Trametinib for Vascular Anomalies
(VATCH Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the safety and effectiveness of Trametinib, a drug already approved for treating melanoma, in individuals with certain vascular anomalies (unusual blood vessel growths) driven by the Ras/MAPK pathway. The goal is to determine if this drug can reduce the symptoms or size of these anomalies. Participants should have a symptomatic vascular anomaly and a known Ras/MAPK pathway variant.
As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants an opportunity to contribute to significant medical advancements.
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, it mentions that prior medications that are not allowable per the study protocol could exclude you from participating, so it's best to discuss your current medications with the study team.
Is there any evidence suggesting that Trametinib is likely to be safe for humans?
Research has shown that Trametinib, a treatment being tested for blood vessel disorders, appears to be safe. The FDA has already approved this drug for treating metastatic melanoma, indicating it has undergone safety testing for that condition. In studies on blood vessel disorders, Trametinib was generally well-tolerated by patients.
For instance, one study found that 80% of patients showed improvement, and those experiencing pain felt much better. Another study demonstrated that Trametinib helped treat early-stage abnormal connections between arteries and veins over a year, with manageable side effects.
While these results are promising, Trametinib remains experimental for blood vessel disorders. Ongoing trials closely monitor its safety. Participants in these studies help researchers understand how well the treatment works across different ages and conditions.12345Why do researchers think this study treatment might be promising?
Trametinib is unique because it targets the Ras/MAPK pathway, a key driver in certain vascular anomalies, which is not specifically targeted by most existing treatments. Current options often focus on symptom management rather than directly addressing the underlying molecular causes. Trametinib, as a MEK inhibitor, directly interferes with the signaling pathway responsible for the growth and maintenance of these anomalies. This targeted approach could offer more effective relief with potentially fewer side effects, making researchers hopeful for its ability to provide a more precise treatment option for patients.
What evidence suggests that Trametinib might be an effective treatment for vascular anomalies?
Research has shown that Trametinib may help treat certain blood vessel problems caused by issues in the Ras/MAPK pathway. Previous studies found that Trametinib, which blocks a specific protein, can significantly reduce these blood vessel issues. In one study, participants experienced more than a 50% improvement in skin problems after using Trametinib. Additionally, Trametinib has proven effective in helping people with complex lymphatic issues, another type of blood vessel condition. This trial will evaluate the effectiveness and safety of Trametinib in children and young adults with Ras/MAPK pathway-driven vascular anomalies, suggesting that Trametinib could be a useful option for managing these types of blood vessel problems.12467
Are You a Good Fit for This Trial?
This trial is for children and young adults (ages 2 months to under 30 years) with vascular anomalies caused by Ras/MAPK pathway changes, who have symptoms needing treatment. Participants must be able to take medicine by mouth or tube, have measurable lesions, good organ function, and agree to use birth control if needed.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive Trametinib for at least 2 years, with regular assessments every 6 cycles (each cycle is 28 days)
Follow-up
Participants are monitored for safety and effectiveness after treatment, with assessments every 6 months
Extension
Optional extension phase for continued monitoring and treatment if beneficial response is observed
What Are the Treatments Tested in This Trial?
Interventions
- Trametinib
Trial Overview
The study tests Trametinib, an FDA-approved cancer drug used here experimentally for vascular anomalies linked to the Ras/MAPK pathway. Participants will take Trametinib daily for at least 2 years (up to 3), with regular clinic visits over a total of up to 3 years.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
This arm will determine the proportion of subjects with an objective beneficial response to trametinib at the end of cycle 6 using an individualized response criterion based on radiologic assessment, Patient Reported Outcomes (PROs) and Clinical Benefit Assessment (CBA). It will also determine the safety of oral trametinib in children and young adults with Ras/MAPK pathway driven vascular anomalies through various laboratory testing and clinical observations.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital of Philadelphia
Lead Sponsor
Arkansas Children's Hospital Research Institute
Collaborator
Nemours Children's Health
Collaborator
Boston Children's Hospital
Collaborator
NYU Langone Health
Collaborator
Children's Healthcare of Atlanta
Collaborator
Oregon Health and Science University
Collaborator
Riley Children's Health
Collaborator
Children's Hospital of Los Angeles (CHLA)
Collaborator
Children's Wisconsin - Milwaukee
Collaborator
Citations
24VA021; VATCH Trametinib for Ras/MAPK Pathway VAs
The purpose of this study is to assess the effectiveness and safety of Trametinib (the "Study Drug") in patients with Ras/MAPK pathway ...
MEK Inhibition Reduces Vascular Malformations and Gene ...
Trametinib was the most effective MEK inhibitor, correcting some dysregulated genes in NRASQ61R EC, including some in the Notch pathway.
Trametinib for Vascular Anomalies · Info for Participants
The purpose of this study is to assess the effectiveness and safety of Trametinib (the "Study Drug") in patients with Ras/MAPK pathway ...
Trametinib treatment for early-stage extracranial ...
In this study, we observed clinical improvement in all of the participants after trametinib, with an average blanching rate of skin lesions exceeding 50%, a ...
5.
journals.lww.com
journals.lww.com/jova/fulltext/2022/09000/inhibiting_pi3k_and_mapk_pathways__clinical.5.aspxInhibiting PI3K and MAPK Pathways
This case report highlights a patient with PHTS treated with sirolimus and trametinib as combined targeted therapy.
34: Updates of Targeted Therapies for Vascular Anomalies
MEK inhibitors like trametinib, for metastatic melanoma and non-small cell lung cancer, are now used for vascular anomalies targeting mutations of the RAS/MAPK ...
Targeted medical therapies for vascular anomalies - PMC - NIH
Several case reports have cited clinical improvement or stability with trametinib use in patients with AVMs who have not been responsive to ...
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