Status:
COMPLETED
Characterization of the Patient Population With Galactosialidosis
Lead Sponsor:
St. Jude Children's Research Hospital
Collaborating Sponsors:
Assisi Foundation
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Conditions:
Galactosialidosis
Eligibility:
All Genders
6+ years
Brief 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 o...
Detailed Description
Individual patient/families will be interviewed by telephone to learn basic demographic information and disease status. Medical records will be requested from primary care providers to provide further...
Eligibility Criteria
Inclusion
- Individuals with suspected or confirmed molecular diagnosis of galactosialidosis who are ≥ 6 months of age.
Exclusion
- Individuals with a lysosomal storage disorder who have been shown to have a mutation in a gene other than that encoding PPCA.
Key Trial Info
Start Date :
February 8 2012
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
April 12 2012
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT01416467
Start Date
February 8 2012
End Date
April 12 2012
Last Update
October 11 2018
Active Locations (1)
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1
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105