Denne side blev automatisk oversat, og nøjagtigheden af oversættelsen er ikke garanteret. Der henvises til engelsk version for en kildetekst.

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 opdateret af: 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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Vaskulære anomalier

Undersøgelsestype

Observationel

Tilmelding (Anslået)

200

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

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

Undersøgelse Kontakt Backup

Navn: Novartis Pharmaceuticals

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

Barn
Voksen
Ældre voksen

Tager imod sunde frivillige

Ingen

Prøveudtagningsmetode

Ikke-sandsynlighedsprøve

Studiebefolkning

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

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

Antal grupper/kohorter

Kohorter og interventioner

Gruppe / kohorte
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.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål	Foranstaltningsbeskrivelse	Tidsramme
Baseline Demografi Tidsramme: Baseline		Baseline
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	Foranstaltningsbeskrivelse	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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Novartis Pharmaceuticals

Efterforskere

Studieleder: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

30. september 2026

Primær færdiggørelse (Anslået)

29. oktober 2027

Studieafslutning (Anslået)

29. oktober 2027

Datoer for studieregistrering

Først indsendt

11. august 2026

Først indsendt, der opfyldte QC-kriterier