Neuren Study for Children with Phelan McDermid Syndrome 

Neuren Study for Children with Phelan McDermid Syndrome 

Study directors: Elysa Marco, MD

Recruiting: Yes

Study Title: A Phase 3 study of NNZ-2591 in Pediatric Participants with Phelan McDermid Syndrome

Condition: Phelan McDermid Syndrome

Purpose: The purpose of this study is to find out whether NNZ-2591, an investigational medication, can improve communication and life skills in children with SHANK3 mutations.

Eligibility

Inclusion criteria:

• 3-12 years old

• Clinical diagnosis of Phelan McDermid Syndrome with documented SHANK3 genetic abnormality.

• Body weight > 10kg (22 lbs) at initial visit

Exclusion criteria:

• Uncontrolled seizure disorders

• Unstable medical or psychiatric illness

• Current treatment with more than 3 psychotropic medications

• Current use of certain medications – contact our study team to discuss

What is Involved?

• Testing: Comprehensive cognitive assessments, psychiatric questionnaires, and medical evaluations

• Frequency of visits: 11 visits over 5 months, 5 in-clinic and 6 telehealth visits

• Costs: No costs will be charged for any of the study procedures. We are able to provide comprehensive travel support to enroll subjects from outside California

• Payment: In return for the time, effort, and travel expenses, participants will be compensated $100 per on-site visit

Location and Contact Information

• Location: Cortica Marin

• Coordinator: Kelly Olvany – kolvany@corticacare.com – (331) 272-1322

• Fill out our Interest Form and we will contact you!