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NCT01416467 · ClinicalTrials.gov registry record
Characterization of the Patient Population With Galactosialidosis
A clinical trial, sponsored by St. Jude Children's Research Hospital.
- Completed
- Registry status
- 3
- Enrollment target
NCT01416467: Completed study, sponsored by St. Jude Children's Research Hospital.
NCT01416467 is a clinical trial that has completed, run by St. Jude Children's Research Hospital. The registered enrollment target is 3 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01416467 has completed, sponsored by St. Jude Children's Research Hospital.
- COMPLETED
- Registry status
- 3 participants
- Enrollment target
Study Summary
The late infantile form of galactosialidosis is potentially amenable to treatment by gene transfer with an adeno-associated viral vector encoding Protective Protein Cathepsin A (PPCA) or by infusion of purified protein. The published literature contains limited descriptions of the disease nor is it known how many patients with the disorder are potentially available for protocol enrollment. This preliminary study is designed to define the demographics and clinical characteristics of the patient population with galactosialidosis. Individuals for whom DNA diagnosis has been performed at St. Jude Children's Research Hospital (SJCRH) will be contacted telephonically to learn their current status. In addition, a letter requesting information regarding patients with galactosialidosis will be sent to all pediatric geneticists throughout the United States. Selected physicians with expertise in lysosomal storage diseases throughout the world will also be contacted. Foundations and Associations for the lysosomal storage disorders will also be contacted in an effort to identify additional potential patients with galactosialidosis. The information to be collected in this preliminary study will facilitate development of specific eligibility criteria for future therapeutic studies.
Primary Outcome
The clinical and demographic data will be tabulated and analyzed for age distribution and disease manifestations with a goal of defining eligibility criteria for future therapeutic protocols.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2012-02-08 |
| Est. Completion | 2012-04-12 |
What the finished NCT01416467 record still lists
NCT01416467 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 3 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT01416467 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01416467 about?
NCT01416467 is a clinical study titled "Characterization of the Patient Population With Galactosialidosis". The late infantile form of galactosialidosis is potentially amenable to treatment by gene transfer with an adeno-associated viral vector encoding Protective Protein Cathepsin A (PPCA) or by infusion of purified protein. The published literature contains limited descriptions of the disease nor is it ...
What is the current status of trial NCT01416467?
This trial is currently completed. The enrollment target is 3 participants. The study started on 2012-02-08. Estimated completion is 2012-04-12.
Who is sponsoring clinical trial NCT01416467?
This trial is sponsored by St. Jude Children's Research Hospital, which has 410 total clinical trials registered on ClinicalTrials.gov.
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