Actively Recruiting

Phase 2
Age: 6Months - 18Years
All Genders
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

Led by Protara Therapeutics · Updated on 2025-10-23

38

Participants Needed

10

Research Sites

132 weeks

Total Duration

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AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 6Months - 18Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Male or female participants 6 months to less than 18 years of age at the time of informed consent/assent
  • Parent or legal representative has given written consent and participant provided assent if applicable
  • Diagnosis of macrocystic or mixed cystic lymphatic malformations with at least 50% macrocystic disease in the head, neck, or mediastinum confirmed by imaging
  • Participants may have had surgery or sclerotherapy for lymphatic malformations, but not within 6 months before consent/assent
Not Eligible

You will not qualify if you...

  • Allergy to penicillin
  • Presence of vascular tumors or combined vascular malformations
  • Microcystic lymphatic malformations or mixed cystic lymphatic malformations with mainly microcystic features
  • Lymphatic malformations located in the orbit (orbital lymphatic malformations) as the target cyst

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 10 locations

1

Children's Hospital of Alabama

Birmingham, Alabama, United States, 35294

Actively Recruiting

2

Arkansas Children's Hospital/UAMS

Little Rock, Arkansas, United States, 72202

Actively Recruiting

3

Children's Hospital of Colorado

Aurora, Colorado, United States, 88045

Actively Recruiting

4

Children's National Medical Center: Children's Research Institute

Washington D.C., District of Columbia, United States, 20010

Actively Recruiting

5

Nemours Children's Clinic - Jacksonville

Jacksonville, Florida, United States, 32207

Actively Recruiting

6

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

Actively Recruiting

7

Oregon Health & Science University

Portland, Oregon, United States, 97239

Actively Recruiting

8

Children's Hospital of Philadelphia: Comprehensive Vascular Anomalies Program

Philadelphia, Pennsylvania, United States, 19104

Withdrawn

9

Vanderbilt University Medical Center

Nashville, Tennessee, United States, 37232

Actively Recruiting

10

Children's Hospital of the King's Daughters

Norfolk, Virginia, United States, 23507

Actively Recruiting

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Research Team

C

Chief Scientific Operations Officer

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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