ReviewCancer journal (Sudbury, Mass.)
Phase 0 clinical trials: conceptions and misconceptions.
Review in Cancer journal (Sudbury, Mass.). The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 papers.
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.
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.
Who cites it
48 citing papers in PubMed, 107 citations in OpenAlex.
- A Phase 0 Trial of Ceritinib in Patients with Brain Metastases and Recurrent Glioblastoma.Clinical cancer research : an official journal of the American Association for Cancer Research · 2022Trial
- Safety and Efficacy of Intratumoral Injections of Chimeric Antigen Receptor (CAR) T Cells in Metastatic Breast Cancer.Cancer immunology research · 2017Trial
- Multi-omics-driven precision medicine.iMeta · 2026Review
- Safety, Tolerability, and Gut Microbiota Impact of Sericin-Derived Oligopeptides (SDOs) from Yellow Silk Cocoons in Healthy Adults: A Randomized, Double-Blind, Placebo-Controlled Trial.Foods (Basel, Switzerland) · 2026Article
- Adoptive T-cell therapies in the clinic.Bioengineering & translational medicine · 2026Review
- Safety and efficacy of a STAT3-targeted cyclic oligonucleotide: From murine models to a phase 1 clinical trial in pet cats with oral cancer.Cancer cell · 2025Article
- An Interventional Radiology Method for In Situ Assessments of Cancer Drug Response: Preclinical Development, Feasibility, and Safety.Radiology. Imaging cancer · 2025Article
- First-in-Human Study of ABY-029, a Novel Fluorescent Peptide that Targets EGFR, Applied to Soft-Tissue Sarcomas.Molecular cancer therapeutics · 2025Article
- Deciphering normal and cancer stem cell niches by spatial transcriptomics: opportunities and challenges.Genes & development · 2025Review
- In Vivo Interactions of Nucleic Acid Nanostructures With Cells.Advanced materials (Deerfield Beach, Fla.) · 2025Review
- Alternatives to animal models to study bacterial infections.Folia microbiologica · 2023Review
- The Future Glioblastoma Clinical Trials Landscape: Early Phase 0, Window of Opportunity, and Adaptive Phase I-III Studies.Current oncology reports · 2023Review
- Review
- Practical Considerations for Next-Generation Adjuvant Development and Translation.Pharmaceutics · 2023Review
- Article
- Strategic, feasibility, economic, and cultural aspects of phase 0 approaches: Is it time to change the drug development process in order to increase productivity?Clinical and translational science · 2022Review
- Translation and validation of the greek version of a questionnaire measuring patient views on participation in clinical trials.BMC health services research · 2021Article
- Leptomeningeal Metastases from Solid Tumors: Recent Advances in Diagnosis and Molecular Approaches.Cancers · 2021Review
- Phase 0 and window of opportunity clinical trial design in neuro-oncology: a RANO review.Neuro-oncology · 2020Article
- A phase 0 analysis of ixazomib in patients with glioblastoma.Molecular and clinical oncology · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
17 authors at 3 institutions in 1 country.
Funding
Abstract
Phase 0 clinical trials, developed in response to the United States Food and Drug Administration (FDA)'s recent exploratory Investigational New Drug (IND) guidance, are intended to expedite the clinical evaluation of new molecular entities. The exploratory IND supports the performance of first-in-human testing of new investigational agents at subtherapeutic doses based on reduced manufacturing and toxicologic requirements, allowing the demonstration of drug-target effects and assessment of pharmacokinetic-pharmacodynamic relationships in humans earlier in clinical development. The objectives of a phase 0 cancer clinical trial are to establish at the very earliest opportunity-before large numbers of patients have been accrued and exposed to potential drug-associated toxicity-whether an agent is modulating its target in a tumor, and consequently whether further clinical development is warranted. We review here the fundamental requirements of clinical studies conducted under an exploratory IND and address some common misconceptions regarding oncologic phase 0 trials.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.