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

31 augustus 2026 bijgewerkt door: Novartis Pharmaceuticals

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

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.

Studie Overzicht

Toestand

Nog niet aan het werven

Conditie

Vasculaire afwijkingen

Studietype

Observationeel

Inschrijving (Geschat)

200

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Naam: Novartis Pharmaceuticals
Telefoonnummer: +41613241111
E-mail: novartis.email@novartis.com

Studie Contact Back-up

Naam: Novartis Pharmaceuticals

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

Kind
Volwassen
Oudere volwassene

Accepteert gezonde vrijwilligers

Nee

Bemonsteringsmethode

Niet-waarschijnlijkheidssteekproef

Studie Bevolking

Pediatric and adult patients with complex vascular anomalies who have been treated for at least three months with mTOR inhibitors, PI3K inhibitors, and/or MEK inhibitors at academic and community vascular centers across the US.

Beschrijving

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.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

Aantal groepen / cohorten

Cohorten en interventies

Groep / Cohort
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.

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat	Maatregel Beschrijving	Tijdsspanne
Baseline Demografieën Tijdsspanne: Baseline		Baseline
Number of Patients by Clinical Characteristics Tijdsspanne: 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.	Baseline
Number of Patients by Treatment Received in Each Line of Therapy Tijdsspanne: Up to 10 years		Up to 10 years

Secundaire uitkomstmaten

Uitkomstmaat	Maatregel Beschrijving	Tijdsspanne
Number of Adverse Events per Person per Year (PPPY) Tijdsspanne: Up to 10 years		Up to 10 years
Total Number of Adverse Events Tijdsspanne: Up to 10 years		Up to 10 years
Percentage of Patients With Adverse Events Tijdsspanne: Up to 10 years		Up to 10 years
Number of Clinical Response Events PPPY Tijdsspanne: Up to 10 years	Clinical response: improvement of function, reduction of symptoms and complications, i.e. pain, infection, bleeding, hospitalization, etc.	Up to 10 years
Total Number of Clinical Response Events Tijdsspanne: Up to 10 years		Up to 10 years
Percentage of Patients Who Experience a Clinical Response Tijdsspanne: Up to 10 years		Up to 10 years
Treatment Duration Tijdsspanne: Up to 10 years		Up to 10 years
Number of Patients by Reason for Treatment Discontinuation Tijdsspanne: Up to 10 years		Up to 10 years
Frequency of Labs and Imaging for Disease Monitoring Tijdsspanne: Up to 10 years		Up to 10 years
Frequency of Adverse Events in Organ Systems Tijdsspanne: Up to 10 years		Up to 10 years
Percentage of Patients With Disease/Quality of Life (QoL) Impact Tijdsspanne: 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).	Up to 10 years
Percentage of Patients With Clinical Parameters Relevant to Routine Care Tijdsspanne: Up to 10 years	Clinical parameters will include dosing and treatment duration and frequency of follow up.	Up to 10 years

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Novartis Pharmaceuticals

Onderzoekers

Studie directeur: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Geschat)

30 september 2026

Primaire voltooiing (Geschat)

29 oktober 2027

Studie voltooiing (Geschat)

29 oktober 2027

Studieregistratiedata

Eerst ingediend

11 augustus 2026

Eerst ingediend dat voldeed aan de QC-criteria