RECRUITING

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

Description

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.

Study Overview

Study Details

Study overview

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.

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

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

Condition
Lymphatic Malformation
Intervention / Treatment

-

Contacts and Locations

Birmingham

Children's Hospital of Alabama, Birmingham, Alabama, United States, 35294

Little Rock

Arkansas Children's Hospital/UAMS, Little Rock, Arkansas, United States, 72202

Aurora

Children's Hospital of Colorado, Aurora, Colorado, United States, 88045

Washington

Children's National Medical Center: Children's Research Institute, Washington, District of Columbia, United States, 20010

Jacksonville

Nemours Children's Clinic - Jacksonville, Jacksonville, Florida, United States, 32207

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229

Philadelphia

Children's Hospital of Philadelphia: Comprehensive Vascular Anomalies Program, Philadelphia, Pennsylvania, United States, 19104

Nashville

Vanderbilt University Medical Center, Nashville, Tennessee, United States, 37232

Norfolk

Children's Hospital of the King's Daughters, Norfolk, Virginia, United States, 23507

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

6 Months to 18 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Protara Therapeutics,

Chief Scientific Operations Officer, STUDY_DIRECTOR, Protara Therapeutics

Study Record Dates

2026-05