Clinical trial NCT05871970: 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
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
Overall status: RECRUITING
Phase: PHASE2
Conditions: Lymphatic Malformation
Keywords: Macrocystic lymphatic malformations
Mixed-cystic lymphatic malformations
Lead sponsor: Protara Therapeutics
Sponsor class: INDUSTRY
First posted: 2023-05-23
Enrollment: 38
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Proportion of participants with clinical success after one treatment cycle of TARA-002
timeFrame: 8 weeks after the last injection
Secondary outcomes:
measure: Safety: Percentage of participants with solicited local site and systemic reactions
timeFrame: 14 days (2 weeks) after each injection
measure: Safety: Percentage of participants with unsolicited adverse events (AEs)
timeFrame: Through study completion (approximately 32 weeks after last injection)
measure: 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
timeFrame: Through study completion (approximately 32 weeks after last injection)
measure: 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
timeFrame: 32 weeks after the last injection
measure: 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
timeFrame: 8 weeks after the last injection
measure: Investigator Assessment: Proportion of participants who demonstrate clinical improvement, as assessed by the Investigator, after one treatment cycle of TARA-002 compared to baseline
timeFrame: 8 weeks after last injection and 32 weeks after last injection
measure: 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
timeFrame: 8 weeks after last injection and 32 weeks after last injection
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Children's Hospital of Alabama
state: Alabama
city: Little Rock
country: United States
facility: Arkansas Children's Hospital/UAMS
state: Arkansas
city: Aurora
country: United States
facility: Children's Hospital of Colorado
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center: Children's Research Institute
state: District of Columbia
city: Jacksonville
country: United States
facility: Nemours Children's Clinic - Jacksonville
state: Florida
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia: Comprehensive Vascular Anomalies Program
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Norfolk
country: United States
facility: Children's Hospital of the King's Daughters
state: Virginia
Study officials:
affiliation: Protara Therapeutics
name: Chief Scientific Operations Officer
role: STUDY_DIRECTOR
References: