Craniofacial – NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
What is the goal of this study?
The goal of this study is to assess the safety and efficacy of the medication NB-001 in children and adolescents with 22q11DS.
- Is NB-001, an investigational drug, safe and well-tolerated in children and adolescents with 22q11 Deletion Syndrome?
- Are there any changes in mood and behavior with use of the investigational drug?
- What are the blood levels of NB-001 during participation in this study?
Who can join the study?
To be part of this study, your child must match all traits in this list:
- Genetic test shows 22q11DS
- Age 6-17 years old
- Symptoms of anxiety, inattention, and/or autism
- Willing to use birth control (if needed)
- Understands and agrees to participate in the study
What will happen if my child takes part in this study?
To be in this study, you will be asked questions and your child will have tests.
The first visit will be in-person at the study doctor’s office.
All other study visits will be done in your home by a study nurse.*
- Your child will take study medicine two times each day: once in the morning and again around dinner time.
- A nurse will periodically come to your house to see how your child is doing, listen to your child's heart, take his/her temperature, and collect blood samples.
- Your child will be asked to pee in a cup so we can test his/her urine.
- Your child's heart beat will be measured through a common procedure called an electrocardiogram (EKG) during which small sticky pads will be placed on your child's body and legs and wires attached for a short period of time; this is not painful.
- A research team member will periodically ask your child a set of questions, either in person or by video. A parent or guardian will be asked questions in a similar way.
* Additional in-person visit(s) may occur, only if necessary, based on clinical judgment.
Who can I contact for more information?
Call (206) 987-6338 or email Maria Benitez-Cortez.