Lymphatic MalformationPhase 2STARBORN-1NCT05871970
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
A Phase 2 study testing TARA-002 in people with Lymphatic Malformation, run by Protara Therapeutics at 11 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Lymphatic Malformation
- What is being tested?
- TARA-002
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 11 sitesUnited States
- Study dates
- 18 Oct 2023to May 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
11 sites in 1 country. Contact the study team or a site near you to ask about taking part.
11 sites in 1 country
United States11 sites, 9 recruiting
- Recruiting
Children's Hospital of Colorado
Aurora, Colorado, 88045
MATTHEW MCCLUNG, matthew.mcclung@childrenscolorado.org
- Recruiting
Children's Hospital of Alabama
Birmingham, Alabama, 35294
TAYLOR BRINKLEY, tebrinkley@uabmc.edu
- Withdrawn
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
- Recruiting
Nemours Children's Clinic - Jacksonville
Jacksonville, Florida, 32207
ROSITA ALMIRA, rosita.almira@nemours.org
- Recruiting
Arkansas Children's Hospital/UAMS
Little Rock, Arkansas, 72202
LEKIVIA COBB, cobblc@archildrens.org
- Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
KALPNABEN PATEL, kalpnaben.patel@vumc.org
- Recruiting
Mount Sinai Hospital
New York, New York, 10029
ARMAND HARB, armand.harb@mountsinai.org
- Recruiting
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
LAURA STONE, stonelj@odu.edu
- Withdrawn
Children's Hospital of Philadelphia: Comprehensive Vascular Anomalies Program
Philadelphia, Pennsylvania, 19104
- Recruiting
Oregon Health & Science University
Portland, Oregon, 97239
LORI RUSSELL, watsonlo@ohsu.edu
- Recruiting
Children's National Medical Center: Children's Research Institute
Washington D.C., District of Columbia, 20010
HENGAMEH BEHZADPOUR, hbehzadpou@childrensnational.org
About this study
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
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.
- Conditions
- Lymphatic Malformation
- Being tested
- TARA-002
- Keywords
- Macrocystic lymphatic malformations, Mixed-cystic lymphatic malformations
- Medical subject headings
- Lymphatic Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TARA-002Biological
All participants will receive up to 4 intracystic injections spaced approximately 6 weeks apart.
| Study group | Role | What they receive |
|---|---|---|
| TARA-002 | Experimental | TARA-002 is a lyophilized biological preparation for injection containing cells of Streptococcus pyogenes (Group A, type 3) Su strain treated with benzylpenicillin. Receives: Biological: TARA-002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants with clinical success after one treatment cycle of TARA-002
Measured: 8 weeks after the last injection
Secondary outcomes (7)
Safety: Percentage of participants with solicited local site and systemic reactions
Measured: 14 days (2 weeks) after each injection
Safety: Percentage of participants with unsolicited adverse events (AEs)
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 8 weeks after last injection and 32 weeks after last injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2023
- Study start18 Oct 2023
- Primary completionDec 2026 (estimated)
- Study completionMay 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lymphatic MalformationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Protara Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chief R&D Officer, Study director, Protara Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05871970
- Sponsor's protocol ID
- TARA-002-201
- Acronym
- STARBORN-1
- Status last verified
- Apr 2026
- First submitted
- 11 May 2023
- Last updated
- 24 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05871970
- Acronym / sponsor ID
- STARBORN-1 / TARA-002-201
- Conditions
- Lymphatic Malformation
- Keywords
- Macrocystic lymphatic malformations, Mixed-cystic lymphatic malformations
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- Protara TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 18 Oct 2023 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- May 2027 (estimated)
- First posted
- 23 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TARA-002 | Experimental | Biological: TARA-002TARA-002 is a lyophilized biological preparation for injection containing cells of Streptococcus pyogenes (Group A, type 3) Su strain treated with benzylpenicillin. |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TARA-002All participants will receive up to 4 intracystic injections spaced approximately 6 weeks apart. | Biological | TARA-002 |
Primary outcome
Proportion of participants with clinical success after one treatment cycle of TARA-002
Time frame: 8 weeks after the last injection
7 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Chief R&D Officer study director
Protara Therapeutics
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing TARA-002 in people with Lymphatic Malformation, run by Protara Therapeutics at 11 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chief R&D Officer (16468440337, clinicaltrials@protaratx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants with clinical success after one treatment cycle of TARA-002 (time frame: 8 weeks after the last injection). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2026.