Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation
Part of paid clinical trials in Boston, Massachusetts.
- Sponsor
- n-Lorem Foundation
- Study ID
- NCT07588581
- Phase
- PHASE1/PHASE2
- Status
- Active Not Recruiting
Conditions
- Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA)
Eligibility Criteria
- Sex
- ALL
- Age
- N/A - N/A
- Healthy Volunteers
- Not accepted
Interventions
- nL-UBTF-001 — DRUGPersonalized antisense oligonucleotide
Study Details
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Childhood-Onset Neurodegeneration with Brain Atrophy (CONDBA) due to a heterozygous missense gain-of-function mutation in UBTF
Key Dates
- First listed
- May 15, 2026
- Start date
- Feb 5, 2025
- Status verified
- May 2026
- Primary completion
- Feb 28, 2027
- Completion
- Feb 28, 2027
Study Design
- Enrollment
- 1 participants (actual)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: Open Label
Primary Outcome Measure
Gross Motor Function [ Time Frame: Baseline to 24-months ]
Locations (1)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | - |
Find similar trials in Boston, MA
By research site