NCT04388774

Low-Dose Ketamine in Children With ADNP Syndrome

A Phase 2A Open-Label Study Evaluating the Safety and Efficacy of Low-Dose Ketamine in Children With ADNP Syndrome

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Alexander Kolevzon · Academic / Other
• Sex: All
• Age: 5 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• ADNP Syndrome

Interventions

Type	Name	Description
DRUG	Ketamine	A single 40-minute intravenous infusion

Timeline

• Start date: 2020-08-19
• Primary completion: 2021-06-08
• Completion: 2021-06-08
• First posted: 2020-05-14
• Last updated: 2023-07-07
• Results posted: 2023-07-07

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study