Track this trial free. DataLookout checks ClinicalTrials.gov daily and emails you which fields changed on NCT07474298 (status, enrollment, completion dates, endpoints, sites) when this record is updated. Free accounts track up to 5 trials.

Track this trial

Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation Associated With Schuurs-Hoeijmakers Syndrome (SHMS)

StatusNot Yet Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Study First Submitted
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug intended for a single participant with Schuurs-Hoeijmakers syndrome (SHMS) due to a pathogenic, de novo, heterozygous missense gain-of-function mutation in PACS1
Trial Details
NCT Number NCT07474298
Lead Sponsor n-Lorem Foundation
Collaborators: The Hospital for Sick Children
Conditions
(from sponsor)		 Schuurs-Hoeijmakers Syndrome
Enrollment 1 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov