Related Experiment Video
Updated: Aug 15, 2025
A Simple Guide Screw Method for Intracranial Xenograft Studies in Mice
Published on: September 26, 2011
Efficacy Endpoints in Phase II Clinical Trials for Meningioma: An Analysis of Recent Clinical Trials
Shinya Watanabe1,2, Takahiro Nonaka3, Makoto Maeda4
1Department of Neurosurgery, Mito Kyodo General Hospital, Tsukuba University Hospital Mito Area Medical Education Center, 3-2-7 Miyamachi, Mito, Ibaraki, 310-0015, Japan. shinya-watanabey@md.tsukuba.ac.jp.
Background:
Response Evaluation Criteria in Solid Tumors (RECIST)-based response rates are commonly used as efficacy endpoints in phase II clinical trials for solid tumors. However, no consensus has been reached concerning adequate efficacy endpoints for phase II clinical trials targeting meningioma. Irregularity of lesions after resection, and varying degrees of dysplasia and histologic subtypes make establishing an appropriate efficacy evaluation difficult.
Methods:
We analyzed primary efficacy endpoints (PEEs) and background factors from 48 trials retrieved from ClinicalTrials.gov ( https://clinicaltrials.gov/ ) using the search criteria "meningioma," "interventional," "phase II," and "study start 4/1/2001 to 3/31/2021." Primary purpose of the study was efficacy endpoint setting in overall population and three subgroups.
Results:
Among 45 PEEs set in the 39 trials included; 33 trials with single PEE, and six trials with double PEEs, 17/45 (38%) trials adopted progression-free survival (PFS) rate, 15/45 (33%) trials response rate (seven Macdonald criteria or modified, three RECIST, three volumetric estimation, one RANO criteria, one unknown), 10/45 (22%) PFS, 1/45 (2%) OS, and 2/45 (4%) other endpoints. Although 26 PEEs were time-to-event endpoints, 19 of the 26 PEEs were single-arm studies.
Conclusions:
Time-to-event efficacy endpoints were often compared to historical data, and two-dimensional evaluation is more suitable than one-dimensional one. Accumulation of prognostic data is essential to standardize time-to-event efficacy endpoints. Considering the difficulty of setting design for phase II clinical studies targeting meningioma, evaluation might be done with multiple efficacy endpoints.
Insights
Establishing efficacy endpoints for meningioma clinical trials is challenging. Progression-free survival and response rates are common, but standardized time-to-event evaluations and multiple endpoints may be necessary.
Area of Science:
- Neuro-oncology
- Clinical Trial Design
- Cancer Research
Background:
- Standardized efficacy endpoints like RECIST are common in solid tumor trials but lack consensus for meningioma.
- Challenges in meningioma trials include irregular post-resection lesions and variable histology.
- Defining appropriate efficacy endpoints for meningioma phase II trials remains difficult.
Purpose of the Study:
- To analyze primary efficacy endpoints (PEEs) used in phase II meningioma clinical trials.
- To identify trends and challenges in setting efficacy endpoints for meningioma studies.
- To provide insights for establishing standardized efficacy evaluation in meningioma research.
Main Methods:
- Systematic review of 48 interventional phase II meningioma trials from ClinicalTrials.gov (2001-2021).
- Analysis of primary efficacy endpoints and background factors across selected trials.
- Categorization of endpoints including progression-free survival, response rates, and overall survival.
Main Results:
- Progression-free survival (38%) and response rates (33%) were the most common primary efficacy endpoints among 45 PEEs in 39 trials.
- Response rate criteria included Macdonald, RECIST, volumetric, and RANO.
- Time-to-event endpoints (26 PEEs) were frequently used, often in single-arm studies (19/26).
Conclusions:
- Time-to-event endpoints are often compared to historical data; two-dimensional evaluation is preferred over one-dimensional.
- Standardizing time-to-event endpoints requires accumulating prognostic data.
- Given the complexities, utilizing multiple efficacy endpoints may be beneficial for meningioma phase II trials.
Related Concept Videos
01:11Clinical Trials: Overview
01:16Clinical Trials
There are four phases in a clinical trial. A phase one...