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NCT01035424 · ClinicalTrials.gov registry record
Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis
A clinical trial of Batten Disease and Late Infantile Neuronal Ceroid Lipofuscinosis, sponsored by Weill Medical College of Cornell University.
- Completed
- Registry status
- 48
- Enrollment target
- 1
- Study location
NCT01035424: Completed study of Batten Disease and Late Infantile Neuronal Ceroid Lipofuscinosis, sponsored by Weill Medical College of Cornell University.
NCT01035424 is a study of Batten Disease and Late Infantile Neuronal Ceroid Lipofuscinosis that has completed, run by Weill Medical College of Cornell University. The registered enrollment target is 48 participants, below the 2,825-participant average among 11 other Batten Disease trials with a reported enrollment target (98% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01035424, a study of Batten Disease and Late Infantile Neuronal Ceroid Lipofuscinosis, has completed, sponsored by Weill Medical College of Cornell University.
- COMPLETED
- Registry status
- 48 participants
- Enrollment target
- 1
- Study location
Study Summary
The primary aim of the study is to assess the genotype - phenotype correlations of the CNS manifestations of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal, rare, recessive disorder of the CNS in children. This study will be accomplished by comparing the genotype to a neurologic assessment and Weill Cornell LINCL scale, the UBDRS scale, the standardized CHQ quality of life scale, and the Mullen scale; magnetic resonance imaging (MRI); and routine clinical evaluations. This study is designed to run parallel to a separate study which is being done by the Department of Genetic Medicine, which will use gene transfer to treat the central nervous system (CNS) manifestations of late infantile neuronal ceroid lipofuscinosis.
Primary Outcome
The Weill Cornell LINCL scale, a 12 point scale which combines assessment of feeding, gait, motor and language to give an overall assessment of various CNS functions
Conditions Studied
Study Locations (1)
New York
- Weill Cornell Medicine - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 48 participants |
| Start Date | 2009-06 |
| Est. Completion | 2016-01 |
What the finished NCT01035424 record still lists
NCT01035424 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 48 participants, a relatively small participant target, below the 2,825-participant average among 11 other Batten Disease trials with a reported enrollment target (98% lower).
The record links to 2 conditions, with Batten Disease appearing as the primary indexed condition, and to 0 interventions.
NCT01035424 reports a single indexed study location in New York.
Frequently Asked Questions
What is clinical trial NCT01035424 about?
NCT01035424 is a clinical study titled "Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis". The primary aim of the study is to assess the genotype - phenotype correlations of the CNS manifestations of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal, rare, recessive disorder of the CNS in children. This study will be accomplished by comparing the genotype to a neurologic asse...
What is the current status of trial NCT01035424?
This trial is currently completed. The enrollment target is 48 participants. The study started on 2009-06. Estimated completion is 2016-01.
What conditions does trial NCT01035424 study?
This clinical trial studies the following conditions: Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis.
Who is sponsoring clinical trial NCT01035424?
This trial is sponsored by Weill Medical College of Cornell University, which has 701 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01035424 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT01035424's enrollment target sits among peer trials
48 6th of 11 higher than 6 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.
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