This is a Phase 2A, single dose, open-label study to evaluate the safety, tolerability, and
efficacy of a low-dose, 40-minute infusion into the veins (intravenous infusion or "IV") of
ketamine in children with ADNP syndrome (Activity-Dependent Neuroprotective Protein). The
study team will enroll 10 participants, ages 5 to 12, at Mount Sinai. The study participation
is expected to last 4 weeks and will include 5 scheduled clinic visits in order to complete
safety monitoring, clinical assessments, and biomarker collection. At the conclusion of this
study, the study team expects to demonstrate the safety and tolerability of low-dose ketamine
in children with ADNP syndrome. Additionally, the study team anticipates identifying
meaningful signals of efficacy in clinical outcome measures using RNA and DNA sequencing to
analyze ADNP protein expression and DNA methylation profiles, a natural process by which
methyl groups are added to the DNA to change its activity, in order to assess sensitivity to
change with low-dose ketamine treatment and inform future phase 3 studies. Ketamine is not
currently approved by the Food and Drug Administration to treat this syndrome, but it is
approved for use in children in other situations, for example in anesthesia.