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 (STARBORN-1)

• ClinicalTrials.gov Identifier: NCT05871970
• Recruitment Status: Recruiting
• First Posted: May 23, 2023
• Last Update Posted: May 3, 2024
• Sponsor: Protara Therapeutics
• Information provided by (Responsible Party): Protara Therapeutics

Tracking Information

• First Submitted Date: May 11, 2023
• First Posted Date: May 23, 2023
• Last Update Posted Date: May 3, 2024
• Actual Study Start Date: October 18, 2023
• Estimated Primary Completion Date: December 2025 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: May 22, 2023): Proportion of participants with clinical success after one treatment cycle of TARA-002 [ Time Frame: 8 weeks after the last injection ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: May 22, 2023)

• Safety: Percentage of participants with solicited local site and systemic reactions [ Time Frame: 14 days (2 weeks) after each injection ]
• Safety: Percentage of participants with unsolicited adverse events (AEs) [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 8 weeks after the last injection ]
• 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 ]
• 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 ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief 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
• 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Lymphatic Malformation

Intervention

Biological: TARA-002

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

Study Arms

Experimental: TARA-002

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

Intervention: Biological: TARA-002

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: May 22, 2023): 38
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: May 2026
• Estimated Primary Completion Date: December 2025 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 6 Months to 18 Years (Child, Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Chief Scientific Operations Officer; 16468440337; clinicaltrials@protaratx.com

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT05871970
• Other Study ID Numbers: TARA-002-201
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Protara Therapeutics
• Original Responsible Party: Same as current
• Current Study Sponsor: Protara Therapeutics
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Chief Scientific Operations Officer; Protara Therapeutics

• PRS Account: Protara Therapeutics
• Verification Date: May 2024