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

N/ANot Yet RecruitingNCT07767877

Novartis Pharmaceuticals200 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

200

Conditions

Vascular Anomalies

Eligibility

Sex: ALLMin age: 0 YearsMax age: 90 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Baseline Demographics

Time frame: Baseline

Number of Patients by Clinical Characteristics

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.

Time frame: Baseline

Number of Patients by Treatment Received in Each Line of Therapy

Time frame: Up to 10 years

Secondary Outcomes

Number of Adverse Events per Person per Year (PPPY)

Time frame: Up to 10 years

Total Number of Adverse Events

Time frame: Up to 10 years

Percentage of Patients With Adverse Events

Time frame: Up to 10 years

Number of Clinical Response Events PPPY

Clinical response: improvement of function, reduction of symptoms and complications, i.e. pain, infection, bleeding, hospitalization, etc.

Time frame: Up to 10 years

Total Number of Clinical Response Events

Time frame: Up to 10 years

Percentage of Patients Who Experience a Clinical Response

Time frame: Up to 10 years

Treatment Duration

Time frame: Up to 10 years

Number of Patients by Reason for Treatment Discontinuation

Time frame: Up to 10 years

Frequency of Labs and Imaging for Disease Monitoring

Time frame: Up to 10 years

Frequency of Adverse Events in Organ Systems

Time frame: Up to 10 years

Percentage of Patients With Disease/Quality of Life (QoL) Impact

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).

Time frame: Up to 10 years

Percentage of Patients With Clinical Parameters Relevant to Routine Care

Clinical parameters will include dosing and treatment duration and frequency of follow up.

Time frame: Up to 10 years

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

Overview

Conditions

Eligibility

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts