3 to 3. Higher scores are better. | Mean | Standard Deviation | Z-scores |
| ||||||||||||||
| Apolipoprotein E (APOE) genotyping | APOE (E4 +/-) genotype is associated with the risk and age of onset of Alzheimer's disease. Results of APOE genotyping (homozygous or heterozygous for the following alleles: E2, E3, E4) were used to explore a relationship between APOE genotype clinical course and response to treatment. | Two patients not included in the denominator for the calculation of percentages because of missing APOE genotype data. | Count of Participants | Participants |
| ||||||||||||||
| Clinical Dementia Rating - Sum of Boxes (CDR-SB) | The CDR-SB scale assesses cognition and daily functioning in patients with Alzheimer's disease across 6 domains (memory, orientation, judgement/problem solving, community affairs, home and hobbies, and personal care). The CDR-SB is a composite rating of cognition and everyday function which incorporates both informant input and direct assessment of performance. Level of impairment in each of the six domains is rated from none (score=0) to severe (score=3). The six domain scores are then summed up to create the CDR-SB. Range: 0-18. Higher scores indicate greater impairment. | Mean | Standard Deviation | Box scores (0-18) |
| ||||||||||||||
| Quantitative EEG (qEEG) | qEEG was used to assess resting-state brain activity, including global relative theta power (4-8 Hz), based on spectral analysis of signals from 21 electrode positions. Higher theta power indicates greater impairment. | Only paired EEGs (at least 1 Baseline and 1 Follow-up EEG) were analyzed. | Mean | Standard Deviation | Proportion (0-1) |
| |||||||||||||
| Initial diagnosis | The initial diagnosis of the participant at screening | Count of Participants | Participants |
| |||||||||||||||
| Site | Sites with 5 or fewer participants included in the mITT population were pooled for analysis | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Phase 2A: Number of Participants With Any Adverse Event of Special Interest (AESI) | The proportion of participants, who experience any AESI during the safety evaluation period, which is from first dose to completion of 8 weeks at the full originally assigned dose. Per protocol, if the first cohort (Cohort A) does not meet the pre-specified Pocock safety stopping boundary within the active arm, that dose will be selected as safe. The dose selected as safe will be the dose carried forward for all participants in the active arm for the remainder of the study. The Pocock boundary is only valid until dose selection. If a higher dose is selected as safe, assignment to lower doses will stop, and all current participants on a lower dose will be titrated up to the selected dose according to uptitration schedule. If Cohort A does not meet the Pocock boundary only evaluation of Cohort A is required for this endpoint. | All participants started at 150 mg BID and were up-titrated to 600 mg BID. As pre-specified, this endpoint presents data from first 60 participants (Cohort A) after completion of 8 weeks on full dose, 600 mg BID, because Pocock safety stopping boundary applies only until dose selection. | Posted | Count of Participants | Participants | From first dose to completion of 8 weeks on the full dose (Week 16) |
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| Primary | Phase 2A: Median Estimated Target Occupancy (TO) of PQ912 in Cerebrospinal Fluid (CSF) at Week 24 | The pharmacokinetics (PK) endpoints in Phase 2A are the derived median values of PQ912 levels in plasma and the corresponding calculated target occupancy (TO) in CSF, following at least 8 weeks of treatment at the dose level being tested. TO in CSF was estimated from plasma PQ912 concentrations. | Pharmacokinetic population, including all randomized participants who have received at least 1 dose of the study drug, and who provided sufficient PK samples to reliably estimate 1 plasma PK concentration post-baseline | Posted | Median | Full Range | Percent (%) | Week 24, pre-dose (trough level) and 2-3h post-dose |
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| Primary | Phase 2A: Median Plasma Concentrations of PQ912 at Week 24 | The pharmacokinetics (PK) endpoints in Phase 2A are the derived median values of PQ912 levels in plasma and the corresponding calculated target occupancy (TO) in CSF, following at least 8 weeks of treatment at the dose level being tested. Blood samples were obtained and PQ912 plasma concentrations were determined using a validated high-pressure liquid chromatography hyphenated with tandem mass spectrometry (LC-MS/MS) method. | Pharmacokinetic population, including all randomized participants who have received at least 1 dose of the study drug, and who provided sufficient PK samples to reliably estimate 1 plasma PK concentration post-baseline | Posted | Median | Full Range | ng/mL | Week 24, pre-dose (trough level) and 2-3h post-dose |
|
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| Primary | Phase 2A: Change From Baseline in Alzheimer's Disease Neuroimaging Initiative (ADNI) Battery Composite (ABC) Score | The within-participant change from baseline in the composite mean of standardized scores from a set of ADNI neuropsychological test measures, the ADNI Battery Composite (ABC, 9-item). The ABC score was calculated from the following 9 measures from the ADNI-1 Neuropsychological Test Battery: Auditory Verbal Learning Test (AVLT)-Immediate Recall, AVLT-Delayed Recall, Number Span Forward, Number Span Backward, Category Fluency, Trail Making Test A, Trail Making Test B, Digit Symbol Substitution, Boston Naming Test. Standardized scores (Z-scores) were calculated using the overall mean and standard deviation of all participants in the modified Intent-to-Treat (mITT) population at baseline as reference. The ABC score for each participant was the mean of the standardized subtest values. Range: -3 to 3; higher scores indicate less impairment. | The mITT population, including all randomized participants who took at least one dose of the study medication and who have a baseline assessment of the primary endpoint for phase 2B (CDR-SB) | Posted | Least Squares Mean | Standard Error | Z-scores | 24 weeks |
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| Primary | Phase 2A: Change From Baseline in Quantitative Electroencephalogram (qEEG) | The within-participant change from baseline of global relative theta wave power (4-8 Hz) in qEEG. qEEG was used to assess resting-state brain activity, including global relative theta power (4-8 Hz), based on spectral analysis of signals from 21 electrode positions. Higher theta power indicates greater impairment. | The modified intent-to-treat (mITT) population, including all randomized participants who took at least one dose of the study medication and who have a baseline assessment of the primary endpoint for phase 2B (CDR-SB). Only paired EEGs (at least 1 evaluable Baseline and 1 evaluable Follow-up EEG) were analyzed. | Posted | Least Squares Mean | Standard Error | Proportion (0-1) | 24 weeks |
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| Primary | Phase 2B: Change From Baseline in Clinical Dementia Rating - Sum of Boxes (CDR-SB) Score | The within-participant change from baseline in CDR-SB scores. The CDR-SB is a composite rating of cognition and everyday function which incorporates both informant input and direct assessment of performance. It assesses through semi-structured interview three cognitive domains (memory, orientation, and judgement/problem solving) and three everyday functional domains (community affairs, home and hobbies, and personal care). Level of impairment in each of the six domains is rated from none (score=0) to severe (score=3). The six domain scores are then summed to create the CDR-SB. Range: 0-18; higher scores indicate greater impairment. | The modified intent-to-treat (mITT) population, including all randomized participants who took at least one dose of the study medication and who have a baseline assessment of the primary endpoint for Phase 2B (CDR-SB) | Posted | Least Squares Mean | Standard Error | Box scores (0-18) | 72 weeks |
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| Secondary | Phase 2B: Key Secondary Efficacy: Change From Baseline in Cognitive Functional Composite-2 (CFC2) Score, a Cognitive Functional Composite | The within-participant change from baseline in CFC2 scores. The CFC2 is a novel composite outcome measure of cognition and everyday function that has been derived from measures used in Alzheimer's Disease Neuroimaging Initiative (ADNI) and optimized for detecting change in early Alzheimer's disease (doi.org/10.1016/j.jalz.2012.05.2187). CFC2 is comprised of selected subtests from the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) (Word Recall, Delayed Word Recall, and Orientation), Clinical Dementia Rating (CDR) sum of the Cognitive Boxes (Memory, Orientation, Judgement, and Problem Solving), and the Functional Activities Questionnaire (FAQ). The total score for each component subtest was multiplied by -1 so that higher scores indicate better performance. The CFC2 score was then calculated as the sum of the transformed raw scores for the component subtests. Range: -67 to 0; higher scores indicate less impairment. | The modified intent-to-treat (mITT) population, including all randomized participants who took at least one dose of the study medication and who have a baseline assessment of the primary endpoint for phase 2B (CDR-SB) | Posted | Least Squares Mean | Standard Error | Total score (-67 to 0) | 72 weeks |
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| Secondary | Phase 2B: Change From Baseline in Alzheimer's Disease Neuroimaging Initiative (ADNI) Battery Composite (ABC, 9-item) Score | The within-participant change from baseline in the composite sum of standardized scores from a set of ADNI neuropsychological test measures, the ADNI Battery Composite (ABC, 9-item). The ABC score is calculated from the following 9 measures from the ADNI-1 Neuropsychological Test Battery: Auditory Verbal Learning Test (AVLT)-Immediate Recall, AVLT-Delayed Recall, Number Span Forward, Number Span Backward, Category Fluency, Trail Making Test A, Trail Making Test B, Digit Symbol Substitution, Boston Naming Test. Standardized scores (Z-scores) were calculated using the overall mean and standard deviation of all participants in the modified intent-to-treat (mITT) population at baseline as reference. The ABC score for each participant was the mean of the standardized subtest values. Range: -3 to 3; higher scores indicate less impairment. | The modified mITT population, including all randomized participants who took at least one dose of the study medication and who have a baseline assessment of the primary endpoint for phase 2B (CDR-SB) | Posted | Least Squares Mean | Standard Error | Z-scores | 72 weeks |
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| Secondary | Phase 2B: Change From Baseline in Quantitative Electroencephalogram (qEEG) | The within-participant change from baseline of global relative theta wave power (4-8 Hz) in qEEG. qEEG was used to assess resting-state brain activity, including global relative theta power (4-8 Hz), based on spectral analysis of signals from 21 electrode positions. Higher theta power indicates greater impairment. | The modified intent-to-treat (mITT) population, including all randomized participants who took at least one dose of the study medication and who have a baseline assessment of the primary endpoint for phase 2B (CDR-SB). Only paired EEGs (at least 1 evaluable Baseline and 1 evaluable Follow-up EEG) were analyzed. | Posted | Least Squares Mean | Standard Error | Proportion (0-1) | 72 weeks |
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| Secondary | Phase 2B: Change From Baseline in Functional Activities Questionnaire (FAQ) | The within-participant change from baseline in FAQ.The FAQ is a validated 10-item questionnaire, completed as a structured interview with the study partner, which rates the study participant's ability to carry out ten complex activities of daily living (doi.org/10.1093/geronj/37.3.323). Each item is rated on a scale from 0 (no impairment) to 3 (dependent). Scores are summed across items to provide a total score. Range: 0 to 30; higher scores indicate greater impairment. | The modified intent-to-treat (mITT) population, including all randomized participants who took at least one dose of the study medication and who have a baseline assessment of the primary endpoint for phase 2B (CDR-SB) | Posted | Least Squares Mean | Standard Error | Total score (0-30) | 72 weeks |
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| Secondary | Phase 2B: Change From Baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-Cog-13) | The within-participant change from baseline in ADAS-Cog-13. The ADAS-Cog-13 is a structured scale that evaluates memory (immediate and delayed word recall; immediate word recognition), receptive and expressive language, orientation, ideational praxis (preparing a letter for mailing), constructional praxis (copying figures), and attention (number cancellation). Ratings of spoken language, language comprehension, word finding difficulty, and ability to remember test instructions also are obtained. The ADAS-Cog total score is calculated by summing the scores of the 13 component subtests. Range: 0-85; higher scores indicate greater impairment. | The modified intent-to-treat (mITT) population, including all randomized participants who took at least one dose of the study medication and who have a baseline assessment of the primary endpoint for phase 2B (CDR-SB) | Posted | Least Squares Mean | Standard Error | Total score (0-85) | 72 weeks |
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| Secondary | Phase 2B: Change From Baseline in Neuropsychiatric Inventory (NPI) | The within-participant change from baseline in NPI. The NPI is a well-validated, reliable, multi-item instrument to assess psychopathology in Alzheimer's disease dementia based on the results of an interview with the study partner. Frequency and severity of 12 neuropsychiatric symptoms are assessed. Range: 0 to 144; higher scores indicate greater impairment. | The modified intent-to-treat (mITT) population, including all randomized participants who took at least one dose of the study medication and who have a baseline assessment of the primary endpoint for phase 2B (CDR-SB) | Posted | Least Squares Mean | Standard Error | Total score (0-144) | 72 weeks |
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| Secondary | Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Rates of All Treatment-emergent Serious Adverse Events (SAEs) | Longer-term safety and tolerability of PQ912 compared between the active and placebo arm as measured by rates of all treatment-emergent SAEs, presented as numbers of participants with at least one SAE and percentages | The safety (SAF) population, including all participants who had received at least one dose of the study medication | Posted | Count of Participants | Participants | 76 weeks |
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| Secondary | Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Suicidality on the Columbia-Suicide Severity Rating Scale (C-SSRS) | Assessment of the longer-term safety and tolerability of PQ912 via the C-SSRS. The C-SSRS is an instrument designed to assess the occurrence, severity, and frequency of suicide-related thoughts and behaviors during the corresponding assessment period. The scale includes suggested questions to elicit the type of information needed to determine if a suicide-related thought or behavior occurred. In this study it was used to 'detect' the appearance of new suicidal ideation or behavior as either "present" or "not present". The tables reflect the total number of participants (and percentage) experiencing Any suicidality, Any suicidal ideation, Any suicidal behavior, and Any self-injurious behavior at screening and subsequent time points. | The safety (SAF) population, including all participants who had received at least one dose of the study medication | Posted | Count of Participants | Participants | Up to 72 weeks |
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| Secondary | Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Mortality Rates | Longer-term safety and tolerability of PQ912 compared between the active and placebo arm as measured by rates of fatal treatment-emergent serious adverse events (SAEs), presented as numbers of participants with a treatment emergent SAE leading to death and percentages. | The safety (SAF) population, including all participants who had received at least one dose of the study medication | Posted | Count of Participants | Participants | 76 weeks |
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| Secondary | Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Rates of All Treatment-emergent Adverse Events (TEAEs) | Longer-term safety and tolerability of PQ912 compared between the active and placebo arm as measured by rates of all TEAEs, presented as numbers of participants with at least one TEAE and percentages | The safety (SAF) population, including all participants who had received at least one dose of the study medication | Posted | Count of Participants | Participants | 76 weeks |
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| Secondary | Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Rates of All Adverse Events of Special Interest (AESIs) | Longer-term safety and tolerability of PQ912 compared between the active and placebo arm as measured by rates of all AESIs presented as numbers of participants with at least one AESI and percentages . | The safety (SAF) population, including all participants who had received at least one dose of the study medication | Posted | Count of Participants | Participants | 76 weeks |
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| Secondary | Phase 2B: Assessment of the Longer-term Safety and Tolerability of PQ912 as Measured by Drug Discontinuation Rates | Longer-term safety and tolerability of PQ912 compared between the active and placebo arm as measured by rates of drug discontinuation due to treatment-emergent adverse events (TEAEs), presented as numbers of participants with a TEAE leading to drug discontinuation and percentages. | The safety (SAF) population, including all participants who had received at least one dose of the study medication | Posted | Count of Participants | Participants | 72 weeks |
|
76 weeks
All participants started at 150 mg BID and were up-titrated to 600 mg BID. As safety data were collected continuously across titration, separation of doses is not possible. Therefore, safety data are reported as one single active Arm/Group. Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | PQ912 | All participants started at 150 mg BID and were up-titrated to 600 mg BID administered orally for up to 72 weeks and therefore, all data from these participants were collected as one single active Arm/Group and separation of doses is not possible. Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B. | 2 | 53 | 11 | 53 | 46 | 53 |
| EG001 | Placebo | All participants received matching placebo BID administered orally for up to 72 weeks Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B. | 0 | 56 | 6 | 56 | 47 | 56 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Metabolic encephalopathy | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Hemorrhage intracranial | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
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| Transient ischaemic attack | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Bacterial sepsis | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Urinary tract infection bacterial | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Urinary tract infection staphylococcal | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
| |
| Squamous cell carcinoma of skin | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (24.1) | Systematic Assessment |
| |
| Adenocarcinoma pancreas | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (24.1) | Systematic Assessment |
| |
| Acute myocardial infarction | Cardiac disorders | MedDRA (24.1) | Systematic Assessment |
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| Angina pectoris | Cardiac disorders | MedDRA (24.1) | Systematic Assessment |
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| Sinus node dysfunction | Cardiac disorders | MedDRA (24.1) | Systematic Assessment |
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| Gastric ulcer perforation | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Adverse drug reaction | General disorders | MedDRA (24.1) | Systematic Assessment |
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| Wound evisceration | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
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| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (24.1) | Systematic Assessment |
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| Ovarian cyst | Reproductive system and breast disorders | MedDRA (24.1) | Systematic Assessment |
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| Metastatic malignant melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (24.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID-19 | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
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| Nasopharyingitis | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
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| Skin laceration | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
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| Skin abrasion | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
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| Depression | Psychiatric disorders | MedDRA (24.1) | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA (24.1) | Systematic Assessment |
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| Blood creatine phosphokinase increased | Investigations | MedDRA (24.1) | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
|
Due to the early termination of the study the intended sample size was not reached and the available sample size for the analysis was limited. However, participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks).
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Vivoryon Therapeutics N.V. | 0049 34555599 | 00 | clinics@vivoryon.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 28, 2024 | Jul 11, 2025 | SAP_001.pdf |
| ICF | No | No | Yes | Informed Consent Form | Jun 14, 2023 | Sep 12, 2025 | ICF_002.pdf |
Not provided
| ID | Term |
|---|---|
| D000544 | Alzheimer Disease |
| D060825 | Cognitive Dysfunction |
| ID | Term |
|---|---|
| D003704 | Dementia |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D024801 | Tauopathies |
| D019636 | Neurodegenerative Diseases |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
| D003072 | Cognition Disorders |
Not provided
Not provided
| Between 18 and 65 years |
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| >=65 years |
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All participants received matching placebo BID administered orally for up to 72 weeks
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| Participants |
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| OG001 | Placebo | All participants received matching placebo BID administered orally for up to 72 weeks. Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B. |
|
|
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| OG001 |
| Placebo |
All participants received matching placebo BID administered orally for up to 72 weeks. Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B. |
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All participants received matching placebo BID administered orally for up to 72 weeks.
Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B.
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All participants received matching placebo BID administered orally for up to 72 weeks. Participants enrolled in Phase 2A remained in the study for seamless inclusion in the Phase 2B evaluation (up to 72 weeks). No new participants were enrolled in Phase 2B. |
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