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

NCT05871970 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: TARA-002-201
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 10

Brief Summary

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

Lymphatic Malformation

Keywords

Macrocystic lymphatic malformations; Mixed-cystic lymphatic malformations

Outcome Measures

Primary Outcomes (1)

• Proportion of participants with clinical success after one treatment cycle of TARA-002

  8 weeks after the last injection

Secondary Outcomes (7)

• Safety: Percentage of participants with solicited local site and systemic reactions

  14 days (2 weeks) after each injection

• Safety: Percentage of participants with unsolicited adverse events (AEs)

  Through study completion (approximately 32 weeks after last injection)

• 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

  Through study completion (approximately 32 weeks after last injection)

• 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

  32 weeks after the last injection

• Clinical Success by LM Type: Proportion of participants with macrocystic LM or mixed cystic LM with clinical success after one treatment cycle of TARA-002

  8 weeks after the last injection

Study Arms (1)

TARA-002

EXPERIMENTAL

TARA-002 is a lyophilized biological preparation for injection containing cells of Streptococcus pyogenes (Group A, type 3) Su strain treated with benzylpenicillin.

Biological: TARA-002

Interventions

TARA-002 BIOLOGICAL

All participants will receive up to 4 intracystic injections spaced approximately 6 weeks apart.

TARA-002

Eligibility Criteria

• Age: 6 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Protara Therapeutics: lead

Study Sites (10)

Children's Hospital of Alabama

Birmingham, Alabama, 35294, United States

RECRUITING

Arkansas Children's Hospital/UAMS

Little Rock, Arkansas, 72202, United States

RECRUITING

Children's Hospital of Colorado

Aurora, Colorado, 88045, United States

RECRUITING

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

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

RECRUITING

Nemours Children's Clinic - Jacksonville

Jacksonville, Florida, 32207, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Oregon Health & Science University

Portland, Oregon, 97239, United States

RECRUITING

Children's Hospital of Philadelphia: Comprehensive Vascular Anomalies Program

Philadelphia, Pennsylvania, 19104, United States

WITHDRAWN

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

RECRUITING

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23507, United States

RECRUITING

MeSH Terms

Conditions

Lymphatic Abnormalities

Condition Hierarchy (Ancestors)

Lymphatic Diseases; Hemic and Lymphatic Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Chief Scientific Operations Officer

  Protara Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Chief Scientific Operations Officer

CONTACT

16468440337; clinicaltrials@protaratx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 11, 2023
• First Posted: May 23, 2023
• Study Start: October 18, 2023
• Primary Completion: December 1, 2025
• Study Completion: May 1, 2026
• Last Updated: October 23, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)