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NCT03307304 · ClinicalTrials.gov registry record
Investigations of Juvenile Neuronal Ceroid Lipofuscinosis
A clinical trial of Batten Disease and Juvenile Neuronal Ceroid Lipofuscinosis (CLN3), sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- Recruiting
- Registry status
- 300
- Enrollment target
- 1
- Study location
NCT03307304 is a study of Batten Disease and Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) that is actively recruiting participants, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). The registered enrollment target is 300 participants, below the 2,802-participant average among 11 other Batten Disease trials with a reported enrollment target (89% lower). The trial reports 1 study location across 1 state.
The verdict
NCT03307304, a study of Batten Disease and Juvenile Neuronal Ceroid Lipofuscinosis (CLN3), is actively recruiting participants, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- RECRUITING
- Registry status
- 300 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: CLN3, or Batten disease, is a genetic disorder. This deadly disease leads to decline of brain and nervous system functions. Symptoms of CLN3 typically occur between 4 and 7 years of age. They include changes in how a person sees, thinks, and moves. CLN3 can also cause seizures. No effective treatments for the disease are yet known. There is limited testing of potential therapies. Researchers want to study CLN3 more so they can improve future therapies. Objective: To identify clinical or biochemical markers that can be used as therapeutic outcome measures for CLN3. Eligibility: People with CLN3. It must be based on Two CLN3 mutations OR One CLN3 mutation AND findings seen with a powerful microscope Family members of a person with CLN3. Design: Participants will have already been referred to NIH for CLN3 evaluation. If participants agree to do the study, they will: 1. give spinal fluid, blood, urine, and skin samples. They may provide other samples if they were already collected. These may include cells, surgical specimens, and DNA. 2. will be seen by multiple healthcare specialists. Participants may provide medical records or photos. Participants will sign a release of medical records form.P Researchers may send samples or clinical data to other investigators. For research testing, the samples will not include the participant s name. For a test in a clinical lab, researchers will include the participant s name. These results will become part of the clinical record at NIH.
Conditions Studied
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 300 participants |
| Start Date | 2017-11-27 |
| Est. Completion | 2050-12-31 |
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)305 total trials
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03307304
The ClinicalTrials.gov registry entry for NCT03307304 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 300 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 2,802-participant average among 11 other Batten Disease trials with a reported enrollment target (89% lower). The listed sponsor is Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which has 305 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Batten Disease appearing as the primary indexed condition, and to 0 interventions.
NCT03307304 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT03307304 about?
NCT03307304 is a clinical study titled "Investigations of Juvenile Neuronal Ceroid Lipofuscinosis". Background: CLN3, or Batten disease, is a genetic disorder. This deadly disease leads to decline of brain and nervous system functions. Symptoms of CLN3 typically occur between 4 and 7 years of age. They include changes in how a person sees, thinks, and moves. CLN3 can also cause seizures. No effec...
What is the current status of trial NCT03307304?
This trial is currently recruiting. The enrollment target is 300 participants. The study started on 2017-11-27. Estimated completion is 2050-12-31.
What conditions does trial NCT03307304 study?
This clinical trial studies the following conditions: Batten Disease, Juvenile Neuronal Ceroid Lipofuscinosis (CLN3).
Who is sponsoring clinical trial NCT03307304?
This trial is sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which has 305 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03307304 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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