|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
22 May 2023 |
|
Main ID: |
NCT00374075 |
|
Date of registration:
|
06/09/2006 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
|
|
Scientific title:
|
In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy |
|
Date of first enrolment:
|
September 2003 |
|
Target sample size:
|
42 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT00374075 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
|
|
Phase:
|
Phase 1
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Kathryn J Swoboda, M.D |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
University of Utah/Primary Children's Medical Center |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Patients must have a diagnosis of SMA, confirmed by genetic testing
- Only patients 2 years of age and older at enrollment will be eligible
Exclusion Criteria:
- Patients taking any medications with known hepatotoxicity, congenital metabolic
disorders or on multiple anticonvulsant medications
- Patients taking medications which may interact with VPA
- Patients on ventilatory support for more than 16 hours per day
- Patients currently enrolled in other treatment trials
Age minimum:
2 Years
Age maximum:
N/A
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Spinal Muscular Atrophy
|
|
Intervention(s)
|
|
Drug: Valproic Acid
|
|
Primary Outcome(s)
|
|
To assess safety and tolerability of VPA in SMA patients greater than 2 years of age
|
|
Secondary Outcome(s)
|
|
DEXA estimates of body composition, bone mineral density and content
|
|
Electrophysiologic measures of denervation
|
|
To look for a potential in vivo effect of VPA on SMN mRNA in patient blood cells at routinely used clinical doses
|
|
Measures of gross motor function
|
|
Measures of pulmonary function
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|