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

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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