Page last updated: 2024-10-16

carnitine and Muscular Atrophy, Spinal

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

Muscular Atrophy, Spinal: A group of disorders marked by progressive degeneration of motor neurons in the spinal cord resulting in weakness and muscular atrophy, usually without evidence of injury to the corticospinal tracts. Diseases in this category include Werdnig-Hoffmann disease and later onset SPINAL MUSCULAR ATROPHIES OF CHILDHOOD, most of which are hereditary. (Adams et al., Principles of Neurology, 6th ed, p1089)

Research Excerpts

ExcerptRelevanceReference
"Valproic acid (VPA) has demonstrated potential as a therapeutic candidate for spinal muscular atrophy (SMA) in vitro and in vivo."2.75SMA 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)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's0 (0.00)29.6817
2010's2 (100.00)24.3611
2020's0 (0.00)2.80

Authors

AuthorsStudies
Swoboda, KJ2
Scott, CB2
Crawford, TO2
Simard, LR2
Reyna, SP2
Krosschell, KJ2
Acsadi, G2
Elsheik, B2
Schroth, MK2
D'Anjou, G2
LaSalle, B2
Prior, TW2
Sorenson, SL1
Maczulski, JA2
Bromberg, MB2
Chan, GM2
Kissel, JT2
Sorenson, S1

Clinical Trials (1)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial)[NCT00227266]Phase 294 participants (Actual)Interventional2005-09-30Completed
[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)

,,
InterventionmV (Mean)
Baseline6 months
Cohort 1a Sitters Placebo Then Treatment2.282.32
Cohort 1b Sitters Treatment2.932.37
Cohort 2 Standers and Walkers - Treatment5.526.56

Max CMAP Amplitude Median

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)

,,
InterventionmV (Median)
Baseline6 months
Cohort 1a Sitters Placebo Then Treatment1.911.44
Cohort 1b Sitters Treatment2.21.8
Cohort 2 Standers and Walkers - Treatment5.35.85

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)

,,
InterventionmVms (Mean)
Baseline6 months
Cohort 1a Sitters Placebo Then Treatment5.465.28
Cohort 1b Sitters Treatment5.455.26
Cohort 2 Standers and Walkers - Treatment14.8516.26

Max CMAP Area (Median)

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)

,,
InterventionmVms (Median)
Baseline6 months
Cohort 1a Sitters Placebo Then Treatment3.63.74
Cohort 1b Sitters Treatment4.63.4
Cohort 2 Standers and Walkers - Treatment13.6516.85

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

,
InterventionScore (Mean)
Baseline visit (0 weeks)6 Month visit (V2)Change from Baseline
Cohort 1a Sitters Placebo Then Treatment20.020.60.6
Cohort 1b Sitters Treatment16.616.80.2

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)

InterventionScore (Mean)
Modified Hammersmith Extend at S1 (-4 weeks)Modified Hammersmith Extend at S2 (0 weeks)
Cohort 2 Experimental47.048.3

Trials

2 trials available for carnitine and Muscular Atrophy, Spinal

ArticleYear
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