Observational studyStatistics in medicine2018
A novel cognitive disease progression model for clinical trials in autosomal-dominant Alzheimer's disease.
Observational study in Statistics in medicine, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 4 registered trials, which are not on this map. Cited by 38 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase II/III Multicenter Randomized, Double-Blind, Placebo-Controlled Platform Trial of Potential Disease Modifying Therapies Utilizing Biomarker, Cognitive, and Clinical Endpoints in Dominantly Inherited Alzheimer's Disease
A Phase II/III Randomized, Double-Blind, Placebo-Controlled, Cognitive Endpoint, Multi-Center Study of Potential Disease Modifying Therapies in Individuals at Risk for and With Dominantly Inherited Alzheimer's Disease
A Phase II/III Multicenter Randomized, Double-Blind, Placebo-Controlled, Two-Stage Adaptive Design, Platform Trial of Investigational Treatments for Primary Prevention of Disease Progression in Dominantly Inherited Alzheimer's Disease
A Phase II/III Multicenter Randomized, Double-Blind, Placebo-Controlled Platform Trial of Potential Disease Modifying Therapies Utilizing Biomarker, Cognitive, and Clinical Endpoints in Dominantly Inherited Alzheimer's Disease
Who cites it
38 citing papers in PubMed.
- The relationship of soluble tau species with Alzheimer's disease amyloid plaque removal and tau pathology.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2025Trial
- Assessment of Efficacy and Safety of Zagotenemab: Results From PERISCOPE-ALZ, a Phase 2 Study in Early Symptomatic Alzheimer Disease.Neurology · 2024Trial
- Consistency of efficacy results across various clinical measures and statistical methods in the lecanemab phase 2 trial of early Alzheimer's disease.Alzheimer's research & therapy · 2022Trial
- A trial of gantenerumab or solanezumab in dominantly inherited Alzheimer's disease.Nature medicine · 2021Trial
- Statistical models for Alzheimer's disease clinical trials: Lessons learned from the DIAN-TU Platform Trial.Journal of Alzheimer's disease : JAD · 2026Article
- 15 years of longitudinal genetic, clinical, cognitive, imaging, and biochemical measures in DIAN.NPJ dementia · 2026Article
- Design considerations for C9orf72 disease prevention trials.Brain : a journal of neurology · 2025Review
- Estimand Endpoints for Longitudinal Measures of Continuous Disease Progression with an Alzheimer's Disease Example.Therapeutic innovation & regulatory science · 2025Article
- Clinical progression on CDR-SB©: Progression-free time at each 0.5 unit level in dominantly inherited and sporadic Alzheimer's disease populations.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2025Article
- The landscape of autosomal-dominant Alzheimer's disease: global distribution and age of onset.Brain : a journal of neurology · 2025Article
- Sample size estimates for biomarker-based outcome measures in clinical trials in autosomal dominant Alzheimer's disease.The journal of prevention of Alzheimer's disease · 2025Article
- Comments on "Novel Non-Linear Models for Clinical Trial Analysis With Longitudinal Data: A Tutorial Using SAS for Both Frequentist and Bayesian Methods".Statistics in medicine · 2025Article
- Longitudinal associations between self-reported exercise levels and cognition in ADAD.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2025Article
- Disease progression trajectory curves to estimate saved time in Alzheimer's disease trials.Contemporary clinical trials · 2025Article
- The Potential of Disease Progression Modeling to Advance Clinical Development and Decision Making.Clinical pharmacology and therapeutics · 2025Review
- Clinical trials for Wolfram syndrome neurodegeneration: Novel design, endpoints, and analysis models.PloS one · 2025Article
- Unlocking Cognitive Analysis Potential in Alzheimer's Disease Clinical Trials: Investigating Hierarchical Linear Models for Analyzing Novel Measurement Burst Design Data.Statistics in medicine · 2024Article
- A framework for translating tauopathy therapeutics: Drug discovery to clinical trials.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2024Review
- Statistical considerations when estimating time-saving treatment effects in Alzheimer's disease clinical trials.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2024Article
- Novel non-linear models for clinical trial analysis with longitudinal data: A tutorial using SAS for both frequentist and Bayesian methods.Statistics in medicine · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors.
Funding
Abstract
Clinical trial outcomes for Alzheimer's disease are typically analyzed by using the mixed model for repeated measures (MMRM) or similar models that compare an efficacy scale change from baseline between treatment arms with or without participants' disease stage as a covariate. The MMRM focuses on a single-point fixed follow-up duration regardless of the exposure for each participant. In contrast to these typical models, we have developed a novel semiparametric cognitive disease progression model (DPM) for autosomal dominant Alzheimer's disease based on the Dominantly Inherited Alzheimer Network (DIAN) observational study. This model includes 3 novel features, in which the DPM (1) aligns and compares participants by disease stage, (2) uses a proportional treatment effect similar to the concept of the Cox proportional hazard ratio, and (3) incorporates extended follow-up data from participants with different follow-up durations using all data until last participant visit. We present the DPM model developed by using the DIAN observational study data and demonstrate through simulation that the cognitive DPM used in hypothetical intervention clinical trials produces substantial gains in power compared with the MMRM.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.