Back to resultsSafety 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)
Primary Purpose
Lymphatic Malformation
Status
Not yet recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
TARA-002
Sponsored by
About this trial
This is an interventional treatment trial for Lymphatic Malformation focused on measuring Macrocystic lymphatic malformations, Mixed-cystic lymphatic malformations
Eligibility 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.
Sites / Locations
- Children's Hospital of the King's Daughters
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
TARA-002
Arm Description
TARA-002 is a lyophilized biological preparation for injection containing cells of Streptococcus pyogenes (Group A, type 3) Su strain treated with benzylpenicillin.
Outcomes
Primary Outcome Measures
Proportion of participants with clinical success after one treatment cycle of TARA-002
Secondary Outcome Measures
Safety: Percentage of participants with solicited local site and systemic reactions
Safety: Percentage of participants with unsolicited adverse events (AEs)
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
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
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
Investigator Assessment: Proportion of participants who demonstrate clinical improvement, as assessed by the Investigator, after one treatment cycle of TARA-002 compared to baseline
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
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05871970
Brief Title
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
Acronym
STARBORN-1
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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
October 2023 (Anticipated)
Primary Completion Date
December 2025 (Anticipated)
Study Completion Date
May 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Protara Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lymphatic Malformation
Keywords
Macrocystic lymphatic malformations, Mixed-cystic lymphatic malformations
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
38 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
TARA-002
Arm Type
Experimental
Arm Description
TARA-002 is a lyophilized biological preparation for injection containing cells of Streptococcus pyogenes (Group A, type 3) Su strain treated with benzylpenicillin.
Intervention Type
Biological
Intervention Name(s)
TARA-002
Intervention Description
All participants will receive up to 4 intracystic injections spaced approximately 6 weeks apart.
Primary Outcome Measure Information:
Title
Proportion of participants with clinical success after one treatment cycle of TARA-002
Time Frame
8 weeks after the last injection
Secondary Outcome Measure Information:
Title
Safety: Percentage of participants with solicited local site and systemic reactions
Time Frame
14 days (2 weeks) after each injection
Title
Safety: Percentage of participants with unsolicited adverse events (AEs)
Time Frame
Through study completion (approximately 32 weeks after last injection)
Title
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)
Title
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
Title
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
Title
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
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Chief Scientific Operations Officer
Phone
16468440337
Email
clinicaltrials@protaratx.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Chief Scientific Operations Officer
Organizational Affiliation
Protara Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital of the King's Daughters
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23507
Country
United States
12. IPD Sharing Statement
Learn more about this trial
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
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