NCT05871970

Safety and Efficacy Study of Intracystic TARA-002 for the Treatment of Lymphatic Malformations in Participants 6 Months to Less Than 18 Years of Age

A Phase 2a/b Single Arm Open Label Study to Evaluate the Safety and Efficacy of Intracystic Administration of TARA-002 in Participants Between 6 Months to Less Than 18 Years of Age for the Treatment of Macrocystic and Mixed Cystic Lymphatic Malformations

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 38 (estimated)
• Sponsor: Protara Therapeutics · Industry
• Sex: All
• Age: 6 Months – 18 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2a/b single arm open label study to evaluate the safety, reactogenicity, and efficacy of intracystic injection of TARA-002 in participants 6 months to less than 18 years of age for the treatment of macrocystic and mixed cystic lymphatic malformations. The Phase 2a safety lead-in, age de-escalation study is designed to establish the safety of TARA-002 in older participants 6 years to less than 18 years before proceeding to younger participants 2 years to less than 6 years, then 6 months to less than 2 years. The Phase 2b is an expansion study in which enrollment of participants will be initiated after safety has been established in each cohort during the Phase 2a safety lead-in study. Each participant will receive up to 4 injections of TARA-002 spaced approximately 6 weeks apart.

Conditions

• Lymphatic Malformation

Interventions

Type	Name	Description
BIOLOGICAL	TARA-002	All participants will receive up to 4 intracystic injections spaced approximately 6 weeks apart.

Timeline

• Start date: 2023-10-18
• Primary completion: 2025-12-01
• Completion: 2026-05-01
• First posted: 2023-05-23
• Last updated: 2025-10-23

Locations

10 sites across 1 country: United States

Regulatory

• FDA-regulated drug study