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

Lymphatic Malformation Clinical Trial

— STARBORN-1  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05871970
• Other study ID #: TARA-002-201
• Status: Recruiting
• Phase: Phase 2
• Start date: October 18, 2023
• Est. completion date: May 2026

Study information

• Verified date: May 2024
• Source: Protara Therapeutics
• Contact: Chief Scientific Operations Officer
• Phone: 16468440337
• Email: clinicaltrials[@]protaratx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 38
• Est. completion date: May 2026
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Congenital Abnormalities
• Lymphangioma
• Lymphatic Abnormalities
• Lymphatic Malformation

Intervention

Biological:
• TARA-002
All participants will receive up to 4 intracystic injections spaced approximately 6 weeks apart.

Locations

Country	Name	City	State
United States	Children's Hospital of Alabama	Birmingham	Alabama
United States	Nemours Children's Clinic - Jacksonville	Jacksonville	Florida
United States	Arkansas Children's Hospital/UAMS	Little Rock	Arkansas
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Children's Hospital of the King's Daughters	Norfolk	Virginia
United States	Children's National Medical Center: Children's Research Institute	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Protara Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of participants with clinical success after one treatment cycle of TARA-002		8 weeks after the last injection
Secondary	Safety: Percentage of participants with solicited local site and systemic reactions		14 days (2 weeks) after each injection
Secondary	Safety: Percentage of participants with unsolicited adverse events (AEs)		Through study completion (approximately 32 weeks after last injection)
Secondary	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)
Secondary	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
Secondary	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
Secondary	Investigator Assessment: Proportion of participants who demonstrate clinical improvement, as assessed by the Investigator, after one treatment cycle of TARA-002 compared to baseline		8 weeks after last injection and 32 weeks after last injection
Secondary	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		8 weeks after last injection and 32 weeks after last injection