Unfortunately, there is no cure for CLOVES. Surgery and other types of medical interventions are the primary treatments for CLOVES overgrowth, vascular anomalies and other related medical issues. However there are many current opportunities for you to participate in research.
Please read below about research opportunities and new targeted treatment medications to treat CLOVES/PROS.
This study is open to adult patients and caregivers over the age of 18, who live in the United States. Participants will receive $40 for their time. If you are interested, please complete this form. Contact Dr. Bryan Sisk with any questions at [email protected]
This is a prospective Phase II multi-center study with an upfront 16-week, randomized, double-blind, placebo-controlled period, and extension periods, to assess the efficacy, safety and pharmacokinetics of alpelisib in pediatric and adult participants with PIK3CA-related overgrowth spectrum (PROS). Enrollment is currently open in Norway and Spain.
Purpose: To identify biomarkers in patients diagnosed with a vascular anomaly*.
Who is eligible: Any participant with a diagnosed vascular anomaly* that are currently not on treatment for the condition and are having labs (blood) drawn as standard of care.
*Vascular anomalies included: Generalized Lymphatic Anomaly (GLA), Gorham-Stout Disease (GSD), Kaposiform Hemangioendothelioma (KHE), Kaposiform Lymphangiomatosis (KLA), Klippel Trenaunay Syndrome (KT), Congenital Lipomatous Overgrowth, Vascular Malformations, and Epidermal Nevi Syndrome (CLOVES)
FOR MORE INFORMATION CALL OR EMAIL Megan Metcalf at 513-803-2606 [email protected]
The genetics of AVMs are poorly understood, and many theories currently exist with regards to their development, including the hypothesis that their development begins early in gestation. Kahle Lab is actively recruiting patients with brain or spinal cord AVMs and their families. Participants must have a formal diagnosis based on imaging. This study is currently open to people in the United States.
If you are interested in participating, please email [email protected] with the following information:
- Mailing Address or Addresses where the swabs and paperwork will be sent
- Active contact telephone number
- Full Names and Ages of participants at each address that will provide DNA samples in your household
- Previously referred to genetics? If so, findings?
8/28/20 – VT30-101 is a 2-part first-in-human trial of topically administered VT30 to subjects with cutaneous venous malformations, lymphatic malformations, or mixed venolymphatic malformations associated with PIK3CA or TEK mutations.
Part 1 is a 4-week treatment, open-label, 4-sequence, escalating repeat-application cohort study, with intra-subject and inter-cohort dose escalation.
Part 2 is a 12-week treatment, randomized, placebo-controlled, double-blind, safety and exploratory efficacy study. Part 2 will be initiated only after the successful completion of Part 1 with results that demonstrate the general safety and tolerability of topically applied VT30. Up to 12 subjects who complete Part 1 may be enrolled into Part 2 of the study.
The primary objective is to evaluate the safety and tolerability of VT30. The study will also determine the dose and regimen of VT30 to be carried into Part 2 of the protocol. Other aims include documenting plasma drug levels of VT30 and VT10 and, on an exploratory basis, examining pharmacologic target engagement and change in potential efficacy readouts.
Compassionate Use Access of PIQRAY (formerly BYL719/alpelisib) [7-9-19]
The Novartis Managed Access Program (MAP) for PIQRAY use in PROS is available to patients in the US where PIQRAY is available for commercial use (as well as other countries where not approved).
The FDA approved PIQRAY for HR+/HER2- advanced breast cancer, and thus PROS patients may seek access to the medication through MAP.
Patients should contact their treating physicians who can, if clinically appropriate, make a request to Novartis for access to treatment using this link on the Novartis website https://www.novartis.com/our-focus/healthcare-professionals/managed-access-programs
New Research Opportunity: MOSAIC – ARQULE [6-5-19]
ArQule is currently conducting the MOSAIC (Miransertib in Overgrowth Syndromes in Adults and Children) clinical trial for patients who have been diagnosed with either Proteus syndrome (PS), which involves a mutation in the AKT gene, or those who have been diagnosed with one of a number of distinct overgrowth disorders characterized by a mutation in the PIK3CA gene, referred to as PIK3CA-related overgrowth spectrum (PROS). There are currently no approved medicinal treatments for PS and PROS, leaving patients with minimal treatment options. ArQule was purchased by Merck in January of 2020.
The MOSAIC trial aims to determine whether miransertib, a drug which inhibits the biological pathway that both AKT and PIK3CA mutations affect, can effectively treat patients who are diagnosed with overgrowth syndromes associated with these mutations.
Based on the mechanism of action of miransertib, trial participants must have a mutation in either the AKT1 or PIK3CA gene to be eligible to participate in the MOSAIC trial. Learn more about how miransertib works below.
- For more information, please visit the Clinical Trial page
- Additional information about the MOSAIC trial can be found here.
New Research Opportunity: BYL719/NOVARTIS [6-13-18]
A medical first: CLOVES Syndrome and overgrowth syndromes:remarkable improvement in the health of 19 pediatric and adult patients using a new therapeutic strategy called Alpelisib BYL719. Dr. Guillaume Canaud at the Necker-Enfants Malades Hospital – AP-HP, the Paris Descartes University, Inserm (INEM Institute Necker Enfants Malades – Centre for Molecular Medicine) and his team recently demonstrated the efficacy of a novel medication, a specific inhibitor called BYL719, in a cohort of 19 patients treated at the Necker-Enfants Malades Hospital – AP-HP and suffering from CLOVES Syndrome (Congenital Lipomatous Overgrowth, Vascular Malformation, Epidermal Naevi) or similar disorders.
Opportunity for CLOVES Research (ongoing)
April 23, 2013 – Research Opportunity with National Institutes of Health (NIH)