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ACTIVE NOT RECRUITING

Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

NCT06014541 · View on ClinicalTrials.gov ↗

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.

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

FieldValue
Enrollment Target 29 participants
Start Date 2023-10-03
Est. Completion 2026-06

Sponsor

Ionis Pharmaceuticals

55 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT06014541

The ClinicalTrials.gov registry entry for NCT06014541 describes a study currently listed as active not recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 29 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ionis Pharmaceuticals, which has 55 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT06014541 reports 5 study locations spanning 5 distinct geographic areas — top geographies include California, Minnesota, Pennsylvania. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

Who is sponsoring clinical trial NCT06014541?

This trial is sponsored by Ionis Pharmaceuticals, which has 55 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial