Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Recruiting study of Batten Disease and Juvenile Neuronal Ceroid Lipofuscinosis (CLN3), sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
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. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Identification of biomarkers
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
What NCT03307304 shows while recruiting
NCT03307304 is an observational study that tracks outcomes without assigning an intervention. The registered 300 participants enrollment target is mid-sized for trials with a published cap, below the 2,802-participant average among 11 other Batten Disease trials with a reported enrollment target (89% lower).
The record links to 2 conditions, with Batten Disease appearing as the primary indexed condition, and to 0 interventions.
NCT03307304 reports a single indexed study location in 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.
Learn More About Clinical Trials
Similar trials for Batten Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03307304's enrollment target sits among peer trials
300 4th of 11 higher than 8 of 11 other Batten Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Batten Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
Inherited Retinal Degenerative Disease Registry
RECRUITING
-
Clinical and Neuropsychological Investigations in Batten Disease
RECRUITING
-
NYSCF Scientific Discovery Biobank
RECRUITING
-
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
RECRUITING · Phase 1
-
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
ACTIVE NOT RECRUITING
-
Gene Therapy for Children With CLN3 Batten Disease
ACTIVE NOT RECRUITING · Phase 1
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00353483 · 300 participants
Effect of Neoadjuvant or Adjuvant Systemic Therapy on Breast Cancers, Bone Marrow Cancer Cells, and Circulating Cancer Cells
- NCT00588185 · 300 participants · NA
[18F]-Fluoro-2-Deoxy-D-Glucose and -[18F] Dihydro-Testosterone Pet Imaging in Patients With Progressive Prostate Cancer
- NCT00682032 · 300 participants · NA
The Effect of Beta-glucan in Non-Small Cell Lung Cancer
- NCT00870064 · 300 participants · NA
The Treatment of Type I Open Fractures in Pediatrics
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI