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mTOR Inhibitor

Topical Rapamycin/Sirolimus for Vascular Anomalies (NOVA Trial)

Phase 1

Recruiting

Led By Ramamoorthy Nagasubramanian, MD

Research Sponsored by Nemours Children's Clinic

Eligibility Criteria Checklist

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

Must have

Patients must not have received any non-FDA approved drug within 4 weeks or 5 half-lives, whichever is longer, prior to starting sirolimus and during treatment with sirolimus

Patients will be more than 36 months and less than 21 years of age

Must not have

Patients who require medications that inhibit/induce CYP3A4 enzyme activity to control concurrent medical conditions

Patients with symptomatic complicated vascular anomalies with severe systemic symptoms that will need systemic therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 32 weeks

Awards & highlights

All Individual Drugs Already Approved

Approved for 5 Other Conditions

No Placebo-Only Group

Summary

This trial will test a topical sirolimus ointment to treat vascular anomalies in pediatric patients and measure its effectiveness and safety.

Who is the study for?

This trial is for children and young adults aged 3 to 21 with complicated vascular anomalies, including those who haven't responded well to other treatments. Participants need proper organ function, controlled medical conditions, and can't be on certain drugs that affect the immune system or are pregnant.

What is being tested?

The study tests a topical ointment containing Sirolimus (1%) on skin lesions caused by vascular anomalies. Over six months, patients apply the ointment and their response is monitored through lesion size, flattening, number of vesicles, infection/bleeding episodes, and pain improvement.

What are the potential side effects?

Since the Sirolimus ointment has limited absorption into the body, only mild side effects are expected. These may include local skin reactions at the application site or slight systemic effects due to minimal drug absorption.

Eligibility Criteria

Inclusion Criteria

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

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I haven't taken any non-FDA approved drugs recently.

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I am between 3 years and 21 years old.

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It has been 6 months or more since my last radiation therapy to the area where my tumor is.

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I have a vascular anomaly that is either new or did not improve with previous treatments.

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It has been over 2 weeks since my last biologic therapy.

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My bone marrow is working well.

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My platelet count is above 50,000 and I haven't had a transfusion in the last week.

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I have Kaposiform Hemangioendothelioma and cannot tolerate or did not respond to sirolimus treatment.

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My liver enzyme ALT levels are less than five times the normal limit.

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My bilirubin levels are within normal range for my age.

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I am mostly independent in my daily activities.

Exclusion Criteria

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

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I need medication that affects liver enzyme activity for another health condition.

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I have severe symptoms from vascular anomalies needing treatment.

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I have HIV or a known immune system problem.

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I am not pregnant or breastfeeding.

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I have a hemangioma.

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I am on long-term steroids or other drugs that weaken my immune system.

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I do not have any severe illnesses that could affect my participation in the trial.

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I am not currently on IV antibiotics for an uncontrolled infection.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 32 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~32 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 32 weeks for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Cyst formation

Decrease in cyst formation

Decrease in discharge

+1 more

Secondary study objectives

ALT level

Bilirubin level

Fasting LDL

+6 more

Other study objectives

Blood pressure

Changes in Height

Changes in Weight

+4 more

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

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: Open label, topical sirolimus armExperimental Treatment1 Intervention

Single arm, open label study of1% sirolimus ointment applied to affected area twice daily for the first four weeks followed by once daily for 5 months.

0 / 19Eligibility criteria met