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

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Massachusetts General Hospital
Study ID
NCT06369974
Phase
PHASE1/PHASE2
Status
Enrolling By Invitation

Conditions

  • Genetic Disease

Eligibility Criteria

Sex
ALL
Age
4 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Antisense oligonucleotide treatment (ASO) — DRUG
    Drug: nL-TUBB4-001; Personalized antisense oligonucleotide

Study Details

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

Key Dates

First listed
Apr 17, 2024
Start date
Sep 18, 2024
Status verified
Feb 2026
Primary completion
Jun 30, 2026
Completion
Jun 30, 2026

Study Design

Enrollment
1 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Intervention
    Open Label

Primary Outcome Measure

Neurological assessments [ Time Frame: Baseline to 24 months ]

Locations (1)

FacilityCityStateZIPSite coordinators
Massachusetts General HospitalBostonMassachusetts02114-

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By research site
Massachusetts General Hospital· Boston, MA

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