Trametinib for Ras/MAPK Pathway Vascular Anomalies in Kids and Teens

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 2 trial evaluates the safety and effectiveness of trametinib in participants aged 2 months to 30 years with Ras/MAPK pathway–driven vascular anomalies. Eligible participants will receive trametinib for up to 3 years, with a minimum 2-year treatment if there is a positive response, and will attend up to 16 clinic visits at CHOP. The study requires measurable disease, sufficient organ function, and contraception for those who can become pregnant. Exclusion criteria include certain cardiac, infectious, hepatic, and other conditions that may complicate treatment. The trial is conducted at CHOP with collaboration from Novartis, CaNVAS, and Baylor College of Medicine.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Signed informed consent and assent (when applicable)
2. Males or females age \> 2 months to \< 30 years at the time of informed consent.
3. Have a documented Ras/MAPK-pathway variant.
4. Have a symptomatic vascular anomaly in need of medical therapy.
5. Have a disease-related lesion or lesions which can be measured
6. Have a Lansky or Karnofsky performance status score of ≥ 50.
7. Have acceptable organ function
8. (For persons who can get pregnant) Have a negative serum or urine pregnancy test within 7 days prior to starting study medication.
9. Must agree to the use an acceptable method of contraception
10. Subjects must be able to swallow tablets or liquid or use a nasogastric or gastric tube

Exclusion Criteria:

1. Subjects seeking treatment for hypertrophic cardiomyopathy without a vascular anomaly
2. Prior medications that are not allowable per the study protocol
3. Confirmed or possible infections
4. History of hepatic sinusoidal obstructive syndrome (veno-occlusive disease of the liver) in the last 3 months.
5. Active gastrointestinal (GI) disease
6. Subjects with history of or current risk of retinal vein occlusion (RVO) or central serous retinopathy (CSR) or uncontrolled glaucoma or ocular hypertension.
7. Allergic reactions: Subjects with a history of allergic reaction attributed to compounds of similar chemical or biological composition to trametinib are not eligible.
8. History of prior and or ongoing cancer or ongoing investigations or treatment for cancer at time of informed consent.
9. Subjects unable to comply with safety monitoring requirements.
10. Subject may not be pregnant or breast feeding.
11. Parents/guardian/LAR or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
12. Debulking or other major surgery performed within 30 days, at time of informed consent;
13. Clinically meaningful bleeding related to VA: Grade 2 within 14 days or Grade 3 and more within 28 days before study treatment start as per CTCAE v. 5.0.

    Sclerotherapy/embolization for vascular complications performed within 14 days before informed consent.
14. Subjects not able to understand and to comply with study instructions and requirements (in patients, legally authorized representatives, or guardians as applicable) at time of informed consent.

Where it's running

• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting

Study contacts

• Study coordinator: CVAP Research Team
• Email: CVAPResearch@chop.edu
• Phone: 267-426-9188

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.