CLINICAL TRIALS PROFILE FOR CARNITOR SF

All Clinical Trials for CARNITOR SF

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00227266 ↗	Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy	Completed	Abbott	Phase 2	2005-09-01	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.
NCT00227266 ↗	Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy	Completed	Families of Spinal Muscular Atrophy	Phase 2	2005-09-01	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.
NCT00227266 ↗	Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy	Completed	Leadiant Biosciences, Inc.	Phase 2	2005-09-01	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.

Clinical Trial Conditions for CARNITOR SF

Condition Name

Condition Name for CARNITOR SF
Intervention	Trials
Spinal Muscular Atrophy	2
Carnitine Deficiency	2
Mixed Phenotype Acute Leukemia	1

Condition MeSH

Condition MeSH for CARNITOR SF
Intervention	Trials
Muscular Atrophy	2
Atrophy	2
Precursor Cell Lymphoblastic Leukemia-Lymphoma	2

Clinical Trial Locations for CARNITOR SF

Trials by Country

Trials by Country for CARNITOR SF
Location	Trials
United States	8
Canada	2
Netherlands	1

Trials by US State

Trials by US State for CARNITOR SF
Location	Trials
Ohio	2
New York	1
Wisconsin	1
Utah	1
Michigan	1

Clinical Trial Progress for CARNITOR SF

Clinical Trial Phase

Clinical Trial Phase for CARNITOR SF
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	1
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for CARNITOR SF
Clinical Trial Phase	Trials
Completed	2
Recruiting	1
Active, not recruiting	1

Clinical Trial Sponsors for CARNITOR SF

Sponsor Name

Sponsor Name for CARNITOR SF
Sponsor	Trials
Abbott	2
Families of Spinal Muscular Atrophy	2
University of Utah	2

Sponsor Type

Sponsor Type for CARNITOR SF
Sponsor	Trials
Other	11
Industry	4
NIH	1