Research
At Stanford, the battle against SMA is in the Clinic and in the Lab.
Clinical Trials
NPTUNE Project
Pilot Therapeutic Trials of Hydroxyurea on Spinal Muscular Atrophy
Additional Information and Resources
Back to SMA at Stanford
NPTUNE Project
Type I Trial (ClinicalTrials.gov Listing)
Types II and III Trial (ClinicalTrials.gov Listing)
Note: These trials are recruiting again. Please contact us for more information about participation.
Blood levels of SMN mRNA and protein will also be measured. [ Time Frame: 14 weeks ]
Pilot Therapeutic Trials of Hydroxyurea on Spinal Muscular Atrophy
Type I Trial (ClinicalTrials.gov Listing)
Types II and III Trial (ClinicalTrials.gov Listing)
Note: These studies have finished enrollment and are no longer recruiting.
Hydroxyurea is a drug that has been shown to increase the amount of working SMN protein produced by certain cells in culture. The purpose of these trials is to test the safety and efficacy of hydroxyurea in treating Type I SMA patients and Types II and III SMA patients.
The Types II and III trial is nearing completion. The Type I trial is estimated to finish around April 2009. Check back here at Stanford's SMA Site for any changes in this projected date or updates on publication information.
Additional Information and Resources
A Complete List of US Spinal Muscular Atrophy Trials at ClinicalTrials.gov
More information about the Stanford SMA Trials can also be found at this site.
Stanford's Clinical Trials Directory
This is a new site modeled after ClinicalTrials.gov. Here, all Clinical Trials currently underway at Stanford are presented.
Stanford/Packard Center for Translational Research in Medicine (SPCTRM)
On the SPCTRM site, there are also links to a Patients Page and a FAQ Page.
Stanford University Research Compliance Office: Participants Corner
This page contains general information about the research process and the rights of its participants.
The Food and Drug Administration
The FDA, part of the US Department of Health and Human Services monitors all clinical trials, ensures that Good Clinical Practice and Code of Federal Regulations are followed. They also have a Page for Patients and a Glossary on their site.
