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)

Protara Therapeutics

Status and phase: Enrolling; Phase 2

Conditions: Lymphatic Malformation

Treatments: Biological: TARA-002

Study type: Interventional

Funder types: Industry

Identifiers

NCT05871970

TARA-002-201

Details and patient eligibility

About

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.

Enrollment: 38 estimated patients

Sex: All

Ages: 6 months to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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38 participants in 1 patient group

TARA-002

Experimental group

Description:

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

Treatment:

Biological: TARA-002