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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)

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

Study Description
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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.

Condition or disease Intervention/treatment Phase
Lymphatic Malformation Biological: TARA-002 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 38 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : October 18, 2023
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : May 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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.
 Biological: TARA-002
All participants will receive up to 4 intracystic injections spaced approximately 6 weeks apart.


Outcome Measures
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Primary Outcome Measures :
  1. Proportion of participants with clinical success after one treatment cycle of TARA-002 [ Time Frame: 8 weeks after the last injection ]

Secondary Outcome Measures :
  1. Safety: Percentage of participants with solicited local site and systemic reactions [ Time Frame: 14 days (2 weeks) after each injection ]
  2. Safety: Percentage of participants with unsolicited adverse events (AEs) [ Time Frame: Through study completion (approximately 32 weeks after last injection) ]
  3. 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) ]
  4. 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 ]
  5. 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 ]
  6. 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 ]
  7. 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 ]

Eligibility Criteria
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Ages Eligible for Study:   6 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05871970


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

Locations
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United States, Alabama
Children's Hospital of Alabama Recruiting
Birmingham, Alabama, United States, 35294
Contact: Use Central Contact         
United States, Arkansas
Arkansas Children's Hospital/UAMS Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Use Central Contact         
United States, District of Columbia
Children's National Medical Center: Children's Research Institute Recruiting
Washington, District of Columbia, United States, 20010
Contact: Use Central Contact         
United States, Florida
Nemours Children's Clinic - Jacksonville Recruiting
Jacksonville, Florida, United States, 32207
Contact: Use Central Contact         
United States, Tennessee
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States, 37232
Contact: Use Central Contact         
United States, Virginia
Children's Hospital of the King's Daughters Recruiting
Norfolk, Virginia, United States, 23507
Contact: Use Central Contact         
Sponsors and Collaborators
Protara Therapeutics
Investigators
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Study Director: Chief Scientific Operations Officer Protara Therapeutics
More Information
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Responsible Party: Protara Therapeutics
ClinicalTrials.gov Identifier: NCT05871970    
Other Study ID Numbers: TARA-002-201
First Posted: May 23, 2023    Key Record Dates
Last Update Posted: May 3, 2024
Last Verified: May 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Protara Therapeutics:
Macrocystic lymphatic malformations
Mixed-cystic lymphatic malformations
Additional relevant MeSH terms:
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Lymphangioma
Lymphatic Abnormalities
Congenital Abnormalities
Lymphatic Vessel Tumors
 Neoplasms by Histologic Type
Neoplasms
Lymphatic Diseases