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NCT06392126Recruitment Completed

Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for a Single Participant With Amyotrophic Lateral Sclerosis (ALS) Due to a Pathogenic Variant in CHCHD10
n-Lorem FoundationPhase 1/2ClinicalTrials.govdrug
Verified
Study focus
Disease/Condition
Amyotrophic Lateral Sclerosis
Drug/Device/Intervention
nL-CHCHD-001
Study type
Interventional
Intervention type
drug
Sponsor & location
Primary sponsor
n-Lorem Foundation
Location
Jacksonville, United States of America
Timeline & enrollment
Phase
Phase 1/2
Start date
Apr 16, 2024
End date
Apr 01, 2025
Enrollment
1 participants
Primary outcome

Clinical Functioning,Clinical Functioning,Clinical Functioning,Clinical Functioning

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug
 designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a
 pathogenic variant in CHCHD10

ICD-10 classifications
Neuralgic amyotrophyMotor neuron diseaseOther degenerative diseases of the nervous systemOther degenerative disorders of nervous system in diseases classified elsewhereMultiple sclerosis
Data source
Registry
ClinicalTrials.gov
Trial ID
NCT06392126
Type
Non-Device Trial
About this record

Access comprehensive clinical trial information for NCT06392126 through Pure Global AI's free database. This Phase 1/2 trial is sponsored by n-Lorem Foundation and is currently Recruitment Completed. The study focuses on Amyotrophic Lateral Sclerosis. Target enrollment is 1 participants.

This page provides complete trial specifications, intervention details, outcomes, and location information. Pure Global AI offers free access to ClinicalTrials.gov data, helping medical device and pharmaceutical companies navigate clinical research efficiently.