Vascular AnomaliesNCT07767877
A Study of Clinical Practice and Real-world Care of Patients Treated With mTOR, PI3K, and MEK Inhibitors for Extracranial Vascular Anomalies
An observational study of Vascular Anomalies, run by Novartis Pharmaceuticals.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 0 to 90 years
- Conditions the study is for
- Vascular Anomalies
- How many people
- 200 participants
- Where
- No sites listed yet
- Study dates
- 30 Sep 2026to 29 Oct 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 90 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
About this study
Official 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
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.
- Conditions
- Vascular Anomalies
- Keywords
- Vascular anomaly, Targeted therapy, Pi3KCA inhibitor, mTOR inhibitor, MEK inhibitor, QoL
- Medical subject headings
- Vascular Malformations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Baseline Demographics
Measured: 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.
Measured: Baseline
Number of Patients by Treatment Received in Each Line of Therapy
Measured: Up to 10 years
Secondary outcomes (12)
Number of Adverse Events per Person per Year (PPPY)
Measured: Up to 10 years
Total Number of Adverse Events
Measured: Up to 10 years
Percentage of Patients With Adverse Events
Measured: 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.
Measured: Up to 10 years
Total Number of Clinical Response Events
Measured: Up to 10 years
Percentage of Patients Who Experience a Clinical Response
Measured: Up to 10 years
Treatment Duration
Measured: Up to 10 years
Number of Patients by Reason for Treatment Discontinuation
Measured: Up to 10 years
Frequency of Labs and Imaging for Disease Monitoring
Measured: Up to 10 years
Frequency of Adverse Events in Organ Systems
Measured: 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).
Measured: 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.
Measured: Up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2026
- Study start30 Sep 2026 (estimated)
- Primary completion29 Oct 2027 (estimated)
- Study completion29 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Vascular AnomaliesStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Retrospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07767877
- Sponsor's protocol ID
- CBYL719F1US02
- Status last verified
- Aug 2026
- First submitted
- 11 Aug 2026
- Last updated
- 1 Sep 2026
- Results posted
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT07767877
- Acronym / sponsor ID
- CBYL719F1US02
- Conditions
- Vascular Anomalies
- Keywords
- Vascular anomaly, Targeted therapy, Pi3KCA inhibitor, mTOR inhibitor, MEK inhibitor, QoL
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 30 Sep 2026 (estimated)
- Primary completion
- 29 Oct 2027 (estimated)
- Study completion
- 29 Oct 2027 (estimated)
- First posted
- 17 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 0 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 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.
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Baseline Demographics
Time frame: Baseline
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.
Number of Patients by Treatment Received in Each Line of Therapy
Time frame: Up to 10 years
12 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
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
Time frame: Up to 10 years
Percentage of Patients With Clinical Parameters Relevant to Routine Care
Time frame: Up to 10 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals contact
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
An observational study of Vascular Anomalies, run by Novartis Pharmaceuticals.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 1 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (+41613241111, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Baseline Demographics (time frame: Baseline). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2026.