Status:
ACTIVE_NOT_RECRUITING
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation
Lead Sponsor:
n-Lorem Foundation
Collaborating Sponsors:
University of North Carolina, Chapel Hill
Conditions:
Bainbridge-Ropers Syndrome
Eligibility:
MALE
4-4 years
Phase:
PHASE1
PHASE2
Brief 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 nons...
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 BRPS due to a pathogenic,...
Eligibility Criteria
Inclusion
- Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s)
- Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records
- Genetically confirmed ASXL3 genetic variant
Exclusion
- Participant has any condition that, in the opinion of the Site Investigator, would ultimately prevent the completion of stud procedures
Key Trial Info
Start Date :
May 19 2025
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2027
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT07197268
Start Date
May 19 2025
End Date
May 1 2027
Last Update
September 29 2025
Active Locations (1)
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1
University of North Carolina Chapel Hill
Chapel Hill, North Carolina, United States, 27599