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

NCT07197268 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-09-29

No results posted yet for this study

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Bainbridge-Ropers Syndrome (BRPS) due to a pathogenic, de novo nonsense variant in ASXL3

Conditions

  • Bainbridge-Ropers Syndrome

Interventions

DRUG

nL-ASXL3-001

Personalized antisense oligonucleotide

Sponsors & Collaborators

  • University of North Carolina, Chapel Hill

    collaborator OTHER

  • n-Lorem Foundation

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
4 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-05-19
Primary Completion
2027-05-31
Completion
2027-05-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07197268 on ClinicalTrials.gov