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Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

StatusActive, Not Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Re-verified, no change to tracked fieldsv3
  2. Trial status: Enrolling by Invitation to Active, Not Recruiting
    • Primary and study completion dates: Feb 2027 to Mar 2027
    • Start date changed from estimate of Feb 2025 to Mar 17, 2025
    v2
  3. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Autosomal Dominant Leukodystrophy (ADLD) due to LMNB1 mutation
Trial Details
NCT Number NCT06816498
Lead Sponsor n-Lorem Foundation
Collaborators: Mayo Clinic
Conditions
(from sponsor)		 Autosomal Dominant Leukodystrophy
Enrollment 1 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov