Skip to main content
OpenTrials
Recruiting

NCT Number: 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

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

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

Age range

6 month–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

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

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

TARA-002

Biological

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

Primary outcomes

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

    Time frame: 8 weeks after the last injection

Secondary outcomes

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

    Time frame: 14 days (2 weeks) after each injection

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

    Time frame: Through study completion (approximately 32 weeks after last injection)

  3. 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

    Time frame: Through study completion (approximately 32 weeks after last injection)

  4. 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

    Time frame: 32 weeks after the last injection

  5. 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

    Time frame: 8 weeks after the last injection

  6. Investigator Assessment: Proportion of participants who demonstrate clinical improvement, as assessed by the Investigator, after one treatment cycle of TARA-002 compared to baseline

    Time frame: 8 weeks after last injection and 32 weeks after last injection

  7. Quality of Life: Change in Quality-of-Life assessment based on Pediatric Quality of Life Inventory (PedsQL) and Visual Analog Scale (VAS) for Pain after one treatment cycle of TARA-002

    Time frame: 8 weeks after last injection and 32 weeks after last injection

Study contacts

Contact information is provided by the study sponsor or research team.

Chief Scientific Operations Officer

CONTACT

[email protected]

16468440337

Sponsors and collaborators

Lead sponsor

Protara Therapeutics

Industry

Registry information

Official study 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

Acronym: STARBORN-1

Important dates

Study start
2023
Primary completion
2026
Study completion
2027
First posted
May 23, 2023
Registry last updated
May 18, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.