Targeted Therapy Based on Genetic Profile for Cancer

Overseen By Apostolia M. Tsimberidou

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: M.D. Anderson Cancer Center

Must not be taking: Steroids, Bevacizumab

Disqualifiers: Uncontrolled hypertension, Cardiac conditions, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 2 months to 3 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how personalized treatments can assist patients with metastatic cancer, where the cancer has spread to other parts of the body. By analyzing the genetic makeup of a patient's tumor, the trial seeks to determine if targeted therapies—designed to address specific genetic changes in cancer cells—are more effective than standard treatments. Participants will be randomly assigned to receive either standard care or a treatment tailored to their tumor's genetic profile. Ideal candidates for this trial have metastatic cancer, have tried other treatments, and have a tumor that can be biopsied. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to benefit from potentially more effective therapies.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you have received chemotherapy, surgery, or radiotherapy recently, you must wait 3-4 weeks before starting the study treatment.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that targeted therapy, customized based on a patient's specific cancer characteristics, is often easy for patients to handle. This method tailors treatment to each person's unique cancer profile, leading to fewer side effects and better outcomes. Evidence from similar treatments indicates that side effects are usually mild and manageable. For instance, patients might feel slightly tired or nauseous, but serious side effects are rare. This suggests that targeted therapy based on a patient's cancer profile is generally safe. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for cancer?

Researchers are excited about targeted therapy based on molecular profiling because it offers a personalized approach to cancer treatment. Unlike standard-of-care therapies that are not selected based on genetic alterations, this targeted therapy identifies and attacks specific genetic changes in cancer cells. This precision can potentially lead to more effective treatment with fewer side effects compared to conventional methods. By honing in on the unique genetic makeup of a patient's cancer, this approach aims to improve outcomes and provide a more tailored treatment experience.

What evidence suggests that this trial's treatments could be effective for metastatic cancer?

This trial will compare two treatment approaches: targeted therapy based on a tumor's genetic makeup and standard-of-care therapy. Studies have shown that targeted therapy, tailored to a tumor's genetic profile, can effectively treat advanced cancer. This method uses genetic information from a patient's tumor to select treatments that specifically attack cancer cells. Research indicates that this approach can lead to better outcomes than traditional chemotherapy, particularly because targeted drugs often perform better when cancer cells have not been extensively treated with other therapies. Early findings suggest that this personalized approach can help manage cancer that has spread to other parts of the body. Overall, using a genetic profile to guide treatment represents an exciting development in cancer care. ^{1 6 7 8 9}

Who Is on the Research Team?

Apostolia M Tsimberidou

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

This trial is for patients with metastatic cancer who have tried standard treatments or decided not to use them. They should be relatively healthy, able to perform daily activities (ECOG 0-1), and have tumors that can be measured or biopsied. Women of childbearing age and men must agree to contraception during the study. Patients with stable brain metastasis off steroids for two weeks are eligible.

Inclusion Criteria

My tumor can be biopsied or I have a recent tumor sample available.

I had cancer before, but I've been cancer-free for over 3 years.

Absolute neutrophil count >= 1,000/ul

Exclusion Criteria

Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns or compliance with clinical study procedures

I have not been treated based on genetic testing results.

I haven't had cancer treatment in the last 3 weeks, or I've recovered from its side effects.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Molecular Profiling

Genomic analysis of tumor sample is performed to identify molecular alterations and assign treatment

2-4 weeks

Treatment

Participants receive either matched targeted therapy or standard-of-care therapy based on randomization

2 months to 3 years

Follow-up

Participants are monitored for progression-free survival and other outcomes

Continuous Monitoring

What Are the Treatments Tested in This Trial?

Interventions

• Standard-of-Care Therapy
• Targeted Therapy Based on Molecular Profiling

Trial Overview The trial compares targeted therapy chosen based on genetic differences in a patient's tumor against standard cancer treatment. It aims to determine if personalized medicine, using information about the tumor's molecular profile, is more effective than traditional approaches not tailored to genetic changes.

How Is the Trial Designed?

Group I: Arm B: Standard-of-Care Therapy Experimental Treatment 1 Intervention

Standard-of-Care treatment not selected on basis of alteration analysis.

Group II: Arm A: Targeted Therapy Active Control 1 Intervention

Personalized treatment, targeted therapy against the alteration based on molecular profiling.

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Tempus AI

Industry Sponsor

Trials

18

Recruited

20,700+

Tempus Labs

Industry Sponsor

Trials

17

Recruited

20,200+

Published Research Related to This Trial

Genotype-guided cancer therapy is crucial for personalizing treatment, as it helps to optimize drug exposure and predict potential toxicities, which is especially important given the variability among patients and the narrow therapeutic range of anticancer drugs.

Over a dozen FDA-approved anticancer drugs now include pharmacogenetic biomarkers, and this review emphasizes the need for more prospective clinical trials to enhance the effectiveness of personalized cancer treatments and address existing barriers to their implementation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Application of genotype-guided cancer therapy in solid tumors. Patel, JN.[2021]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Risk and benefit for umbrella trials in oncology: a systematic review and meta-analysis. Strzebonska, K., Blukacz, M., Wasylewski, MT., et al.[2023]

Citations

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Targeted Therapy and Molecular Profiling in Cancer Care The integration of molecular profiling and targeted therapy into cancer care has significantly improved the way cancer is treated. These ...

2. jamanetwork.com jamanetwork.com/journals/jamaoncology/fullarticle/2771755

Site-Specific and Targeted Therapy Based on Molecular ... This clinical trial assesses the clinical use of site-specific treatment, including molecularly targeted therapy based on next-generation ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12077269/

Impact of molecular diagnostics and targeted cancer ... This study further supports evidence indicating that patients with advanced cancer benefit from molecular profiling and targeted therapy.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10948510/

Molecular diagnostics tailoring personalized cancer therapy ... This is based on the hypothesis that targeted drug exert better effects in cancer cells prior to their exposure to several lines of chemotherapy ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S2949820124000869

Off-label drug use in oncology: integrating molecular and ... Recent advancements in cancer treatment have shifted the paradigm from chemotherapy to targeted therapies and immunotherapies, proving effective even in early- ...

6. cancer.org cancer.org/cancer/managing-cancer/treatment-types/precision-medicine.html

Precision or Personalized Medicine Targeted therapy (treatment designed to attack a specific target on cancer cells); Immunotherapy (medicines used to help the body's immune ...

7. nature.com nature.com/articles/s41698-025-00942-5

Tumor diagnosis recharacterization enabled by ... Final efficacy and safety data, and exploratory molecular profiling from the phase III ALUR study of alectinib versus chemotherapy in ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S2468294224000728

Personalized medicine: An alternative for cancer treatment Personalized medicine is a new cancer treatment technique that focuses on tailoring medication to each patient's specific genetic, biochemical, and lifestyle ...

9. targetedonc.com targetedonc.com/view/evolution-of-personalized-cancer-care-with-molecular-profiling

Evolution of Personalized Cancer Care With Molecular ... Molecular oncology is revolutionizing cancer care by using genetic profiling to tailor personalized treatments, improving efficacy, minimizing adverse events.