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NCT07222371 · ClinicalTrials.gov registry record · Phase 1

An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

A Phase 1 study of Genetic Disease and TUBB4A-Related Leukodystrophy, sponsored by University of California, San Diego.

Active
Registry status
Phase 1
Development phase
1
Enrollment target
1
Study location

NCT07222371: Active Phase 1 study of Genetic Disease and TUBB4A-Related Leukodystrophy, sponsored by University of California, San Diego.

NCT07222371 is a Phase 1 study of Genetic Disease and TUBB4A-Related Leukodystrophy that is active but no longer recruiting, run by University of California, San Diego. The registered enrollment target is 1 participants, below the 2,561-participant average among 30 other Genetic Disease trials with a reported enrollment target (100% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT07222371, a Phase 1 study of Genetic Disease and TUBB4A-Related Leukodystrophy, is active but no longer recruiting, sponsored by University of California, San Diego.

ACTIVE NOT RECRUITING
Registry status
Phase 1
Development phase
1 participants
Enrollment target
1
Study location

Study Summary

This current study is aimed for the treatment of an individual participant with a form of TUBB4A-related leukodystrophy with hypomyelination.

Primary Outcome

The Barry Albright Dystonia Scale (BAD) is a clinical tool designed to evaluate the severity of dystonia in children. This scale assesses dystonia in eight different body regions including the eyes, mouth, neck, trunk, and each of the four limbs. Each region is scored on a severity scale from 0 (no dystonia) to 4 (severe dystonia), allow for quantification of the overall dystonia as well as distribution of dystonic movements across the body. The BAD will be performed at each study timepoint as s

Conditions Studied

Genetic Disease TUBB4A-Related Leukodystrophy

Interventions

  • DRUG nL-TUBB4-001

Study Locations (1)

California

  • Rady Children's Hospital San Diego - San Diego

Trial Details

FieldValue
Enrollment Target 1 participants
Start Date 2025-09-19
Est. Completion 2027-09
Phase Phase 1
University of California, San Diego

775 total trials

What the registry record for NCT07222371 still lists

NCT07222371 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 1 participants, a relatively small participant target, below the 2,561-participant average among 30 other Genetic Disease trials with a reported enrollment target (100% lower).

The record links to 2 conditions, with Genetic Disease appearing as the primary indexed condition, and to 1 intervention - of which nL-TUBB4-001 is the first listed.

NCT07222371 reports a single indexed study location in California.

Frequently Asked Questions

What is clinical trial NCT07222371 about?

NCT07222371 is a clinical study titled "An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy". This current study is aimed for the treatment of an individual participant with a form of TUBB4A-related leukodystrophy with hypomyelination.

What is the current status of trial NCT07222371?

This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2025-09-19. Estimated completion is 2027-09.

What conditions does trial NCT07222371 study?

This clinical trial studies the following conditions: Genetic Disease, TUBB4A-Related Leukodystrophy.

What interventions are being tested in trial NCT07222371?

The interventions under investigation include: nL-TUBB4-001 (DRUG).

Who is sponsoring clinical trial NCT07222371?

This trial is sponsored by University of California, San Diego, which has 775 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT07222371 being conducted?

This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Learn More About Clinical Trials

Similar trials for Genetic Disease

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT07222371's enrollment target sits among peer trials

1 30th of 30 the lowest of 30 other Genetic Disease trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Genetic Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Related registry views

Source: ClinicalTrials.gov NCT07222371, the US trial registry maintained by the National Library of Medicine. NCT07222371 (small enrollment · single site footprint · active not recruiting) retrieved and formatted by PlainTrial, see methodology.