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 (STARBORN-1)

Why are we doing this research?

The main reason for this research study is to test the safety, side effects, and efficacy (how well the drug works) of an experimental study drug called TARA-002 that is being developed for treating Lymphatic Malformations (LMs).

Who can participate?

You may take part in this study if you are 6 months of age to less than 18 years of age with macrocystic and mixed cystic LMs.

Conditions

Hematology - Hemangioma and Vascular Malformation

What will happen in the study?

The study drug will be administered into one cyst, referred to as the “target cyst” for treatment. The study drug will be given a series of up to 4 injections into the target cyst which occur approximately 6 weeks apart. Participants will receive all 4 injections unless the target cyst becomes no longer injectable (for example, if by physical exam and/or ultrasound, it is noted that there is no remaining cyst to Inject.)

We expect that you will be in this research study for a total of 54 weeks, if you receive all 4 injections. If you are not benefiting from the therapy, the research doctor can stop treatment at any time during the study.

What are the good things that can happen from this research?

We cannot promise any benefits to you or others from your taking part in this research. However, taking part in this study could possibly help to treat your lymphatic malformation(s). Your condition could improve, stay the same, or get worse. The data collected from you during this study may help doctors learn more about the study drug and your disease, which may help future participants.

What are the bad things that can happen from this research?

The drug used in this study may affect how different parts of your body work such as your liver, kidneys, heart, and blood, causing side effects.

Will you/your child be paid to be in this research study?

If you agree to take part in this research study, we will pay you $50.00 per visit, or $400.00 total. You (your child) will be reimbursed for your time, effort and travel (if applicable) while you are in this research study.

Clinical Trials / Research Studies
Clinical Trials / Research Studies