Ignite Creation Date:
2024-05-05 @ 12:03 PM
Last Modification Date:
2024-10-26 @ 9:19 AM
Study NCT ID:
NCT00227266
Status:
COMPLETED
Last Update Posted:
2011-09-26
First Post:
2005-09-23
Brief Title:
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
Sponsor:
University of Utah
Organization:
University of Utah
Study Overview
Official Title:
Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy SMA CARNI-VAL Trial
Status:
COMPLETED
Status Verified Date:
2011-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid VPA and carnitine in patients with Spinal Muscular Atrophy SMA 2 to 17 years of age Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA sitters 2 - 8 years of age Cohort 2 is an open label protocol for SMA standers and walkers 3 - 17 years of age to explore responsiveness of efficacy outcomes Outcome measures will include blood chemistries functional testing pulmonary function testing electrophysiological evaluations PedsQL quality of life assessment quantitative assessments of survival motor neuron SMN mRNA from blood samples growth and vital sign parameters Six centers will enroll a total of 90 patients
Detailed Description:
This is a multi-center phase II trial of a combined regimen of oral valproic acid VPA and carnitine in patients with Spinal Muscular Atrophy SMA 2 to 17 years of age Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA sitters 2 - 8 years of age Subjects will undergo two baseline assessments over 4 to 6 week period then will be randomized to treatment or placebo for the next six months All subjects will then be placed on active treatment for the subsequent six month period Cohort 2 is an open label protocol for SMA standers and walkers 3 - 17 years of age to explore responsiveness of efficacy outcomes Subjects will undergo two baseline assessments over a four to six week period followed by one year active treatment with VPA and carnitine Outcome measures are performed every 3 to 6 months and include blood chemistries functional testing pulmonary function testing electrophysiological evaluations PedsQL quality of life assessment quantitative assessments of survival motor neuron SMN mRNA from blood samples growth and vital sign parameters Six centers will enroll a total of 90 patients
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None