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

Trial Summary

What is the purpose of this trial?

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.

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.

What data supports the effectiveness of the treatment TARA-002 for lymphatic malformations?

The treatment TARA-002 is similar to the immunostimulant OK-432, which has shown excellent long-term outcomes for patients with lymphatic malformations. This suggests that TARA-002 might also be effective for this condition. ^{1 2 3 4 5}

Research Team

Chief Scientific Operations Officer

Principal Investigator

Protara Therapeutics

Eligibility Criteria

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 target cyst is in the eye socket area.

My condition involves blood vessel tumors or malformations.

My condition involves small cysts in my lymphatic system.

Timeline

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

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]

References

1. China pubmed.ncbi.nlm.nih.gov

[Management for lymphatic malformation in infants: a single center experience]. [2018]

2. United States pubmed.ncbi.nlm.nih.gov

Risk Factors for Sequelae After Surgery for Lymphatic Malformations in Children. [2023]

3. Norway pubmed.ncbi.nlm.nih.gov

Patients with lymphatic malformations who receive the immunostimulant OK-432 experience excellent long-term outcomes. [2015]

4. United States pubmed.ncbi.nlm.nih.gov

Pediatric admissions and procedures for lymphatic malformations in the United States: 1997 and 2000. [2007]

5. India pubmed.ncbi.nlm.nih.gov

Efficiency and clinical results of bleomycin injection in the treatment of lymphatic malformations. [2023]