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NCT Number: NCT07767877

A Study 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.

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Key information

Conditions

Vascular Anomalies Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Vascular Malformations

Age range

0 year–90 year

Sex eligibility

All sexes

Study type

Observational

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Baseline Demographics

    Time frame: Baseline

  2. Number of Patients by Clinical Characteristics

    Time frame: 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.

  3. Number of Patients by Treatment Received in Each Line of Therapy

    Time frame: Up to 10 years

Secondary outcomes

  1. Number of Adverse Events per Person per Year (PPPY)

    Time frame: Up to 10 years

  2. Total Number of Adverse Events

    Time frame: Up to 10 years

  3. Percentage of Patients With Adverse Events

    Time frame: Up to 10 years

  4. Number of Clinical Response Events PPPY

    Time frame: Up to 10 years

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

  5. Total Number of Clinical Response Events

    Time frame: Up to 10 years

  6. Percentage of Patients Who Experience a Clinical Response

    Time frame: Up to 10 years

  7. Treatment Duration

    Time frame: Up to 10 years

  8. Number of Patients by Reason for Treatment Discontinuation

    Time frame: Up to 10 years

  9. Frequency of Labs and Imaging for Disease Monitoring

    Time frame: Up to 10 years

  10. Frequency of Adverse Events in Organ Systems

    Time frame: Up to 10 years

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

    Time frame: 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).

  12. Percentage of Patients With Clinical Parameters Relevant to Routine Care

    Time frame: Up to 10 years

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

Study contacts

Contact information is provided by the study sponsor or research team.

Novartis Pharmaceuticals

CONTACT

[email protected]

+41613241111

Novartis Pharmaceuticals

CONTACT

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Aug 17, 2026
Registry last updated
Aug 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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