TARA-002 Injection for Lymphatic Malformations

(STARBORN-1 Trial)

Recruiting at 9 trial locations

Overseen By Chief Scientific Operations Officer

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Protara Therapeutics

Disqualifiers: Penicillin allergy, Vascular tumors, Microcystic LM, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

38 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called TARA-002 for children and teens with specific types of lymphatic malformations, where lymph vessels form cysts in areas like the head or neck. The goal is to assess the safety and effectiveness of the treatment when injected directly into the cysts. Researchers aim to understand how the body reacts to this treatment and whether it reduces the size of the cysts. Children with macrocystic lymphatic malformations in the head, neck, or chest area who haven't received treatment in the last six months might be suitable candidates. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to contact the trial sponsor for more details.

Is there any evidence suggesting that TARA-002 is likely to be safe for humans?

Research has shown that TARA-002 is being carefully monitored for safety in treating lymphatic malformations, which are cyst-like growths. Previous patients demonstrated that TARA-002 is generally well-tolerated, with most people not experiencing serious side effects. The treatment involves injecting a special preparation of bacteria cells that have been made safe.

The studies prioritize safety, especially for children of different ages. Doctors administer the treatment in several doses over time, allowing them to closely monitor for any reactions. Although this treatment is still under investigation, its presence in a Phase 2 trial indicates that earlier testing showed it was safe enough to continue. Always consult a doctor to determine if joining a trial is appropriate. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

TARA-002 is unique because it uses a lyophilized biological preparation containing cells of Streptococcus pyogenes treated with benzylpenicillin. Unlike other treatments for lymphatic malformations, which often involve surgery or sclerotherapy, TARA-002 offers a different approach by potentially harnessing the body's immune response. Researchers are excited about this treatment because it represents a novel method of addressing the condition, with the potential for fewer side effects and a non-invasive administration.

What evidence suggests that TARA-002 might be an effective treatment for lymphatic malformations?

Research has shown that TARA-002, the investigational treatment in this trial, offers promising results for treating lymphatic malformations. In earlier studies, TARA-002 completely eliminated the problem in some cases and significantly reduced it in others over 12 months. This treatment effectively shrinks or eliminates troublesome cysts. TARA-002 works by injecting a specially prepared form of bacteria into the cysts, helping the body fight and reduce them. Although still under study, these results suggest it could effectively manage lymphatic malformations in young patients. ^{5 6 7 8 9}

Who Is on the Research Team?

Chief Scientific Operations Officer

Principal Investigator

Protara Therapeutics

Are You a Good Fit for This Trial?

This trial is for kids and teens from 6 months to under 18 years with large or mixed fluid-filled lumps in the head, neck, or chest area. They can join if they've had treatments before but not within the last six months. Kids allergic to penicillin, with blood vessel tumors, mostly small cysts, or eye socket involvement can't participate.

Inclusion Criteria

I had surgery or sclerotherapy for my LM, but not in the last 6 months.

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

I am between 6 months and 18 years old.

Exclusion Criteria

My condition involves small cysts in my lymphatic system.

Penicillin allergy

My target cyst is in the eye socket area.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 2a Safety Lead-in

Safety lead-in, age de-escalation study to establish the safety of TARA-002 in older participants before proceeding to younger cohorts

Variable, based on cohort completion

Phase 2b Expansion

Expansion study where participants receive up to 4 injections of TARA-002 spaced approximately 6 weeks apart

18-24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• TARA-002

Trial Overview The study tests TARA-002 injections directly into these lumps. It's a two-part study: first ensuring it's safe for older kids (6-17 years) then younger ones (6 months-5 years). If safe, more kids will get up to four shots about six weeks apart in the second part of the study.

How Is the Trial Designed?

Group I: TARA-002 Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Protara Therapeutics

Lead Sponsor

Trials

5

Recruited

270+

Published Research Related to This Trial

An estimated 3200 pediatric admissions for lymphatic malformation (LM) occurred in 1997 and 2000, primarily in urban teaching hospitals, indicating a significant healthcare burden for this condition.

Surgical excision was the most common treatment for LM, especially in children under 2 years old, while hospital charges for these admissions increased from $26 million in 1997 to $35 million in 2000, reflecting rising healthcare costs.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pediatric admissions and procedures for lymphatic malformations in the United States: 1997 and 2000. Harsha, WJ., Perkins, JA., Lewis, CW., et al.[2007]

In a study of 131 patients treated with the immunostimulant OK-432 for lymphatic malformations, 70% achieved good or excellent clinical outcomes, indicating its efficacy as a treatment option.

The study found that factors such as the number of injections and lesion localization influenced treatment outcomes, while the time to follow-up and cyst size did not significantly affect results.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patients with lymphatic malformations who receive the immunostimulant OK-432 experience excellent long-term outcomes. Ghaffarpour, N., Petrini, B., Svensson, LA., et al.[2015]

Endoscopic partial resection combined with cautery and postoperative treatments resulted in a higher cure rate (78.0%) for lymphatic malformation in infants compared to other methods, such as pinyangmycin and dexamethasone injection (69.0%) and surgical resection (72.1%).

The study identified that the range of the lymphatic malformation and its histological type are significant independent factors affecting treatment outcomes, particularly in cervicofacial cases, highlighting the need for tailored therapeutic approaches.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Management for lymphatic malformation in infants: a single center experience]. Han, T., Zou, J., Chen, H., et al.[2018]

In a study of 112 children who underwent surgery for lymphatic malformations, those younger than 1 year had a significantly higher risk of permanent postoperative complications, particularly neurological issues like peripheral facial palsy.

Microcystic malformations and cervicofacial locations were strongly associated with complications, suggesting that less aggressive surgical approaches may be preferable to minimize risks, as non-radical resections showed fewer complications without compromising long-term cure rates.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Risk Factors for Sequelae After Surgery for Lymphatic Malformations in Children. Moreno-Alfonso, JC., Triana, P., Miguel Ferrero, M., et al.[2023]

Intralesional injection of the sclerosing agent Bleomycin significantly reduced the size of macrocystic lymphatic malformations (LMs) in children, with a mean size decrease from 55.2 mm to 23.8 mm, indicating its efficacy as a treatment option.

While Bleomycin was less effective for microcystic lesions, it still allowed for surgical resection, suggesting it can be a useful part of a treatment plan for various types of LMs.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficiency and clinical results of bleomycin injection in the treatment of lymphatic malformations. Siki, FO., Sarikaya, M., Gunduz, M., et al.[2023]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05871970?term=AREA%5BConditionSearch%5D(%22Lymphatic%20Abnormalities%22)&rank=6

Safety and Efficacy Study of Intracystic TARA-002 for the ... Data collected at the beginning of a clinical study for all participants and for each arm or comparison group. These data include demographics, such as age, sex ...

2. ir.protaratx.com ir.protaratx.com/news-releases/news-release-details/protara-therapeutics-announces-positive-interim-results

Press Release TARA-002 demonstrates 100% complete response rate at any time and 67% 12-month landmark complete response rate in BCG-Unresponsive patients

3. chop.edu chop.edu/research-studies/tara-002-lymphatic-malformations

Tara-002 in Lymphatic Malformations The purpose of this study is to find out if the study drug is safe and effective (can help) people with LMs. Participants receive the study drug by injection ...

4. ctv.veeva.com ctv.veeva.com/study/safety-and-efficacy-study-of-intracystic-tara-002-for-the-treatment-of-lymphatic-malformations-in-pa

Safety and Efficacy Study of Intracystic TARA-002 for the ... This is a Phase 2a/b single arm open label study to evaluate the safety, reactogenicity, and efficacy of intracystic injection of TARA-002 ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT05871970

Safety and Efficacy Study of Intracystic TARA-002 for the ... 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 ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05871970

Safety and Efficacy Study of Intracystic TARA-002 for the ... 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 ...

7. cincinnatichildrens.org cincinnatichildrens.org/service/c/clinical-trials/studies/TARA-002-021425

Clinical Trials / Research Studies ... This research study is to test the safety, side effects, and efficacy (how well the drug works) of an experimental study drug called TARA-002.

8. ir.protaratx.com ir.protaratx.com/news-releases/news-release-details/protara-therapeutics-announces-completion-first-cohort-phase-2

Press Release TARA-002 has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of LMs. About ...

9. cancer.gov cancer.gov/clinicaltrials/NCI-2025-05432

Safety and Efficacy Study of Intracystic TARA-002 for the ... 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 ...