Study to Evaluate Safety and Efficacy of Intracystic TARA-002 for Macrocystic and Mixed Cystic Lymphatic Malformations in Children and Adolescents 6 Months to Less Than 18 Years

What this Trial Is About

Researchers are studying the safety, immune response, and effectiveness of TARA-002 administered by injection directly into cysts in children and adolescents aged 6 months to less than 18 years with macrocystic and mixed cystic lymphatic malformations. This Phase 2a/b open-label study includes an initial safety lead-in phase with older children aged 6 to less than 18 years, followed by younger age groups, before expanding enrollment. The purpose is to evaluate TARA-002 for these lymphatic malformations which are abnormal lymphatic vessel growths in the head, neck, or mediastinum areas. Participants will receive up to four intracystic injections of TARA-002 spaced about six weeks apart. The study drug is a biological preparation containing treated cells from Streptococcus pyogenes bacteria. The Phase 2a safety lead-in assesses safety in age-defined groups stepwise, and Phase 2b enrolls more participants once safety is established. Each injection cycle is carefully monitored to assess reactions and efficacy. During the study, participants will undergo imaging and clinical assessments to confirm lymphatic malformation type and response to treatment. Researchers will evaluate clinical success about eight weeks after the last injection and durability of response up to 32 weeks later. Safety monitoring includes tracking local and systemic reactions and adverse events throughout the treatment and follow-up periods. Quality of life and pain assessments will also be collected. Total participation spans several months including treatment and observation phases.

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