~0 spots leftby May 2025

Targeted Therapy for Advanced Rare Cancers

(PREDiCTc Trial)

Recruiting in Palo Alto (17 mi)
Dr. Cheryl Ho - Division of Medical ...
Overseen byCheryl Ho, MD
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Academic
Recruiting
Sponsor: British Columbia Cancer Agency
Must be taking: Targeted therapy
Disqualifiers: Other trials, Unfit, others
No Placebo Group
Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

This pilot clinical trial aims to assess the real world quality of life and survival of patients treated with targeted therapy that has preliminary evidence of efficacy in subjects with advanced rare cancers or cancer harbouring rare molecular aberrations. The treatment has been granted conditional or full approved by Health Canada (HC) as effective and safe. Due to the rarity of the cancer or molecular aberration the uncertainty level of the health technology assessment (HTA) by the pan Canadian Oncology Review (pCODR) was too high for consideration of funding or it was not submitted for consideration. Consequently, the goal of this study is to generate real world evidence to support HTA decision making throughout the life cycle of the product.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it requires that you are currently receiving a specific targeted therapy approved by Health Canada. You cannot participate if you've been treated with another agent or in another trial within 14 days before joining this one.

What data supports the effectiveness of the targeted therapy for advanced rare cancers?

Research shows that using targeted therapies based on specific genetic changes in rare cancers can be beneficial. In one study, 38% of patients with rare tumors had genetic changes that could be targeted with specific drugs, and some patients experienced positive responses, such as tumor shrinkage or stable disease.12345

What safety data exists for targeted therapies in humans?

The safety of targeted therapies has been evaluated in various clinical trials, including umbrella trials in precision oncology, which assess the risk and benefit of these treatments. These studies suggest that targeted therapies can be safely administered to patients with advanced cancers, although specific safety profiles may vary depending on the genetic changes within the tumors.36789

How is targeted therapy different from other treatments for advanced rare cancers?

Targeted therapy for advanced rare cancers is unique because it uses precision medicine to identify and attack specific genetic changes in cancer cells, offering a personalized treatment option where standard treatments often don't exist. This approach can lead to better outcomes by focusing on the unique genetic makeup of each patient's cancer.1231011

Research Team

Dr. Cheryl Ho - Division of Medical ...

Cheryl Ho, MD

Principal Investigator

BC Cancer

Eligibility Criteria

This trial is for adults over 18 with advanced rare cancers or those with uncommon molecular changes, who are expected to live at least 12 weeks. They must be on a Health Canada approved targeted therapy not covered by national funding, have stable brain metastases if present, and be physically able to undergo treatment (ECOG 0-2). Participants need functioning major organs and the ability to consent.

Inclusion Criteria

You are expected to live for at least 12 more weeks.
I am able to care for myself and move around.
I have brain metastases that are either symptom-free or treated.
See 4 more

Exclusion Criteria

My doctor thinks I am not fit for this treatment plan.
Inability to complete quality of life questionnaires
I haven't taken any experimental drugs or joined another clinical trial in the last 14 days.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive targeted therapy for advanced rare cancers or cancers with rare molecular aberrations

12 weeks
Regular interval radiographic imaging and collection of patient-reported outcomes

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Targeted Therapy (Targeted Therapy)
Trial OverviewThe PREDiCTc trial is evaluating the quality of life and survival rates in patients using targeted therapies for rare cancers. These treatments have some evidence of working but aren't widely funded due to their novelty. The study uses CT imaging and Quality of Life (QOL) assessments as tools.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Targeted therapyExperimental Treatment2 Interventions

Targeted Therapy is already approved in Canada for the following indications:

🇨🇦
Approved in Canada as Targeted Therapy for:
  • Advanced rare cancers
  • Cancer harbouring rare molecular aberrations

Find a Clinic Near You

Research Locations NearbySelect from list below to view details:
BC CancerVancouver, Canada
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Who Is Running the Clinical Trial?

British Columbia Cancer Agency

Lead Sponsor

Trials
181
Patients Recruited
95,900+

Findings from Research

Rare cancers, such as bone and soft-tissue sarcomas and neuroendocrine tumors, represent about 25% of all cancers but are often under-researched, leading to limited treatment options and poorer prognoses for patients.
Recent advancements in molecular analyses and innovative imaging techniques are enhancing the diagnosis and treatment of these rare cancers, emphasizing the need for multidisciplinary collaboration and the establishment of dedicated reference centers to improve patient outcomes.
Biology-guided precision medicine in rare cancers: Lessons from sarcomas and neuroendocrine tumours.van der Graaf, WTA., Tesselaar, MET., McVeigh, TP., et al.[2022]
Clinical Next-Generation Sequencing for Precision Oncology in Rare Cancers.Groisberg, R., Hong, DS., Roszik, J., et al.[2020]
Rationale and Design of the Targeted Agent and Profiling Utilization Registry (TAPUR) Study.Mangat, PK., Halabi, S., Bruinooge, SS., et al.[2022]
Clinical trial design for rare cancers: why a less conventional route may be required.Panageas, KS.[2018]
The genetic complexity of common cancers and the promise of personalized medicine: is there any hope?Arnedos, M., Vielh, P., Soria, JC., et al.[2022]
In a systematic review of 31 sub-trials involving 1637 cancer patients, the overall objective response rate (ORR) for therapies in umbrella trials was 17.7%, indicating that only about one in five patients responded positively to treatment.
The combination of targeted therapies with chemotherapy showed a significantly higher ORR of 39.0% compared to 13.3% for targeted therapies alone, but the overall drug-related death rate was low at 0.8%, suggesting that while umbrella trials may not guarantee increased benefits, they have a manageable safety profile.
Risk and benefit for umbrella trials in oncology: a systematic review and meta-analysis.Strzebonska, K., Blukacz, M., Wasylewski, MT., et al.[2023]
Comprehensive Genomic and Transcriptomic Analysis for Guiding Therapeutic Decisions in Patients with Rare Cancers.Horak, P., Heining, C., Kreutzfeldt, S., et al.[2022]
Patients with Rare Cancers in the Drug Rediscovery Protocol (DRUP) Benefit from Genomics-Guided Treatment.Hoes, LR., van Berge Henegouwen, JM., van der Wijngaart, H., et al.[2023]
Comprehensive Genomic Profiling of Rare Tumors: Routes to Targeted Therapies.Wang, S., Chen, R., Tang, Y., et al.[2020]
Platform study of genotyping-guided precision medicine for rare solid tumours: a study protocol for a phase II, non-randomised, 18-month, open-label, multiarm, single-centre clinical trial testing the safety and efficacy of multiple Chinese-approved targeted drugs and PD-1 inhibitors in the treatment of metastatic rare tumours.Wang, S., Huang, HY., Wu, D., et al.[2021]
Interdisciplinary team science to understand and intercept rare cancers.Fröhling, S.[2022]

References

Biology-guided precision medicine in rare cancers: Lessons from sarcomas and neuroendocrine tumours. [2022]
Clinical Next-Generation Sequencing for Precision Oncology in Rare Cancers. [2020]
Rationale and Design of the Targeted Agent and Profiling Utilization Registry (TAPUR) Study. [2022]
Clinical trial design for rare cancers: why a less conventional route may be required. [2018]
The genetic complexity of common cancers and the promise of personalized medicine: is there any hope? [2022]
Risk and benefit for umbrella trials in oncology: a systematic review and meta-analysis. [2023]
Comprehensive Genomic and Transcriptomic Analysis for Guiding Therapeutic Decisions in Patients with Rare Cancers. [2022]
Patients with Rare Cancers in the Drug Rediscovery Protocol (DRUP) Benefit from Genomics-Guided Treatment. [2023]
Comprehensive Genomic Profiling of Rare Tumors: Routes to Targeted Therapies. [2020]
Platform study of genotyping-guided precision medicine for rare solid tumours: a study protocol for a phase II, non-randomised, 18-month, open-label, multiarm, single-centre clinical trial testing the safety and efficacy of multiple Chinese-approved targeted drugs and PD-1 inhibitors in the treatment of metastatic rare tumours. [2021]
11.United Statespubmed.ncbi.nlm.nih.gov
Interdisciplinary team science to understand and intercept rare cancers. [2022]