Phase 1 Vascular Malformations Clinical Trials

2 recruitingPhase 1

What is a Phase 1 trial?

Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.

Showing 1–2 of 2 trials

Recruiting

Phase 1Phase 2

Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CYH33 in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)

PIK3CA-Related Overgrowth Spectrum (PROS)PIK3CA-related Vascular Malformations (PRVM)

Haihe Biopharma Co., Ltd.141 enrolled4 locationsNCT06975618

Recruiting

Phase 1

5 year study of 6 children each year with vascular anomalies who have severe complications failing to respond to the usual treatments will be offered a new use of an old medication- sirolimus

PIKC3A related overgrowth syndrome (PROS)Vascular tumours (tufted angiomas and kaposiform haemangioendotheliomas)Complex lymphatic malformations+2 more

Sydney Children's Hospital, Randwick30 enrolled1 locationACTRN12616000943448