# Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation

> **NCT07197268** · PHASE1,PHASE2 · ACTIVE_NOT_RECRUITING · sponsor: **n-Lorem Foundation** · enrollment: 1 (actual)

## Conditions studied

- Bainbridge-Ropers Syndrome

## Interventions

- **DRUG:** nL-ASXL3-001

## Key facts

- **NCT ID:** NCT07197268
- **Lead sponsor:** n-Lorem Foundation
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2025-05-19
- **Primary completion:** 2027-05
- **Final completion:** 2027-05
- **Target enrollment:** 1 (ACTUAL)
- **Last updated:** 2025-09-29

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07197268

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07197268, "Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT07197268. Licensed CC0.

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