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New directions in clinical trials for frontotemporal lobar degeneration: Methods and outcome measures
Boxer AL, Gold M, Feldman H, Boeve BF, Dickinson SL, Fillit H, Ho C, Paul R et al.. Alzheimer's & dementia : the journal of the Alzheimer's Association, 2020-01-06
Topic: Risk, prevention and care in population studies
Introduction Frontotemporal lobar degeneration (FTLD) is the most common form of dementia for those under 60 years of age. Increasing numbers of therapeutics targeting FTLD syndromes are being developed. Methods In March 2018, the Association for Frontotemporal Degeneration convened the Frontotemporal Degeneration Study Group meeting in Washington, DC, to discuss advances in the clinical science of FTLD. Results Challenges exist for conducting clinical trials in FTLD. Two of the greatest challenges are (1) the heterogeneity of FTLD syndromes leading to difficulties in efficiently measuring treatment effects and (2) the rarity of FTLD disorders leading to recruitment challenges. Discussion New personalized endpoints that are clinically meaningful to individuals and their families should be developed. Personalized approaches to analyzing MRI data, development of new fluid biomarkers and wearable technologies will help to improve the power to detect treatment effects in FTLD clinical trials and enable new, clinical trial designs, possibly leveraged from the experience of oncology trials. A computational visualization and analysis platform that can support novel analyses of combined clinical, genetic, imaging, biomarker data with other novel modalities will be critical to the success of these endeavors.
Key findings
- Results Challenges exist for conducting clinical trials in FTLD.
Why it matters
A 2018 Association for Frontotemporal Degeneration meeting report naming two main obstacles to drug trials in frontotemporal lobar degeneration (FTLD): the condition varies so much between patients that treatment effects are hard to measure, and it is rare enough that recruitment is hard. It calls for endpoints that are meaningful to patients and families, and suggests personalised MRI analysis, new fluid biomarkers and wearable devices to make trials more sensitive. These are proposals, not tested results, and the report is about FTLD trial methods, not Alzheimer's treatment.
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Study details
- Evidence strengthLow
How this study scored 48/100
Published in a leading specialty journal. Scored zero for study design, sample size, effect reported, studied in people and randomised / blinded because the record does not state it.
| Component | Points | Why |
|---|---|---|
| Journal tier | 9 / 10 | Tier 2 — leading specialty journal (Alzheimer's & dementia : the journal of the Alzheimer's Association) |
| Journal's citation record | 2 / 4 | Alzheimer s & Dementia: journal h-index 228 (OpenAlex, 2026-10-29) |
| Study design | 0 / 12 | No study design recorded — scores zero, not guessed (not stated in the record — scored 0) |
| Strength of evidence | 1 / 5 | Rated “low” in our summary |
| Peer-review validation | 10 / 10 | Peer-reviewed. Published as an article in a named peer-reviewed journal. |
| How recent | 2 / 5 | 2020 — within 10 year(s) of the newest study here |
| Sample size | 0 / 4 | Participant count not stated — scores zero (not stated in the record — scored 0) |
| Effect reported | 0 / 3 | No effect size reported — scores zero (not stated in the record — scored 0) |
| Studied in people | 0 / 3 | Not stated whether this was studied in people — scores zero (not stated in the record — scored 0) |
| Randomised / blinded | 0 / 2 | No randomisation or blinding stated — scores zero (not stated in the record — scored 0) |
| Times cited | 4 / 7 | Cited 71 times (OpenAlex, 2026-10-10) |
| Citations per year | 3 / 5 | 8.9 citations a year over 8 year(s) since 2020 |
| Impact for its field and year | 3 / 6 | Cited 6.3× the average for papers of its field and year (OpenAlex FWCI) |
| Cited by studies here | 2 / 6 | Cited by 6 other studies in this library |
| Still being cited | 2 / 4 | Cited 23 times in 2024–2026 (2026 still in progress; OpenAlex) |
| Senior author's track record | 2 / 4 | Last-listed author Nadine A. Tatton: h-index 31 (OpenAlex, whole career) |
| Institution's track record | 3 / 3 | Harvard University: institution h-index 3,235 (OpenAlex) |
| Multi-centre collaboration | 3 / 3 | OpenAlex lists 29 institution(s) in 3 countries |
| Indexed in PubMed | 2 / 2 | Indexed in PubMed (PMID 31668596) |
| Free to read | 0 / 2 | Behind a paywall — scores zero |
| Total | 48 / 100 | — |
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Read the original
- DOI: 10.1016/j.jalz.2019.06.4956
- PubMed: 31668596
- Original source →
- Free full text: PubMed Central →
Full text: free to read — OpenAlex lists this copy as free to read: the link above (checked 6 Feb 1020).
Cite this study APA, Vancouver, BibTeX · download .bib or .ris
Built from the record alzhub holds for this paper, not looked up. Volume, issue and page numbers are not held in the record. The record's author list ends in “et al.”, so not every author is named. Check the citation against the original before you use it.
APA 7th
Boxer, A. L., Gold, M., Feldman, H., Boeve, B. F., Dickinson, S. L., Fillit, H., et al. (2020). New directions in clinical trials for frontotemporal lobar degeneration: Methods and outcome measures. Alzheimer's & dementia : the journal of the Alzheimer's Association. https://doi.org/10.1016/j.jalz.2019.06.4956
Vancouver
Boxer AL, Gold M, Feldman H, Boeve BF, Dickinson SL, Fillit H, et al. New directions in clinical trials for frontotemporal lobar degeneration: Methods and outcome measures. Alzheimer's & dementia : the journal of the Alzheimer's Association. 2020. doi:10.1016/j.jalz.2019.06.4956. PMID: 31668596.
BibTeX
@article{boxer2020-31668596,
author = {Boxer, A. L. and Gold, M. and Feldman, H. and Boeve, B. F. and Dickinson, S. L. and Fillit, H. and Ho, C. and Paul, R. and others},
title = {{New directions in clinical trials for frontotemporal lobar degeneration: Methods and outcome measures}},
journal = {Alzheimer's \& dementia : the journal of the Alzheimer's Association},
year = {2020},
doi = {10.1016/j.jalz.2019.06.4956},
pmid = {31668596},
url = {https://doi.org/10.1016/j.jalz.2019.06.4956}
}
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