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NCT06014541 · ClinicalTrials.gov registry record
Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
A clinical trial of Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, sponsored by Ionis Pharmaceuticals.
- Active
- Registry status
- 29
- Enrollment target
- 5
- Study locations
NCT06014541: Active study of Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, sponsored by Ionis Pharmaceuticals.
NCT06014541 is a study of Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome that is active but no longer recruiting, run by Ionis Pharmaceuticals. The registered enrollment target is 29 participants. The trial reports 5 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06014541, a study of Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, is active but no longer recruiting, sponsored by Ionis Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- 29 participants
- Enrollment target
- 5
- Study locations
Study Summary
The purpose of the study is to prospectively assess longitudinal changes in biomarkers (MECP2, potential biomarkers of target engagement and disease activity) in cerebrospinal fluid (CSF) and blood; characterize longitudinal changes in performance on clinical scales (clinician-reported measures of neurodevelopment and functioning) and caregiver-reported outcome assessments (communication, gastrointestinal, social-emotional-adaptive behavioral measures); evaluate longitudinal changes in caregiver-reported health-related quality-of-life measures; and assess the frequency, type, and severity of seizures over time.
Conditions Studied
Study Locations (5)
California
- UCSD - Rady Children's Hospital - San Diego
Minnesota
- Gillette Children's Specialty Healthcare - Saint Paul
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Tennessee
- Vanderbilt University Medical Center - Nashville
Texas
- Baylor College of Medicine - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 29 participants |
| Start Date | 2023-10-03 |
| Est. Completion | 2026-06 |
What the registry record for NCT06014541 still lists
NCT06014541 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 29 participants, a relatively small participant target.
The record links to 1 condition, with Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome appearing as the primary indexed condition, and to 0 interventions.
NCT06014541 lists 5 locations in 5 states (California, Minnesota, Pennsylvania).
Frequently Asked Questions
What is clinical trial NCT06014541 about?
NCT06014541 is a clinical study titled "Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome". The purpose of the study is to prospectively assess longitudinal changes in biomarkers (MECP2, potential biomarkers of target engagement and disease activity) in cerebrospinal fluid (CSF) and blood; characterize longitudinal changes in performance on clinical scales (clinician-reported measures of n...
What is the current status of trial NCT06014541?
This trial is currently active not recruiting. The enrollment target is 29 participants. The study started on 2023-10-03. Estimated completion is 2026-06.
What conditions does trial NCT06014541 study?
This clinical trial studies the following conditions: Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome.
Who is sponsoring clinical trial NCT06014541?
This trial is sponsored by Ionis Pharmaceuticals, which has 76 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06014541 being conducted?
This trial has 5 study locations across California, Minnesota, Pennsylvania, Tennessee, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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