What side effects are associated with this type of intervention?

Common treatments for melanoma include surgery, immunotherapy, targeted therapy, chemotherapy, and radiation therapy. Immunotherapy drugs like checkpoint inhibitors (e.g., pembrolizumab, nivolumab) can cause side effects such as fatigue, rash, and inflammation of organs (e.g., colitis, hepatitis). Targeted therapies, such as BRAF and MEK inhibitors (e.g., vemurafenib, dabrafenib, trametinib), often lead to side effects like skin rash, fever, and joint pain. Chemotherapy can cause nausea, vomiting, and hair loss. Radiation therapy may result in skin irritation and fatigue. For treatments similar to Tipifarnib, which targets HRAS mutations, side effects may include gastrointestinal issues, fatigue, and hematologic abnormalities, although specific data on HRAS inhibitors in melanoma is limited.

What prior FDA approvals exist?

FDA-approved treatments for melanoma include immunotherapies and targeted therapies. Pembrolizumab, an immune checkpoint inhibitor, has shown significant improvement in recurrence-free survival for patients with BRAF V600 mutated tumors. Dabrafenib plus trametinib, a combination of BRAF and MEK inhibitors, is another approved option for patients with BRAF V600 mutations, improving both recurrence-free and overall survival. Vemurafenib, a BRAF inhibitor, combined with rituximab, has also been used in treating melanoma, particularly in cases with BRAF mutations. While these treatments focus on BRAF mutations, HRAS inhibition, as studied with Tipifarnib, is not specifically mentioned for melanoma in the provided context.

What other types of research exist?

Promising alternatives to Tipifarnib for melanoma patients include: 1) Dabrafenib plus Trametinib for patients with BRAFV600E mutations, which has shown efficacy in multiple trials. 2) Pembrolizumab, an immune checkpoint inhibitor, which has demonstrated significant survival benefits in melanoma. 3) Nivolumab, another immune checkpoint inhibitor, often used in combination with Ipilimumab for enhanced efficacy. These therapies represent advanced options for melanoma treatment in 2024.

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Farnesyltransferase Inhibitor

Tipifarnib for Cancer

Phase 2

Waitlist Available

Led By Christine A Pratilas

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 be >=12 months and =< 21 years of age at the time of study enrollment

Patients must have a body surface area >= 0.29 m^2 at enrollment

Must not have

Patients receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant

Patients with uncontrolled infection

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, prior to cycle 5, end of treatment, assessed up to 7 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests tipifarnib, a drug that may help stop cancer growth, on children with certain advanced cancers that have a specific genetic change. The drug works by targeting and blocking the growth of these cancer cells.

Who is the study for?

This trial is for children and young adults up to 21 years old with advanced solid tumors, lymphoma, or histiocytic disorders that have a change in the HRAS gene. They should be able to swallow tablets, have recovered from previous cancer treatments, and meet certain health criteria like blood counts. Pregnant individuals or those on certain medications are excluded.

What is being tested?

The effectiveness of Tipifarnib is being tested on patients whose cancers have returned or spread and have an HRAS gene alteration. This drug aims to block cancer cell growth linked to this genetic change and potentially shrink tumors.

What are the potential side effects?

While not explicitly listed here, similar drugs can cause side effects such as fatigue, nausea, liver issues, low blood counts leading to infection risk or bleeding problems, rash or other skin changes.

Eligibility Criteria

Inclusion Criteria

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

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I am between 12 and 21 years old.

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My body surface area is at least 0.29 square meters.

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My nerve-related side effects from previous treatments are mild.

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I am enrolled in APEC1621SC and assigned to APEC1621M due to a specific mutation.

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I have fluid buildup due to cancer.

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I have never been treated with tipifarnib.

Exclusion Criteria

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

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I am taking medication to prevent graft-versus-host disease after a bone marrow transplant.

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I have an infection that is not responding to treatment.

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I am not taking medication that strongly affects liver enzymes.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, prior to cycle 5, end of treatment, assessed up to 7 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, prior to cycle 5, end of treatment, assessed up to 7 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, prior to cycle 5, end of treatment, assessed up to 7 years 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 (complete response + partial response) in pediatric patients treated with tipifarnib

Secondary study objectives

Percentage of patients experiencing grade 3 or higher adverse events

Progression free survival (PFS)

Other study objectives

Biomarker analysis

Neoplasms

Side effects data

From undefined Phase 3 trial • 144 Patients • NCT00093470

58%

White blood cell decreased

56%

Anemia

54%

Platelet count decreased

35%

Neutrophil count decreased

Fatigue

Rash maculo-papular

Myalgia

Confusion

Febrile neutropenia

Catheter related infection

Infections and infestations - Other, specify

Hypokalemia

Peripheral sensory neuropathy

Eye disorders - Other, specify

Back pain

Treatment related secondary malignancy

Psychosis

Agitation

Acute coronary syndrome

Diarrhea

Nausea

Lung infection

Peripheral nerve infection

Sinusitis

Alanine aminotransferase increased

Aspartate aminotransferase increased

GGT increased

100%

80%

60%

40%

20%

Study treatment Arm

Arm A (Tipifarnib)

Arm B (Clinical Observation)

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 (tipifarnib)Experimental Treatment7 Interventions

Patients receive tipifarnib PO or via nasogastric or gastric tube BID on days 1-7 and 15-21. Treatment repeats every 28 days for up to 26 cycles (2 years) in the absence of disease progression or unacceptable toxicity. Patients undergo tumor disease evaluation with PET scan, CT scan, MRI, or MIBG scintigraphy throughout the trial. Patients may undergo bone marrow aspiration or biopsy at baseline, or if there is suspicion of bone marrow metastasis, or when a complete or partial response is identified, or if there is disease progression in the marrow suspected. Patients may undergo blood specimen collections throughout the trial.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Positron Emission Tomography

2011

Completed Phase 2

~2200

Biospecimen Collection

2004

Completed Phase 3

~2020

Computed Tomography

2017

Completed Phase 2

~2740

Bone Marrow Aspiration and Biopsy

2016

Completed Phase 1

~40

Magnetic Resonance Imaging

2017

Completed Phase 3

~1160

Radionuclide Imaging

2004

Completed Phase 2

~50

Tipifarnib

2019

Completed Phase 3

~1000

0 / 9Eligibility 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

Common treatments for melanoma, particularly those targeting specific genetic mutations, include BRAF and MEK inhibitors. These therapies work by blocking the activity of mutated proteins that drive cancer cell growth and survival. For instance, BRAF inhibitors like vemurafenib target the BRAF V600E mutation, while MEK inhibitors like cobimetinib inhibit the downstream MEK proteins in the same pathway. These targeted therapies are crucial for melanoma patients as they offer a more personalized treatment approach, potentially leading to better outcomes and fewer side effects compared to traditional chemotherapy. Similar to HRAS inhibition studied in trials like Tipifarnib, these treatments aim to disrupt specific molecular pathways critical for tumor growth, providing a strategic advantage in managing the disease.

Novel BRAF mutation in melanoma: A case report.The safety and efficacy of cobimetinib for the treatment of BRAF V600E or V600K melanoma.