NCT07197294

Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)

An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA) Due to MAPK8IP3 Mutation

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: n-Lorem Foundation · Academic / Other
• Sex: Male
• Age: 5 Years – 5 Years
• Healthy volunteers: Not accepted

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Neurodevelopmental Disorder with or without Brain Abnormalities (NEDBA) due to a heterozygous pathogenic missense mutation in MAPK8IP3

Detailed description

This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with NEDBA due to a heterozygous pathogenic missense mutation in MAPK8IP3

Conditions

• Neurodevelopmental Disorder With or Without Variable Brain Abnormalities

Interventions

Type	Name	Description
DRUG	nL-MAPK8-001	Personalized antisense oligonucleotide

Timeline

• Start date: 2025-02-24
• Primary completion: 2027-02-01
• Completion: 2027-02-01
• First posted: 2025-09-29
• Last updated: 2025-09-29

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study