What side effects are associated with this type of intervention?

Common treatments for cancer, particularly those involving MEK inhibitors like Trametinib, often have a range of side effects. These can include diarrhea, dermatitis acneiform, maculopapular rash, fatigue, dry skin, nausea, dyspnea, and vomiting. Other targeted therapies and immunotherapies may also cause ocular side effects, severe skin reactions, and gastrointestinal toxicities. While these treatments can be effective, they require careful management of these potential adverse effects.

What prior FDA approvals exist?

The FDA has approved several targeted therapies for cancer treatment that involve MEK1 and MEK2 inhibition. Trametinib, often used in combination with dabrafenib, is approved for treating advanced non-small cell lung cancer (NSCLC) with BRAF V600E mutations. This combination has shown significant efficacy, with high overall response rates and disease control rates. Additionally, other targeted therapies like vemurafenib and dabrafenib alone have been approved for cancers with BRAF mutations, such as melanoma and hairy cell leukemia. These approvals highlight the effectiveness of targeting specific genetic mutations in cancer treatment.

What other types of research exist?

Promising alternatives to Trametinib for cancer treatment in 2024 include: 1) PI3K/PTEN/AKT/mTOR pathway inhibitors such as Temsirolimus and Everolimus, which have shown potential in endometrial cancer and other malignancies. 2) Immune checkpoint inhibitors like anti-PD-1 and anti-CTLA-4 therapies, which have demonstrated efficacy in various cancers including lung cancer and mesothelioma. 3) RET inhibitors such as Selpercatinib and Pralsetinib, particularly for RET-mutated tumors. 4) BRAF inhibitors like Vemurafenib, especially in combination with Rituximab for BRAF-mutated cancers. These agents represent novel and promising options for targeted cancer therapy.

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MEK Inhibitor

Trametinib for Cancer With NF1 Genetic Changes

Philadelphia, PA

Phase 2

Waitlist Available

Led By Jason J Luke

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients must have deleterious inactivating mutations of NF-1, or another aberration, as determined via the MATCH Master Protocol

Be older than 18 years old

Must not have

Patients who previously received MEK inhibitors (including, but not limited to, trametinib, binimetinib, cobimetinib, selumetinib, RO4987655 (CH4987655), GDC-0623 and pimasertib) will be excluded

Patients must not have known hypersensitivity to trametinib or compounds of similar chemical or biologic composition or to dimethyl sulfoxide (DMSO)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up assessed at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration

Awards & highlights

No Placebo-Only Group

Summary

This trial tests trametinib, an oral medication, in patients with advanced cancers having an NF1 mutation. Trametinib works by blocking proteins that help cancer cells grow, aiming to stop or slow down the cancer. Trametinib is approved for treating certain types of advanced cancers.

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Who is the study for?

This trial is for cancer patients with a specific genetic change called NF1 mutation. They must have normal heart function, controlled blood pressure, and no history of severe lung disease or eye problems. Those who've had certain monoclonal antibody therapies or MEK inhibitors are excluded.Check my eligibility

What is being tested?

Researchers are testing Trametinib to see if it can shrink tumors or halt their growth in cancers with the NF1 mutation. Trametinib targets proteins that may be essential for the growth of these cancer cells.See study design

What are the potential side effects?

Trametinib may cause side effects such as skin rash, diarrhea, fatigue, and possibly affect heart function. It's also important to watch out for signs of lung issues since patients with a history of lung disease aren't eligible.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My cancer has a specific genetic change related to NF-1.

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Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have not taken any MEK inhibitor medications.

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I am not allergic to trametinib, similar drugs, or DMSO.

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I have never had interstitial lung disease or pneumonitis.

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I don't have, nor am I at risk for, a blocked vein in my eye.

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Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ assessed at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~assessed at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration

This trial's timeline: 3 weeks for screening, Varies for treatment, and assessed at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Objective Response Rate (ORR)

Secondary study objectives

6-month Progression-free Survival (PFS) Rate

Progression Free Survival (PFS)

Side effects data

From 2023 Phase 1 & 2 trial • 27 Patients • NCT01902173

100%

Fatigue

67%

Edema limbs

67%

Pleuritic pain

67%

Nausea

67%

Anorexia

67%

Vomiting

67%

Myalgia

67%

Weight loss

67%

Hyperglycemia

67%

Dyspnea

67%

Lymphocyte count decreased

67%

Pain in extremity

33%

Hyponatremia

33%

Hypophosphatemia

33%

Hyperhidrosis

33%

Diarrhea

33%

Aspartate aminotransferase increased

33%

Rash maculo-papular

33%

Bronchial infection

33%

Hypokalemia

33%

Flu like symptoms

33%

Generalized muscle weakness

33%

Urinary frequency

33%

Pain of skin

33%

Depression

33%

Palmar-plantar erythrodysesthesia syndrome

33%

Fall

33%

Urinary incontinence

33%

Cough

33%

Constipation

33%

Hypercalcemia

33%

Thromboembolic event

33%

Hypocalcemia

33%

Muscle weakness upper limb

33%

Telangiectasia

33%

Vascular disorders-Other

33%

Musculoskeletal and connective tiss disorder - Other

33%

Neoplasms benign, malignant and unspecified - Other

33%

Alkaline phosphatase increased

33%

Fever

33%

Lung infection

33%

Hypotension

33%

Anemia

33%

Cardiac disorders-Other

33%

Chills

33%

Gait disturbance

33%

Arthralgia

33%

Bone pain

33%

Pruritus

33%

Dizziness

33%

Skin and subcutaneous tissue disorders - Other

33%

INR increased

33%

Hypoalbuminemia

33%

Insomnia

33%

Eye disorders-Other

33%

Bruising

33%

Bullous dermatitis

100%

80%

60%

40%

20%

Study treatment Arm

Dabrafenib + GSK2141795 50 mg

Dabrafenib + GSK2141795 75 mg

Dabrafenib + Trametinib 1.5 mg + GSK2141795 25 mg

Dabrafenib + Trametinib 1.5 mg + GSK2141795 50 mg

Dabrafenib + Trametinib 1.5 mg + GSK2141795 75 mg

Dabrafenib + Trametinib 2 mg + GSK2141795 75 mg

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (trametinib)Experimental Treatment1 Intervention

Patients receive trametinib dimethyl sulfoxide PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Trametinib Dimethyl Sulfoxide

2014

Completed Phase 2

~40

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Targeted cancer therapies, such as Trametinib, work by inhibiting specific proteins involved in cancer cell growth and survival. Trametinib blocks MEK1 and MEK2, proteins in the MAPK/ERK pathway, which is often overactive in cancer cells. By disrupting this pathway, Trametinib can slow down or stop the growth of cancer cells. This targeted approach is crucial for cancer patients as it offers a more personalized treatment, potentially leading to better efficacy and reduced toxicity compared to conventional chemotherapy.