A Study of Clinical Practice and Real-world Care of Patients Treated With mTOR, PI3K, and MEK Inhibitors for Extracranial Vascular Anomalies

NCT07767877 — Not yet recruiting, 200 participants, Novartis Pharmaceuticals

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Targeted Therapies for Vascular Anomalies: A MultiCenter Real World Registry A Review of Clinical Practice and Real-world Care of Patients Treated With mTOR, PI3K, and MEK Inhibitors for Extracranial Vascular Anomalies

Registry	NCT07767877
Status	Not yet recruiting
Study type	Observational
Enrollment	200 (Estimated)
Sponsor	Novartis Pharmaceuticals (Industry)
Conditions	Vascular Anomalies
Dates	start 2026-09-30 (estimated), primary completion 2027-10-29 (estimated), completion 2027-10-29 (estimated), first posted 2026-08-17, last updated 2026-09-01

-Summary

This study aims to describe real-world patient characteristics, treatment patterns, and adverse events associated with targeted therapies used in patients with complex vascular anomalies. The study will create an active registry for participating centers to enter data on patients with complex vascular anomalies being treated with sirolimus/everolimus (mTOR inhibitors), with/without trametinib (MEK inhibitor), or alpelisib (PIK3CA inhibitor). Tertiary care centers in the United States (US) that receive referrals for complex vascular anomaly cases and use Electronic Health Records (EHRs) will contribute patient medical chart reviews to this registry.

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+Arms and interventions

Arm	Type	Interventions	Description
Complex Vascular Anomaly Group			Patients with vascular anomalies that were treated with mTOR inhibitors (sirolimus/everolimus), a MEK inhibitor (trametinib), and a PIK3CA inhibitor (alpelisib) for a minimum of 3 months.

+Outcome measures

Primary outcomes

Baseline Demographics [Baseline]
Number of Patients by Clinical Characteristics [Baseline]: Characteristics include vascular anomaly diagnosis, family history of vascular anomalies, cancer diagnosis, disease severity and anatomic locations involved, associated complications, other medical and surgical interventions, and other medications used.
Number of Patients by Treatment Received in Each Line of Therapy [Up to 10 years]

Secondary outcomes

Number of Adverse Events per Person per Year (PPPY) [Up to 10 years]
Total Number of Adverse Events [Up to 10 years]
Percentage of Patients With Adverse Events [Up to 10 years]
Number of Clinical Response Events PPPY [Up to 10 years]: Clinical response: improvement of function, reduction of symptoms and complications, i.e. pain, infection, bleeding, hospitalization, etc.
Total Number of Clinical Response Events [Up to 10 years]
Percentage of Patients Who Experience a Clinical Response [Up to 10 years]
Treatment Duration [Up to 10 years]
Number of Patients by Reason for Treatment Discontinuation [Up to 10 years]
Frequency of Labs and Imaging for Disease Monitoring [Up to 10 years]
Frequency of Adverse Events in Organ Systems [Up to 10 years]
Percentage of Patients With Disease/Quality of Life (QoL) Impact [Up to 10 years]: Since this is not a clinical trial and validated instruments are not frequently used in routine clinical visits, disease/QoL impact will be defined by impact on daily activities (walking ambulation, hobbies), demand on multidisciplinary care and impact on emotions (mood, self-esteem).
Percentage of Patients With Clinical Parameters Relevant to Routine Care [Up to 10 years]: Clinical parameters will include dosing and treatment duration and frequency of follow up.

+Eligibility

Sex: All, ages: 0 Years to 90 Years

Inclusion criteria:

* Diagnosed with a spectrum of vascular anomalies including but not limited to congenital vascular and lymphatic anomalies, vascular tumors and lymphatic malformations, and acquired vascular malformations.
* Treated with ≥1 of mammalian target of rapamycin (mTOR) inhibitors, mitogen-activated protein kinase/ERK kinase (MEK) inhibitors and phosphoinositide 3-kinase (PI3K) inhibitors continuously for 3 months.

Exclusion criteria:

* Patients diagnosed with a vascular anomaly who have not been treated with mTOR inhibitors, MEK inhibitors and PI3K inhibitors for at least 3 months.
* Patients with other complex medical conditions; i.e. rare genetic syndromes.
* Patients receiving many other systemic therapies making data collection not feasible.
* Recurrent use of immunosuppressive agents, i.e. systemic steroids or targeted medical therapies for oncologic disorders, etc.
* Patients with significant gaps in data collection.
* Patients with concurrent enrollment in interventional trials.

Other protocol-defined inclusion/exclusion criteria may apply.

+Investigators

Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals