Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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.
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.
Conditions Studied
Interventions
- DRUG nL-TUBB4-001
Study Locations (1)
California
- Rady Children's Hospital San Diego - San Diego
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2025-09-19 |
| Est. Completion | 2027-09 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07222371
The ClinicalTrials.gov registry entry for NCT07222371 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 1 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 2,561-participant average among 30 other Genetic Disease trials with a reported enrollment target (100% lower). The listed sponsor is University of California, San Diego, which has 775 total studies on file at ClinicalTrials.gov.
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 1 study location spanning 1 distinct geographic area - top geographies include 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.
-
Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A
ACTIVE NOT RECRUITING · Phase 1
-
An Adaptive Clinical Trial of BeginNGS Newborn Screening for Hundreds of Genetic Diseases by Genome Sequencing
RECRUITING · NA
-
Delineating the Molecular Spectrum and the Clinical, Imaging and Neuronal Phenotype of Chopra-Amiel-Gordon Syndrome
RECRUITING
-
Rett Syndrome Registry
RECRUITING
-
Natural History Study of Patients with HPDL Mutations
RECRUITING
-
Diagnostic Odyssey: Whole Genome Sequencing (WGS)
RECRUITING
Explore more on PlainTrial
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.