Evidence map›Paper›PMID 25776824›Full record

ReviewNature reviews. Neurology2015

The promise of futility trials in neurological diseases.

Marcus W Koch, Lawrence Korngut, David G Patry, Yahya Agha-Khani, Christopher White, Justyna R Sarna, Michael Yeung, V Wee Yong, Daniel Y C Heng, Gary Cutter and 1 more

Abstract readReview
PubMed Publisher
In one paragraph

Review in Nature reviews. Neurology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

12 citing papers in PubMed.

  1. An Online Weight Loss Intervention for People With Obesity and Poorly Controlled Asthma.The journal of allergy and clinical immunology. In practice · 2022
    Trial
  2. Trial
  3. Trial
  4. Article
  5. Feasibility of Simon Two-Stage Futility Trials in People with Early, Symptomatically Treated Parkinson's Disease.Movement disorders : official journal of the Movement Disorder Society · 2026
    Article
  6. Review
  7. Improving the efficiency of clinical trials in multiple sclerosis.Multiple sclerosis (Houndmills, Basingstoke, England) · 2023
    Review
  8. Review
  9. Article
  10. Review
  11. The futility study--Progress over the last decade.Contemporary clinical trials · 2015
    Article
  12. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors.

Marcus W Koch1] Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada. [2] Department of Community Health Sciences, TRW Building, University of Calgary, 3280 Hospital Drive NW, Calgary, AB T2N 4Z6, Canada.
Lawrence KorngutDepartment of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.
David G PatryDepartment of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.
Yahya Agha-KhaniDepartment of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.
Christopher WhiteDepartment of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.
Justyna R SarnaDepartment of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.
Michael YeungDepartment of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.
V Wee YongDepartment of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.
Daniel Y C HengDepartment of Oncology, Tom Baker Cancer Centre, 1331 29th Street NW, Calgary, AB T2N 4N2, Canada.
Gary CutterDepartment of Biostatistics, School of Public Health, University of Alabama at Birmingham, 1665 University Boulevard, Birmingham, AL 35294-0022, USA.
Luanne MetzDepartment of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Double-blinded randomized controlled trials (RCTs) have contributed much important evidence to guide treatment decisions in neurology. RCTs are relatively straightforward to conduct, provided that they investigate common diseases, have clearly defined outcome measures, and are of short duration. In neurology, however, many diseases are uncommon, have no consensus outcome measures, and develop over decades. Basic research into neurological diseases continues to identify candidate therapies faster than they can be tested for their clinical utility, leading to a 'translational gap'. Futility trials were initially developed in oncology to efficiently test candidate therapies in phase II trials. As single-arm unblinded studies, futility trials are relatively easy to conduct, and they generally require smaller sample sizes than RCTs. In this article, we discuss futility models, highlighting their advantages as well as challenges to their application in several neurological diseases, including Parkinson disease, stroke and multiple sclerosis.

Indexed as

Clinical Trials, Phase II as TopicCognition DisordersHumansMedical FutilityModels, BiologicalNervous System DiseasesRandomized Controlled Trials as TopicSample Size

Identifiers

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.