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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. august 2026 oppdatert av: 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.

Studieoversikt

Status

Har ikke rekruttert ennå

Forhold

Vaskulære anomalier

Studietype

Observasjonsmessig

Registrering (Antatt)

200

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Navn: Novartis Pharmaceuticals
Telefonnummer: +41613241111
E-post: novartis.email@novartis.com

Studer Kontakt Backup

Navn: Novartis Pharmaceuticals

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

Barn
Voksen
Eldre voksen

Tar imot friske frivillige

Nei

Prøvetakingsmetode

Ikke-sannsynlighetsprøve

Studiepopulasjon

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.

Beskrivelse

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.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

Antall grupper / kohorter

Kohorter og intervensjoner

Gruppe / Kohort
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.

Hva måler studien?

Primære resultatmål

Resultatmål	Tiltaksbeskrivelse	Tidsramme
Pasientens grunnleggende demografi Tidsramme: Utgangsverdi		Utgangsverdi
Number of Patients by Clinical Characteristics Tidsramme: 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 Tidsramme: Up to 10 years		Up to 10 years

Sekundære resultatmål

Resultatmål	Tiltaksbeskrivelse	Tidsramme
Number of Adverse Events per Person per Year (PPPY) Tidsramme: Up to 10 years		Up to 10 years
Total Number of Adverse Events Tidsramme: Up to 10 years		Up to 10 years
Percentage of Patients With Adverse Events Tidsramme: Up to 10 years		Up to 10 years
Number of Clinical Response Events PPPY Tidsramme: 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 Tidsramme: Up to 10 years		Up to 10 years
Percentage of Patients Who Experience a Clinical Response Tidsramme: Up to 10 years		Up to 10 years
Treatment Duration Tidsramme: Up to 10 years		Up to 10 years
Number of Patients by Reason for Treatment Discontinuation Tidsramme: Up to 10 years		Up to 10 years
Frequency of Labs and Imaging for Disease Monitoring Tidsramme: Up to 10 years		Up to 10 years
Frequency of Adverse Events in Organ Systems Tidsramme: Up to 10 years		Up to 10 years
Percentage of Patients With Disease/Quality of Life (QoL) Impact Tidsramme: 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 Tidsramme: Up to 10 years	Clinical parameters will include dosing and treatment duration and frequency of follow up.	Up to 10 years

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Novartis Pharmaceuticals

Etterforskere

Studieleder: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Antatt)

30. september 2026

Primær fullføring (Antatt)

29. oktober 2027

Studiet fullført (Antatt)

29. oktober 2027

Datoer for studieregistrering

Først innsendt

11. august 2026

Først innsendt som oppfylte QC-kriteriene