Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2019 Feb;8(2):585-592.
doi: 10.1002/cam4.1958. Epub 2019 Jan 10.

Phase I and phase II clinical trials in sarcoma: Implications for drug discovery and development

Daniel Y Lee  1 , Arthur P Staddon  2 , Jacob E Shabason  3 , Ronnie Sebro  1   4   5   6
Affiliations
Review

Phase I and phase II clinical trials in sarcoma: Implications for drug discovery and development

Daniel Y Lee et al. Cancer Med. 2019 Feb.

Abstract

Background: There has been limited progress in the development of novel therapeutics for the treatment of sarcomas. A review of phase I and II clinical trials for sarcomas may give insight into factors influencing sarcoma drug development.

Methods: An exhaustive analysis of phase I and II clinical trials testing drugs for human sarcoma patients between 1 January 2000 and 1 June 2018 was performed using the PubMed search engine, the Thomson Web of Science, and the National Clinical Trials registry. Recorded outcomes included tested drugs, tested histological subtypes, whether the drug was initially developed for sarcoma, reported funding sources, and whether studies led to phase III trials.

Results: Out of 238 studies meeting inclusion criteria, 87% (207 studies) reported funding sources. Of these, 59.9% (124/207) reported industry funding, 52.7% (109/207) reported government funding, and 27.5% (57/207) reported private funding. Only 5% (12/238) of phase I and II trials resulted in phase III trials, with 11 of 12 studies funded by industry. Approximately 90% (214/238) of studies tested drugs that were not initially tested in sarcoma, and 60.1% (143/238) of studies grouped different sarcoma histological subtypes together in the same study.

Conclusion: Industry has funded the majority of phase I and II sarcoma clinical trials that have led to phase III trials. There was a high rate of drugs approved for other cancers and then secondarily tested in sarcoma. Most trials tended to group different sarcoma subtypes rather than studying each subtype separately.

Keywords: clinical trial.

PubMed Disclaimer

Figures

Figure 1
Figure 1
PRISMA diagram demonstrating selection of papers in this review of phase I and phase II sarcoma clinical trials
Figure 2
Figure 2
Distribution of funding sources for (A) all clinical trials and (B) studies leading to phase III trials
See this image and copyright information in PMC

References

    1. Burningham Z, Hashibe M, Spector L, Schiffman JD. The epidemiology of sarcoma. Clin Sarcoma Res. 2012;2:14. - PMC - PubMed
    1. Hui J. Epidemiology and etiology of sarcomas. Surg Clin North Am. 2016;96(5):901‐914. - PubMed
    1. Mirabello L, Troisi RJ, Savage SA. Osteosarcoma incidence and survival rates from 1973 to 2004: data from the surveillance, epidemiology, and end results program. Cancer. 2009;115(7):1531‐1543. - PMC - PubMed
    1. Sarcoma, Soft Tissue: Statistics. Cancer. Net. https://www.cancer.net/cancer-types/sarcoma-soft-tissue/statistics. Published June 25, 2012. Accessed August 2, 2018.
    1. Savina M, Le Cesne A, Blay J‐Y, et al. Patterns of care and outcomes of patients with METAstatic soft tissue SARComa in a real‐life setting: the METASARC observational study. BMC Med. 2017;15:78. - PMC - PubMed

Publication types