Evidence map›Paper›PMID 38530629›Full record

ArticleTherapeutic innovation & regulatory science2024

Recent Status of Phase I Clinical Trials for Brain Tumors: A Regulatory Science Study of Exploratory Efficacy Endpoints.

Shinya Watanabe, Takahiro Nonaka, Makoto Maeda, Masanobu Yamada, Narushi Sugii, Koichi Hashimoto, Shingo Takano, Tomoyoshi Koyanagi, Yoshihiro Arakawa, Eiichi Ishikawa

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Therapeutic innovation & regulatory science, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
0.4field-weighted citation impact, top 39% of its field
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

2 citing papers in PubMed, 1 citations in OpenAlex.

  1. Review
  2. Article
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

10 authors at 4 institutions in 1 country.

Shinya WatanabeDepartment of Neurosurgery, Mito Kyodo General Hospital, Tsukuba University Hospital Mito Area Medical Education Center, 3-2-7 Miyamachi, Mito, 310-0015, Ibaraki, Japan. shinya-watanabey@md.tsukuba.ac.jp.ORCID 0000-0002-9953-8665
Takahiro NonakaDepartment of Health and Medical Innovation, Graduate School of Medicine, Osaka Metropolitan University School, Osaka, Japan.
Makoto MaedaDepartment of, Pharmacy, National Cancer Center Hospital, Tokyo, Japan.
Masanobu YamadaTsukuba Clinical Research and Development Organization, University of Tsukuba, Tsukuba, Japan.
Narushi SugiiDepartment of Neurosurgery, University of Tsukuba Hospital, Tsukuba, Japan.
Koichi HashimotoTsukuba Clinical Research and Development Organization, University of Tsukuba, Tsukuba, Japan.
Shingo TakanoDepartment of Neurosurgery, University of Tsukuba Hospital, Tsukuba, Japan.
Tomoyoshi KoyanagiTsukuba Clinical Research and Development Organization, University of Tsukuba, Tsukuba, Japan.
Yoshihiro ArakawaTsukuba Clinical Research and Development Organization, University of Tsukuba, Tsukuba, Japan.
Eiichi IshikawaDepartment of Neurosurgery, Institute of Medicine, University of Tsukuba, Tsukuba, Japan.
University of Tsukuba · JPOsaka Metropolitan University · JPTokyo National Hospital · JPUniversity of Tsukuba Hospital · JP

Funding

Japan Agency for Medical Research and Development (AMED) JP23mk0101235
6 · The paper itself

Abstract

backgroundAppropriate exploratory efficacy data from Phase I trials are vital for subsequent phases. Owing to the uniqueness of brain tumors (BTs), use of different strategies to evaluate efficacy is warranted. We studied exploratory efficacy evaluation in Phase I trials involving BTs.

methodsUsing Clarivate's Cortellis

resultsSelected studies included a median of 1.5 PEs (range, 1-6) and 5 SEs (range, 0-19). Efficacy endpoints were included as PEs and SEs in 2 (5%) and 31 (78%) trials, respectively. Among the latter 31 trials that included 94 efficacy endpoints, 24, 22, 20, 9, and 8 reflected overall response rate (ORR), progression-free survival (PFS), overall survival (OS), duration of response (DOR), and disease control rate (DCR), respectively. ORR for BT was determined using various methods; however, the Response Evaluation Criteria in Solid Tumors (RECIST) was used less frequently in Group-A than in Group-B (p = 0.0039).

conclusionsRecent Phase I trials included efficacy endpoints as SEs, with ORR, PFS, or OS included in ~ 50% trials and DOR or DCR in ~ 25%. No established criteria exist for imaging evaluation of BTs. Phase I trials involving mixed solid tumor cohorts revealed challenges in designing methods to assess the exploratory efficacy of BTs.

Indexed as

Brain NeoplasmsClinical Trials, Phase I as TopicEndpoint DeterminationHumansProgression-Free SurvivalTreatment OutcomeBrain tumorsEfficacy endpointGlioblastomaMeningiomaPhase I clinical trialRegulatory science

Identifiers

PMID38530629
PMCPMC11169007
OpenAlexW4393190311

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.