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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2023-10-18
Study Completion:2026-05
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05871970

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.

Ages Eligible for Study:6 Months to 18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Male or female participants 6 months to less than 18 years of age at the time of informed consent/assent form was signed
  2. * 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
  3. * 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
  4. * Participants who may have had surgical or sclerotherapy treatment for their LM, but not within six months of the consent/assent form being signed
  1. * Penicillin allergy
  2. * Vascular tumors or combined vascular malformations
  3. * Microcystic LM or mixed cystic LM with predominant microcystic features
  4. * LMs of the orbit (orbital LM) as target cyst

Contacts and Locations

Study Contact

Chief Scientific Operations Officer
CONTACT
16468440337
clinicaltrials@protaratx.com

Principal Investigator

Chief Scientific Operations Officer
STUDY_DIRECTOR
Protara Therapeutics

Study Locations (Sites)

Children's Hospital of Alabama
Birmingham, Alabama, 35294
United States
Arkansas Children's Hospital/UAMS
Little Rock, Arkansas, 72202
United States
Children's Hospital of Colorado
Aurora, Colorado, 88045
United States
Children's National Medical Center: Children's Research Institute
Washington, District of Columbia, 20010
United States
Nemours Children's Clinic - Jacksonville
Jacksonville, Florida, 32207
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Children's Hospital of Philadelphia: Comprehensive Vascular Anomalies Program
Philadelphia, Pennsylvania, 19104
United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
United States

Collaborators and Investigators

Sponsor: Protara Therapeutics

  • Chief Scientific Operations Officer, STUDY_DIRECTOR, Protara Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-10-18
Study Completion Date2026-05

Study Record Updates

Study Start Date2023-10-18
Study Completion Date2026-05

Terms related to this study

Keywords Provided by Researchers

  • Macrocystic lymphatic malformations
  • Mixed-cystic lymphatic malformations

Additional Relevant MeSH Terms

  • Lymphatic Malformation