Page last updated: 2024-10-16

carnitine and Adult Spinal Muscular Atrophy

carnitine has been researched along with Adult Spinal Muscular Atrophy in 2 studies

Research Excerpts

Excerpt	Relevance	Reference
"Valproic acid (VPA) has demonstrated potential as a therapeutic candidate for spinal muscular atrophy (SMA) in vitro and in vivo."	2.75	SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy. ( Acsadi, G; Bromberg, MB; Chan, GM; Crawford, TO; D'Anjou, G; Elsheik, B; Kissel, JT; Krosschell, KJ; LaSalle, B; Maczulski, JA; Prior, TW; Reyna, SP; Schroth, MK; Scott, CB; Simard, LR; Sorenson, SL; Swoboda, KJ, 2010)

Research

Studies (2)

Timeframe	Studies, this research(%)	All Research%
pre-1990	0 (0.00)	18.7374
1990's	0 (0.00)	18.2507
2000's	0 (0.00)	29.6817
2010's	2 (100.00)	24.3611
2020's	0 (0.00)	2.80

Authors

Authors	Studies
Swoboda, KJ	2
Scott, CB	2
Crawford, TO	2
Simard, LR	2
Reyna, SP	2
Krosschell, KJ	2
Acsadi, G	2
Elsheik, B	2
Schroth, MK	2
D'Anjou, G	2
LaSalle, B	2
Prior, TW	2
Sorenson, SL	1
Maczulski, JA	2
Bromberg, MB	2
Chan, GM	2
Kissel, JT	2
Sorenson, S	1

Clinical Trials (1)

Trial Overview

Trial			Phase	Enrollment	Study Type	Start Date	Status
Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial)[NCT00227266]			Phase 2	94 participants (Actual)	Interventional	2005-09-30	Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

Max CMAP Amplitude (Mean)

The maximum Compound Motor Action Potential (CMAP) is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This is done multiple times, the outcome used is the highest peak, or response observed. (NCT00227266)
Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)

Intervention	mV (Mean)
	Baseline	6 months
Cohort 1a Sitters Placebo Then Treatment	2.28	2.32
Cohort 1b Sitters Treatment	2.93	2.37
Cohort 2 Standers and Walkers - Treatment	5.52	6.56

Max CMAP Amplitude Median

Intervention	mV (Median)
	Baseline	6 months
Cohort 1a Sitters Placebo Then Treatment	1.91	1.44
Cohort 1b Sitters Treatment	2.2	1.8
Cohort 2 Standers and Walkers - Treatment	5.3	5.85

Max CMAP Area (Mean)

The maximum Compound Motor Action Potential (CMAP) area is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This procedure is repeated multiple times. The maximum area is the response that results in the largest area under the response curve. (NCT00227266)
Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)

Intervention	mVms (Mean)
	Baseline	6 months
Cohort 1a Sitters Placebo Then Treatment	5.46	5.28
Cohort 1b Sitters Treatment	5.45	5.26
Cohort 2 Standers and Walkers - Treatment	14.85	16.26

Max CMAP Area (Median)

Intervention	mVms (Median)
	Baseline	6 months
Cohort 1a Sitters Placebo Then Treatment	3.6	3.74
Cohort 1b Sitters Treatment	4.6	3.4
Cohort 2 Standers and Walkers - Treatment	13.65	16.85

Modified Hammersmith Change From Baseline to 6 Months

Comparison of Modified Hammersmith Change from baseline to 6 months. Scores range from 0 to 40. A higher score indicates a better outcome. This scale is used to assess gross motor abilities of non-ambulant children with SMA in multiple research trials as well as in clinical settings. (NCT00227266)
Timeframe: 0 months, 6 months

Intervention	Score (Mean)
	Baseline visit (0 weeks)	6 Month visit (V2)	Change from Baseline
Cohort 1a Sitters Placebo Then Treatment	20.0	20.6	0.6
Cohort 1b Sitters Treatment	16.6	16.8	0.2

Modified Hammersmith Extend Baseline

"Baseline Modified Hammersmith Extend testing. The baseline test is the score they receive during their screening visits. This scale ranges from 0 to 56. A higher score indicates a better outcome.~This scale is used to assess gross motor abilities of children with SMA in multiple research trials as well as in clinical settings." (NCT00227266)
Timeframe: 1 month prior to enrollment, at enrollment (0 months)

Intervention	Score (Mean)
	Modified Hammersmith Extend at S1 (-4 weeks)	Modified Hammersmith Extend at S2 (0 weeks)
Cohort 2 Experimental	47.0	48.3

Trials

2 trials available for carnitine and Adult Spinal Muscular Atrophy

Article	Year
SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy. PloS one, 2010, Aug-19, Volume: 5, Issue:8 Topics: Age Factors; Body Composition; Body Mass Index; Body Weight; Bone Density; Carnitine; Child; Child,	2010
SMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy. PloS one, 2011, Volume: 6, Issue:7 Topics: Action Potentials; Adolescent; Anticonvulsants; Carnitine; Child; Child, Preschool; Demography; Fema	2011