A Phase 2 Trial of ART-001 for Slow-Flow Vascular Malformations
Background
Patients with slow-flow vascular malformations (SFVMs) such as venous malformations (VM), lymphatic malformations (LM), or Klippel-Trenaunay Syndrome (KTS) often have specific mutations in the PI3Kα gene. This study tested ART-001 (serabelisib), a selective PI3Kα inhibitor, to see how well it works and how safe it is.
Methods
This was a multicenter, randomized, double-blind trial. Participants aged 2 years and older with VM, LM, or KTS received either 50 mg or 100 mg of ART-001 for 24 weeks. The main goal was to see if there was a ≥ 20% reduction in lesion volume at week 24. We also looked at safety, pain levels, and quality of life.
Results
A total of 35 patients participated (average age: 14 years; VM: 17, KTS: 13, LM: 5). The response rates were:
- 50 mg dose: 29.4% (10.3-56.0% confidence interval)
- 100 mg dose: 33.3% (13.3-59.0% confidence interval)
Mean reductions in lesion volume were:
- 50 mg: -2.3%
- 100 mg: -12.6%
No serious side effects were linked to the drug. Most side effects were mild to moderate and temporary. The drug’s effects on the body were similar in children and adults, although children had lower drug levels.
Conclusion
ART-001 was effective and well-tolerated for treating SFVMs. These findings encourage further research on ART-001 for SFVMs and related conditions to confirm its benefits and safety.
Trial Registration
Japan Registry of Clinical Trial, jRCT2071210027. Registered May 25, 2021. View Trial Details.
Clinical Trial Importance
Clinical trials are essential for developing safe treatments. To bring these advancements into everyday healthcare, we offer DocSym, an AI-driven platform that combines clinical standards and research for easy access by healthcare providers.
Streamlining Healthcare
Our mobile apps facilitate scheduling, treatment monitoring, and telemedicine, simplifying patient care management and enhancing service delivery.
Improving Outcomes with AI
By integrating AI, clinics can optimize workflows, improve patient outcomes, and reduce paperwork. Discover more about our solutions at aidevmd.com.
