Vascular Malformations
Recent clinical, regulatory, research and industry developments relating to this disease.
Comprehensive phenotypic and genomic characterization of venous malformations.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 3 clinical trials expected to report results, the earliest in Q1 2027.
- Q1 2027A Modular Open Label, Signal Seeking, Phase II Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations (TARGET-VM)
- Q4 2029A Phase I/II, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetic Characteristics, and Efficacy of CYH33 (a Selective PI3Kα Inhibitor) in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)
- Q3 2031A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation
Clinical MilestonesViewHide
- 2026-06-29A Phase I/II, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetic Characteristics, and Efficacy of CYH33 (a Selective PI3Kα Inhibitor) in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)Results expected Q4 2029
- 2026-06-12A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA MutationResults expected Q3 2031
- 2026-05-05A Modular Open Label, Signal Seeking, Phase II Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations (TARGET-VM)Results expected Q1 2027
- 2026-07-31An Open-Label, Single-Arm Exploratory Clinical Study of Everolimus for the Treatment of Vascular MalformationsPrimary completion
- 2026-07-31ClinicalAn Open-Label, Single-Arm Exploratory Clinical Study of Everolimus for the Treatment of Vascular MalformationsPrimary completion
- 2026-06-29ClinicalA Phase I/II, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetic Characteristics, and Efficacy of CYH33 (a Selective PI3Kα Inhibitor) in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)Results expected Q4 2029
- 2026-06-12ClinicalA Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA MutationResults expected Q3 2031
- 2026-05-05ClinicalA Modular Open Label, Signal Seeking, Phase II Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations (TARGET-VM)Results expected Q1 2027
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes4
- Middle Cerebral Artery1
- Proto-Oncogene Proteins c-akt1
- Stroke1
- Vascular Malformations1
Leading journals2
- Human pathology1
- Scientific reports1
Leading researchers8
- Chang JY1
- Ha SH1
- Hata K1
- Hirose K1
- Hori Y1
- Imanaka-Yoshida K1
- Jeong S1
- Kang DW1
Affiliations (unnormalised)6
- Asan Medical Center1
- Gangneung Asan Hospital1
- Genome Information Research Center1
- Gil Medical Center1
- Graduate School of Medicine1
- Hanyang University College of Medicine1
Reference
Authoritative identity, definition & identifiers.
A spectrum of congenital, inherited, or acquired abnormalities in BLOOD VESSELS that can adversely affect the normal blood flow in ARTERIES or VEINS. Most are congenital defects such as abnormal communications between blood vessels (fistula), shunting of arterial blood directly into veins bypassing the CAPILLARIES (arteriovenous malformations), formation of large dilated blood blood-filled vessels (cavernous angioma), and swollen capillaries (capillary telangiectases). In rare cases, vascular malformations can result from trauma or diseases.
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.