Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome : A Prospective and Retrospective Observational/Non-interventional Study to Characterize Biomarkers and Disease Progression in Patients With MECP2 Duplication Syndrome

This is a multi-center, non-randomized, non-interventional prospective and retrospective study in up to 40 participants with MECP2 duplication syndrome (MDS) who can undergo general anesthesia or conscious sedation to collect fluid biomarkers (CSF and blood), undergo electrophysiological assessments (electroencephalogram [EEG], evoked potentials [EP], pupillometry), clinical assessments and caregiver reported outcomes measures, to be used in support of the development of therapies for MDS. The study duration for each participant will be approximately 110 weeks..

Medienart:

Klinische Studie

Erscheinungsjahr:

2024

Erschienen:

2024

Enthalten in:

ClinicalTrials.gov - (2024) vom: 22. März Zur Gesamtaufnahme - year:2024

Sprache:

Englisch

Links:

Volltext [kostenfrei]

Themen:

610
Disease Progression
Recruitment Status: Recruiting
Rett Syndrome
Study Type: Observational
Syndrome

Anmerkungen:

Source: Link to the current ClinicalTrials.gov record., First posted: August 28, 2023, Last downloaded: ClinicalTrials.gov processed this data on March 27, 2024, Last updated: March 27, 2024

Study ID:

NCT06014541
NH00006

Veröffentlichungen zur Studie:

fisyears:

Förderinstitution / Projekttitel:

PPN (Katalog-ID):

CTG000121150