An observational study in Vascular Anomalies, sponsored by Novartis Pharmaceuticals. Not yet recruiting. Open to participants aged 0 Years to 90 Years. Per ClinicalTrials.gov, last updated 2026-09-01.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Observational
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.
79 studies on the registry are indexed under Vascular Malformations; 33 are open to participants now.
This study's planned enrollment of 200 is close to the median of 200 across 27 observational studies indexed under Vascular Malformations.
Browse Vascular Malformations studies →Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion criteria:
Other protocol-defined inclusion/exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
This study also gives Alpelisib. No intervention listed here names it: that was found by reading the record.
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
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
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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