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COMPLETED

A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)

NCT03822650 · View on ClinicalTrials.gov ↗

Study Summary

CLN5 is a form of Batten Disease, a neurodegenerative disorder in children causing psychomotor regression, seizures, blindness, loss of ambulation and premature death, and has no available treatments. The purpose of this study is to investigate the clinical characteristics and natural clinical progression of symptoms in individuals with CLN5. This natural history study is important to better understand disease course to be able to determine clinically relevant outcome measures for use in future clinical trials.

Study Locations (1)

New York

  • University of Rochester Medical Center — Rochester

Trial Details

FieldValue
Enrollment Target 37 participants
Start Date 2019-03-13
Est. Completion 2024-05-30

Sponsor

Neurogene

3 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT03822650

The ClinicalTrials.gov registry entry for NCT03822650 describes a study currently listed as completed. 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 37 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Neurogene, which has 3 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 Ceroid Lipofuscinosis, Neuronal 5 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: NCT03822650 reports 1 study location spanning 1 distinct geographic area — top geographies include New York. 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 NCT03822650 about?

NCT03822650 is a clinical study titled "A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)". CLN5 is a form of Batten Disease, a neurodegenerative disorder in children causing psychomotor regression, seizures, blindness, loss of ambulation and premature death, and has no available treatments. The purpose of this study is to investigate the clinical characteristics and natural clinical prog...

What is the current status of trial NCT03822650?

This trial is currently completed. The enrollment target is 37 participants. The study started on 2019-03-13. Estimated completion is 2024-05-30.

What conditions does trial NCT03822650 study?

This clinical trial studies the following conditions: Ceroid Lipofuscinosis, Neuronal 5. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

Who is sponsoring clinical trial NCT03822650?

This trial is sponsored by Neurogene, which has 3 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

Where is trial NCT03822650 being conducted?

This trial has 1 study location across New York. 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