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.
Male or female participants 6 months to less than 18 years of age at the time of informed consent/assent form was signed
Participants whose parent/LAR(s) have voluntarily given written consent and participants who provided assent (if applicable) after the study has been explained to them
Participants with macrocystic LM or mixed cystic LM (≥ 50% macrocystic disease measured by volume) of the Head/Neck/Mediastinum according to the ISSVA 2018 criteria (ISSVA 2018) measured via LM imaging at Screening to confirm, upon central review, the diagnosis of macrocystic or mixed cystic LM
Participants who may have had surgical or sclerotherapy treatment for their LM, but not within six months of the consent/assent form being signed
Penicillin allergy
Vascular tumors or combined vascular malformations
Microcystic LM or mixed cystic LM with predominant microcystic features
LMs of the orbit (orbital LM) as target cyst
Single group
TARA-002
Biological/VaccineAll participants will receive up to 4 intracystic injections spaced approximately 6 weeks apart.
Proportion of participants with clinical success after one treatment cycle of TARA-002
Safety: Percentage of participants with solicited local site and systemic reactions
Safety: Percentage of participants with unsolicited adverse events (AEs)
Safety: Percentage of participants with SAEs, AEs of special interest, AEs leading to premature discontinuation of study intervention, AEs leading to withdrawal from study, AEs with an outcome of death, and MAAEs
Durable Response: Proportion of participants with clinical success after one treatment cycle of TARA-002 assessed at 8 weeks after the last injection and maintained clinical success at 32 weeks after the last injection
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