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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 32 weeks
Awards & highlights
NOVA Trial 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.Check my eligibility
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.See study design
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.
NOVA Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I haven't taken any non-FDA approved drugs recently.
Select...
I am between 3 years and 21 years old.
Select...
It has been 6 months or more since my last radiation therapy to the area where my tumor is.
Select...
I have a vascular anomaly that is either new or did not improve with previous treatments.
Select...
It has been over 2 weeks since my last biologic therapy.
Select...
My bone marrow is working well.
Select...
My platelet count is above 50,000 and I haven't had a transfusion in the last week.
Select...
I have Kaposiform Hemangioendothelioma and cannot tolerate or did not respond to sirolimus treatment.
Select...
My liver enzyme ALT levels are less than five times the normal limit.
Select...
My bilirubin levels are within normal range for my age.
Select...
I am mostly independent in my daily activities.
NOVA Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 32 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~32 weeks
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Cyst formation
Decrease in cyst formation
Decrease in discharge
+1 moreSecondary outcome measures
ALT level
Bilirubin level
Fasting LDL
+6 moreOther outcome measures
Blood pressure
Changes in Height
Changes in Weight
+4 moreSide effects data
From 2020 Phase 2 & 3 trial • 852 Patients • NCT0112002849%
Infections and infestations
20%
Surgical and medical procedures
17%
Immune system disorders
14%
Gastrointestinal disorders
12%
Investigations
10%
Metabolism and nutrition disorders
9%
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
7%
Injury, poisoning and procedural complications
5%
Cardiac disorders
4%
Respiratory, thoracic and mediastinal disorders
4%
Renal and urinary disorders
4%
Nervous system disorders
3%
General disorders and administration site conditions
2%
Blood and lymphatic system disorders
2%
Musculoskeletal and connective tissue disorders
2%
Vascular disorders
2%
Hepatobiliary disorders
1%
Eye disorders
1%
Skin and subcutaneous tissue disorders
100%
80%
60%
40%
20%
0%
Study treatment Arm
Period 2: Sirolimus
Period 1: Alemtuzumab/Tacrolimus
Period 1: Basiliximab/Tacrolimus
Period 2: Tacrolimus
NOVA 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.
Find a Location
Who is running the clinical trial?
Nemours Children's ClinicLead Sponsor
124 Previous Clinical Trials
18,101 Total Patients Enrolled
Ramamoorthy Nagasubramanian, MDPrincipal InvestigatorNemours
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I need medication that affects liver enzyme activity for another health condition.I have severe symptoms from vascular anomalies needing treatment.I haven't taken any non-FDA approved drugs recently.My hemoglobin level is at least 8.0 gm/dL.I have HIV or a known immune system problem.It has been more than 2 weeks since my last major surgery.I haven't had chemotherapy that lowers my blood cell count in the last 4 weeks.It has been over a week since I last received treatment that helps with blood cell counts.I had surgery or a radiology procedure to manage my disease and can start using sirolimus cream.I am not pregnant or breastfeeding.I am between 3 years and 21 years old.I have a hemangioma.It has been 6 months or more since my last radiation therapy to the area where my tumor is.I have a vascular anomaly that is either new or did not improve with previous treatments.I am not currently taking, nor have I taken in the last week, any medication that increases the breakdown of other medications.It has been over 2 weeks since my last biologic therapy.Your blood albumin level is 2 grams per deciliter or higher.My bone marrow is working well.My platelet count is above 50,000 and I haven't had a transfusion in the last week.I have Kaposiform Hemangioendothelioma and cannot tolerate or did not respond to sirolimus treatment.The ratio of protein to creatinine in your urine is less than 0.3 g/l.Your "bad" cholesterol (LDL) level when fasting is less than 160 mg/dL.I am not taking, nor have I taken in the last week, strong CYP3A4 inhibitors.I am on long-term steroids or other drugs that weaken my immune system.My organs are working well.My kidney function is appropriate for my age.I do not have any severe illnesses that could affect my participation in the trial.I agree to use effective birth control while on sirolimus and for 3 months after.My liver enzyme ALT levels are less than five times the normal limit.I am not currently on IV antibiotics for an uncontrolled infection.My kidney function is normal.My bilirubin levels are within normal range for my age.I am mostly independent in my daily activities.
Research Study Groups:
This trial has the following groups:- Group 1: Open label, topical sirolimus arm
Awards:
This trial has 3 awards, including:- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are individuals over 20 years of age being enrolled in the clinical trial?
"Those hoping to be included in this research must fit within the age range of 36 months and 21 years old. An additional 109 studies are available for minors, while older individuals can choose from 166 separate trials."
Answered by AI
What medical issues does the Open label, topical sirolimus arm address?
"Open label topical sirolimus is an approved therapy for combatting rejection in patients who have undergone a liver, kidney or other transplant and suffer from chronic graft versus host disease."
Answered by AI
What potential risks do individuals face when using open label, topical sirolimus?
"Due to the small amount of data backing its efficacy and safety, Open Label Topical Sirolimus Arm was estimated at a 1 on Power's scale."
Answered by AI
To whom is this research open for participation?
"This trial is seeking approximately 30 candidates between three and 21 years of age suffering with vascular anomalies."
Answered by AI
What is the participant capacity of this research endeavor?
"Yes, the information on clinicaltrials.gov confirms that this investigation is actively searching for subjects. The trial was first published on April 1st 2020 and has since been modified as recently as March 23rd 2023. This experiment necessitates 30 participants to be recruited from two distinct facilities."
Answered by AI
What results have been produced by past experiments using Open label, topical sirolimus?
"Currently, there are 122 live investigations into Open label, topical sirolimus arm. Of these studies, 12 of them have moved to Phase 3 trials. The majority of the clinical sites for this research can be located in Chicago, Illinois; however across 1085 locations globally are conducting tests related to Open label, topical sirolimus arm."
Answered by AI
Are there any vacant slots available for this clinical trial?
"Affirmative, clinicaltrials.gov hosts information indicating that this medical trial is currently recruiting patients. This research project was initially published on April 1st 2020 and has been updated most recently on March 23rd 2023. The study requires 30 participants from 2 different sites to be enrolled."
Answered by AI
What are the primary aims of this research endeavor?
"During the 24-week trial period, researchers will monitor cyst formation as a primary outcome measure. Additionally, they will assess Fasting LDL (measured in mg/dl), Total Bilirubin (measured in mg/dL) and Sirolimus level (in ng/ml)."
Answered by AI
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