P3‐309: Advancing research and treatment of frontotemporal lobar degeneration (ARTFL): Preparing for clinical trials for ftld in north america
Bibliographic record
Abstract
The objective was to prepare for clinical trials of disease-modifying agents for frontotemporal lobar degeneration (FTLD) by building a network of investigators, recruitment tools, biomarker measurements, and training opportunities throughout North America. New treatments that target specific molecules (and genes) underlying FTLD including tau (MAPT), progranulin (GRN), TDP-43 (TARBP) and chromosome 9 open reading frame 72 (C9ORF72) are rapidly entering human clinical trials. Only a few randomized placebo controlled trials have ever been completed in FTLD, and little infrastructure exists to support new studies, especially trials defined based on molecular pathology as compared to clinical syndrome. Investigators at 14 centers pooled preliminary data regarding patient populations, potential enrollment, and barriers to clinical trial participation. We developed strategies for evaluating subjects using clinical measures and novel neuropsychological batteries, including the new FTLD module from the National Alzheimer's Coordinating Center (NACC) Uniform Data Set (UDS) and tests from a NINDS funded executive function battery (EXAMINER). We identified a need for funding investigator training and pilot projects focused on biomarker development. Two research projects funded through the NINDS and the National Center for Advancing Clinical and Translational Science (NCATS) Rare Disease Research Consortium Network. ARTFL is a partnership between academic investigators and patient advocacy groups including the AFTD, Cure PSP, Bluefield Project, Tau Consortium, CBD Solutions, and ADDF. One project will develop an online research registry and evaluates patients with sporadic FTLD syndromes with predictable underlying pathologies (progressive supranuclear palsy, semantic variant primary progressive aphasia, and frontotemporal dementia with amyotrophic lateral sclerosis) in order to identify clinical and biomarker measures to facilitate clinical trials. Novel neuropsychological batteries, genotyping, and clinical assessments will be performed in 650 participants. The second project focuses on longitudinal evaluation of both symptomatic and asymptomatic familial FTLD cases, using the same assessments and neuroimaging in 910 participants. The first pilot project focuses on AV1451 tau PET imaging in FTLD. Sites have IRB approval and will begin enrollment in spring 2015. Data will be publicly available to interested investigators. The data, recruitment tools, biomarkers, and training opportunities developed by ARTFL will facilitate new clinical trials in FTLD.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.037 | 0.010 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.003 | 0.002 |
| Scholarly communication | 0.003 | 0.003 |
| Open science | 0.002 | 0.003 |
| Research integrity | 0.005 | 0.005 |
| Insufficient payload (model declined to judge) | 0.010 | 0.004 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".