NCT04801966

Safety and Oversight of the Individually Tailored Treatment Approach: A Novel Pilot Study

• Status: Terminated
• Phase: N/A
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Peter MacCallum Cancer Centre, Australia · Academic / Other
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

This study is looking at outcomes in people with advanced cancers who have exhausted standard treatment options and are accessing off indication or unregistered drugs or combinations of drugs through compassionate access from the manufacturer.

Detailed description

Some advanced cancers have numerous standard treatment options that have proven efficacy in clinical trials. However, in other cancers, there may be few or no standard treatment options with proven efficacy as determined in a large clinical trial. This may be particularly the case for rare cancers in which there is a lack of clinical research. When seriously ill patients run out of standard treatment options, they will often consider non-standard treatment options (such as treatments that are currently unapproved by the regulatory agency for the given indication). The majority of clinicians and researchers agree that this is best received in a clinical trial setting as this provides ethical and clinical oversight, as well as addresses prospectively defined research questions which can be publicly reported. This allows the conclusions of the research to be available to the entire clinical and research community. In general, an access program enables patient access to a non-reimbursed therapeutic agent, outside of a clinical trial setting. Compassionate access is typically for therapeutics that are not yet approved or TGA registered, and are still considered investigational. In general, there is a negotiation between the pharmaceutical company and the clinician and patient regarding access to the therapeutic agent, as well as whether the medicine will be provided free of charge, or on some form of cost-sharing arrangement. In Australia, access to TGA non-registered medicines also requires an application via the "Special Access Scheme". For most cancer patients, the use falls under category A, for a patient defined as seriously ill. This sub-study generally pertains to compassionate access to therapeutic agents. Given the ad hoc nature of compassionate access for patients, there is relatively little reported data on clinical outcomes. Compassionate access is an established process with increasing demands. This study is designed to provide a framework for which patients treated with compassionate access therapeutics can register, so that some of the limitations of ad hoc compassionate access programs can be overcome. A study committee will prospectively assess each individual patient's detailed treatment approach in an objective and time-efficient manner. If approved, the patient may be eligible to register into the treatment phase of the study. The study committee is essential to provide a balanced approach to understanding the rationale for the study treatment, as well as potential safety issues that may arise. As previously reported, this is an essential component to improving patient oversight as well as equity

Conditions

• Cancer

Interventions

Type	Name	Description
DRUG	Trametinib	2 mg per day, continuous
DRUG	Cobimetinib	60 mg/day for 21 days of a 28 day cycle
DRUG	Binimetinib	45 mg/twice a day, continuous
DRUG	Alpelisib	300 mg/day, continuous
DRUG	Vemurafenib	960 mg/twice per day, continuous
DRUG	Dabrafenib	150 mg/twicce per day, continuous
DRUG	Encorafenib	450 mg/day, continuous
DRUG	Palbociclib	125 mg/day, day 1-21 of a 28 day cycle
DRUG	Olaparib	300 mg/twice per day, continuous
DRUG	Ribociclib	600 mg/day, on day 1 -21 of a 28 day cycle
DRUG	Abemaciclib	150 mg twice/day, continuous
DRUG	Talazoparib	1 mg/day, on day 1 -28 of each 28 day cycle
DRUG	Nivolumab	240 mg IV once every 2 weeks
DRUG	Atezolizumab	1200 mg IV on Day 1 of a 21 day cycle
DRUG	Pembrolizumab	200 mg IV on day 1 of a 21 day cycle

Timeline

• Start date: 2021-09-23
• Primary completion: 2022-12-30
• Completion: 2022-12-30
• First posted: 2021-03-17
• Last updated: 2023-05-10

Locations

1 site across 1 country: Australia