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Clinical Trial Summary

This is a Phase I Clinical Trial. Phase I studies are designed to determine the amount of investigational drugs that can be safely tolerated and to define the side effects that limit the dose. The drug administered in this study is KML-001. It is a highly soluble, orally available arsenic agent. It is currently being tested to determine its effects on telomerase activity.

In other words, the purpose of this research study is to find the highest dose of KML001, that can be given without causing severe side effects when it is combined with a standard, commercially available anti-cancer drug called cisplatin.


Clinical Trial Description

Telomerase is the enzyme synthesizing the specific DNA sequences found at the telomeres in the body's chromosomes. It is thus responsible for maintaining the length of telomeres. Telomerase has been detected in human cancer cells and is found to be 10-20 times more active than in normal body cells. This provides a selective growth advantage to many types of tumors. If telomerase activity was to be turned off, then telomeres in cancer cells would shorten, just like they do in normal body cells. This would prevent the cancer cells from dividing uncontrollably in their early stages of development. In the event that a tumor has already thoroughly developed, it may be removed and anti-telomerase therapy could be administered to prevent relapse.

This study is being offered to patients with advanced cancer which has either no standard therapy or which has progressed after treatment with one or more standard treatments.

The primary objective of this study :

To determine the maximum tolerated dose of KML001 in combination with cisplatin in patients with advanced malignancy. This objective has been met. The study will be reopened with expansion cohorts in advanced small cell lung cancer and non-small cell lung cancer to better assess the activity of the combination, pending (Institutional Review Board (IRB) approval.

Secondary Objectives of the study:

To determine the pharmacokinetics of KML001 and cisplatin in this combination. To assess the response rate, disease-free survival and survival associated with this regimen.

To correlate indications of patient benefit (response or stable disease) with pretreatment specimens

The highest safest doses are determined by increasing the doses of cisplatin and KML001 in successive groups of patients until at least some of them have serious side effects. All patients on this study will receive the same dose of cisplatin, which is known to have antitumor effects. The doses of KML001 will be increased in successive groups of patients. It is possible that those entering the study early may receive suboptimal doses of KML001. At the end of the study we hope to determine the appropriate dose of the KML001 in combination with cisplatin, learn about its side effects and understand how the body metabolizes the drug.

Laboratory data from the University of Maryland Greenebaum Cancer Center (UMGCC) has demonstrated that the combination of KML001 and cisplatin is synergistic in lung cancer cell lines. Cisplatin is the best established agent for the treatment of lung cancer and most treatment regimens have been established with cisplatin (or its congener, carboplatin). This synergism is particularly interesting given that there is an anti-telomere effect for cisplatin. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT01110226
Study type Interventional
Source University of Maryland, Baltimore
Contact
Status Terminated
Phase Phase 1
Start date April 27, 2010
Completion date October 27, 2015