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Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

NCT06369974 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2026-03-02

No results posted yet for this study

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single pediatric participant with TUBB4A associated leukodystrophy.

Conditions

Interventions

DRUG

Antisense oligonucleotide treatment (ASO)

Drug: nL-TUBB4-001; Personalized antisense oligonucleotide

Sponsors & Collaborators

  • n-Lorem Foundation

    collaborator OTHER

  • Massachusetts General Hospital

    lead OTHER

Principal Investigators

  • Florian Eichler, MD · Massachusetts General Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-09-18
Primary Completion
2026-06-30
Completion
2026-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

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 NCT06369974 on ClinicalTrials.gov