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Treatment of lymphatic malformations in children using TARA-002

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

PHASE2 · Protara Therapeutics · NCT05871970

This study is testing if a new treatment called TARA-002 can safely help reduce lymphatic malformations in children aged 6 months to under 18 years.

Quick facts

PhasePHASE2
Study typeInterventional
Enrollment38 (estimated)
Ages6 Months to 18 Years
SexAll
SponsorProtara Therapeutics (industry)
Locations10 sites (Birmingham, Alabama and 9 other locations)
Trial IDNCT05871970 on ClinicalTrials.gov

What this trial studies

This Phase 2a/b open-label study evaluates the safety and efficacy of TARA-002, administered via intracystic injection, for treating macrocystic and mixed cystic lymphatic malformations in children aged 6 months to less than 18 years. The study begins with a safety lead-in phase for older participants before expanding to younger cohorts. Participants will receive up to four injections spaced approximately six weeks apart, with safety being established at each age level before proceeding to the next. The study aims to confirm the effectiveness of TARA-002 in reducing lymphatic malformations.

Who should consider this trial

Good fit: Ideal candidates are children aged 6 months to less than 18 years with macrocystic or mixed cystic lymphatic malformations.

Not a fit: Patients with microcystic lymphatic malformations or those with vascular tumors may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for children suffering from lymphatic malformations, potentially improving their quality of life.

How similar studies have performed: While this approach is novel, similar studies targeting lymphatic malformations have shown promise, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

For more information on eligibility criteria, please contact the sponsor.

Where this trial is running

Birmingham, Alabama and 9 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Lymphatic Malformation, Macrocystic lymphatic malformations, Mixed-cystic lymphatic malformations

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.