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NCT03951298 · ClinicalTrials.gov registry record
I-Tracking Neurodegeneration in Early Wolfram Syndrome
A clinical trial, sponsored by Washington University School of Medicine.
- Completed
- Registry status
- 127
- Enrollment target
NCT03951298: Completed study, sponsored by Washington University School of Medicine.
NCT03951298 is a clinical trial that has completed, run by Washington University School of Medicine. The registered enrollment target is 127 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03951298 has completed, sponsored by Washington University School of Medicine.
- COMPLETED
- Registry status
- 127 participants
- Enrollment target
Study Summary
Wolfram syndrome (WFS; OMIM #222300) is a rare autosomal recessive disease clinically defined in 1938 as the combination of childhood-onset insulin dependent diabetes, optic nerve atrophy, diabetes insipidus and deafness. Based on early descriptions, neurological features were thought to appear later in the disease with death occurring in middle adulthood. Importantly, the major causative gene (WFS1) was identified in 1998. This discovery allowed researchers to determine that the WFS1 gene encodes the protein wolframin, which helps protect cells from endoplasmic reticulum (ER) stress-mediated apoptosis, potentially via intracellular calcium homeostasis. Pathogenic mutations in WFS1 can result in death or dysfunction of cells that are under high ER stress, such as insulin-producing pancreatic β cells, causing insulin dependent diabetes. In addition, knowing the causative gene has allowed researchers to identify patients by their WFS1 mutation rather than the classic set of symptoms, leading to the increasing realization that the WFS1-related phenotype (including neurologic symptoms) is much more variable than previously understood. The first iteration of this grant (HD070855 "Tracking Neurodegeneration in Early Wolfram Syndrome") contributed to this shift in understanding. In this time, the research team has built a successful annual research clinic for WFS, that has met or exceeded recruitment goals for patients and controls, validated a clinical severity rating scale for WFS, described an unexpectedly early neurophenotype of reduced balance, smell identification and ventral pons volume, identified alterations in traditional diffusion tensor imaging (DTI) metrics that suggest hypomyelination as a pervasive neuropathological feature of WFS and provided justification for the selection of two primary outcomes (visual acuity and ventral pons volume) in a newly funded clinical efficacy study in WFS (Barrett, PI).
Primary Outcome
MRI measures of regional brain volumes over time
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 127 participants |
| Start Date | 2018-08-10 |
| Est. Completion | 2023-12-31 |
What the finished NCT03951298 record still lists
NCT03951298 is an observational study that tracks outcomes without assigning an intervention. The registered 127 participants enrollment target is mid-sized for trials with a published cap.
The record links to 0 conditions, and to 0 interventions.
NCT03951298 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03951298 about?
NCT03951298 is a clinical study titled "I-Tracking Neurodegeneration in Early Wolfram Syndrome". Wolfram syndrome (WFS; OMIM #222300) is a rare autosomal recessive disease clinically defined in 1938 as the combination of childhood-onset insulin dependent diabetes, optic nerve atrophy, diabetes insipidus and deafness. Based on early descriptions, neurological features were thought to appear late...
What is the current status of trial NCT03951298?
This trial is currently completed. The enrollment target is 127 participants. The study started on 2018-08-10. Estimated completion is 2023-12-31.
Who is sponsoring clinical trial NCT03951298?
This trial is sponsored by Washington University School of Medicine, which has 1,502 total clinical trials registered on ClinicalTrials.gov.
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