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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 augusti 2026 uppdaterad 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.

Studieöversikt

Status

Har inte rekryterat ännu

Betingelser

Vaskulära anomalier

Studietyp

Observationell

Inskrivning (Beräknad)

200

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

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

Studera Kontakt Backup

Namn: Novartis Pharmaceuticals

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

Barn
Vuxen
Äldre vuxen

Tar emot friska volontärer

Nej

Testmetod

Icke-sannolikhetsprov

Studera befolkning

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.

Beskrivning

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

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

Antal grupper/kohorter

Kohorter och interventioner

Grupp / 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.

Vad mäter studien?

Primära resultatmått

Resultatmått	Åtgärdsbeskrivning	Tidsram
Demografiska baslinjeuppgifter Tidsram: Utgångsvärde		Utgångsvärde
Number of Patients by Clinical Characteristics Tidsram: 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 Tidsram: Up to 10 years		Up to 10 years

Sekundära resultatmått

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

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Novartis Pharmaceuticals

Utredare

Studierektor: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Beräknad)

30 september 2026

Primärt slutförande (Beräknad)

29 oktober 2027

Avslutad studie (Beräknad)

29 oktober 2027

Studieregistreringsdatum

Först inskickad

11 augusti 2026

Först inskickad som uppfyllde QC-kriterierna