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Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy

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ClinicalTrials.gov Identifier: NCT00227266
Recruitment Status : Completed
First Posted : September 27, 2005
Results First Posted : May 3, 2011
Last Update Posted : September 26, 2011
Sponsor:
Collaborators:
Families of Spinal Muscular Atrophy
Leadiant Biosciences, Inc.
Abbott
Information provided by (Responsible Party):
Kathryn Swoboda, University of Utah

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Spinal Muscular Atrophy
Interventions Drug: Valproic Acid and Levocarnitine
Drug: Placebo
Enrollment 94
Recruitment Details Subject's were recruited during the periods of September 2005 to September 2006 across the United States.
Pre-assignment Details  
Arm/Group Title Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment Cohort 2 Standers and Walkers - Treatment
Hide Arm/Group Description Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form. Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form. Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Period Title: Overall Study
Started 31 30 33
Completed 30 30 29
Not Completed 1 0 4
Reason Not Completed
Withdrawal by Subject             0             0             1
Protocol Violation             1             0             1
Excessive weight gain             0             0             2
Arm/Group Title Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment Cohort 2 Standers and Walkers - Treatment Total
Hide Arm/Group Description Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form. Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form. Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form. Total of all reporting groups
Overall Number of Baseline Participants 31 30 33 94
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants 30 participants 33 participants 94 participants
<=18 years
31
 100.0%
30
 100.0%
33
 100.0%
94
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 31 participants 30 participants 33 participants 94 participants
4.4  (1.9) 4.3  (2.1) 7.3  (3.7) 5.4  (3.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants 30 participants 33 participants 94 participants
Female
11
  35.5%
17
  56.7%
11
  33.3%
39
  41.5%
Male
20
  64.5%
13
  43.3%
22
  66.7%
55
  58.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants 30 participants 33 participants 94 participants
Hispanic or Latino
2
   6.5%
1
   3.3%
0
   0.0%
3
   3.2%
Not Hispanic or Latino
29
  93.5%
27
  90.0%
30
  90.9%
86
  91.5%
Unknown or Not Reported
0
   0.0%
2
   6.7%
3
   9.1%
5
   5.3%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants 30 participants 33 participants 94 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
   3.2%
2
   6.7%
1
   3.0%
4
   4.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   3.2%
0
   0.0%
0
   0.0%
1
   1.1%
White
26
  83.9%
25
  83.3%
29
  87.9%
80
  85.1%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
3
   9.7%
3
  10.0%
3
   9.1%
9
   9.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 31 participants 30 participants 33 participants 94 participants
United States 25 26 29 80
Canada 6 4 4 14
1.Primary Outcome
Title Safety Labs
Hide Description Participants will have labs drawn regularly to maintain appropriate dosing and monitor liver function
Time Frame -4 wks, 0, 2 wks, 3 mo, 6 mo, 9 mo, 12 mo for safety labs; throughout for AEs
Outcome Measure Data Not Reported
2.Primary Outcome
Title Efficacy, Measured Through Motor Function Assessments
Hide Description [Not Specified]
Time Frame -4wks, 0, 3 mo, 6 mo, 12 mo
Outcome Measure Data Not Reported
3.Primary Outcome
Title Modified Hammersmith Change From Baseline to 6 Months
Hide Description 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.
Time Frame 0 months, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis only pertains to cohort 1a and 1b.
Arm/Group Title Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 31 30
Mean (Standard Deviation)
Unit of Measure: Score
Baseline visit (0 weeks) 20.0  (9.3) 16.6  (8.7)
6 Month visit (V2) 20.6  (8.1) 16.8  (7.9)
Change from Baseline 0.6  (3.98) 0.2  (2.88)
4.Secondary Outcome
Title Quantitative Assessment of SMN mRNA From Blood Samples
Hide Description [Not Specified]
Time Frame -4wks or 0, 3 mo, 6 mo, 12 mo
Outcome Measure Data Not Reported
5.Secondary Outcome
Title Peds QL™ Assessment: Parental Version (All), Child Versions (> 5yrs)
Hide Description [Not Specified]
Time Frame -4wks, 0, 3mo, 6mo, 12mo
Outcome Measure Data Not Reported
6.Secondary Outcome
Title Max CMAP Amplitude (Mean)
Hide Description 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.
Time Frame 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment Cohort 2 Standers and Walkers - Treatment
Hide Arm/Group Description:
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Overall Number of Participants Analyzed 23 21 24
Mean (Standard Deviation)
Unit of Measure: mV
Baseline 2.28  (1.55) 2.93  (1.56) 5.52  (2.56)
6 months 2.32  (1.75) 2.37  (1.82) 6.56  (2.99)
7.Secondary Outcome
Title Max CMAP Amplitude Median
Hide Description 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.
Time Frame 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment Cohort 2 Standers and Walkers - Treatment
Hide Arm/Group Description:
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Overall Number of Participants Analyzed 23 21 24
Median (Full Range)
Unit of Measure: mV
Baseline
1.91
(0.5 to 7.66)
2.2
(0.5 to 6.66)
5.3
(1.2 to 10.42)
6 months
1.44
(0.5 to 6.14)
1.8
(0.3 to 7.81)
5.85
(1.50 to 12.20)
8.Secondary Outcome
Title Ulnar MUNE
Hide Description [Not Specified]
Time Frame -4 wks, 0, 3 mo, 6 mo, 12 mo
Outcome Measure Data Not Reported
9.Secondary Outcome
Title Growth and Vital Sign Parameters
Hide Description [Not Specified]
Time Frame -4 wks, 0, 3mo, 6mo, 12mo
Outcome Measure Data Not Reported
10.Secondary Outcome
Title Nutritional Status
Hide Description [Not Specified]
Time Frame -4 wks, 0, 3mo, 6mo, 12mo
Outcome Measure Data Not Reported
11.Secondary Outcome
Title DEXA
Hide Description [Not Specified]
Time Frame 0, 6mo, 12mo
Outcome Measure Data Not Reported
12.Secondary Outcome
Title Max CMAP Area (Mean)
Hide Description 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.
Time Frame 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment Cohort 2 Standers and Walkers - Treatment
Hide Arm/Group Description:
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Overall Number of Participants Analyzed 23 21 24
Mean (Standard Deviation)
Unit of Measure: mVms
Baseline 5.46  (5.03) 5.45  (4.23) 14.85  (7.68)
6 months 5.28  (4.49) 5.26  (4.65) 16.26  (7.13)
13.Secondary Outcome
Title Max CMAP Area (Median)
Hide Description 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.
Time Frame 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment Cohort 2 Standers and Walkers - Treatment
Hide Arm/Group Description:
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form.
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
Overall Number of Participants Analyzed 23 21 24
Median (Full Range)
Unit of Measure: mVms
Baseline
3.6
(0.7 to 19.71)
4.6
(1.3 to 16.78)
13.65
(2.6 to 38.29)
6 months
3.74
(0.6 to 16.13)
3.4
(0.6 to 18.81)
16.85
(3.7 to 29.10)
14.Post-Hoc Outcome
Title Modified Hammersmith Extend Baseline
Hide Description

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.

Time Frame 1 month prior to enrollment, at enrollment (0 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was determined per protocol
Arm/Group Title Cohort 2 Experimental
Hide Arm/Group Description:
Patients in cohort 2 (SMA "standers and walkers") will receive VPA + Carnitine treatment for the entire 12 month time period.
Overall Number of Participants Analyzed 33
Mean (Full Range)
Unit of Measure: Score
Modified Hammersmith Extend at S1 (-4 weeks)
47.0
(29 to 56)
Modified Hammersmith Extend at S2 (0 weeks)
48.3
(36 to 56)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort 2 Experimental
Comments MHFMS-Extend was not normally distributed at p=0.048. Test-retest reliability of MHFMS-Extend measurements from the first (S1) to the second (S2) screening visit was analyzed using Spearman’s correlation.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Spearman's correlation
Comments [Not Specified]
Method of Estimation Estimation Parameter Spearman's correlation
Estimated Value 0.93
Estimation Comments [Not Specified]
Time Frame Phase 1 Serious Adverse Events (time period during which placebo (1a) and treatment (1b)were randomly treated): 6 months. And Phase 1 and 2 Adverse Events: 12 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment Cohort 2 Standers and Walkers - Treatment
Hide Arm/Group Description Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor or equivalent placebo in the liquid form. Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form. Dosage of the VPA will start at 10-20 mg/kg/day divided into two or tree doses. The dose will be adjusted to achieve a therapeutic trough level of 50-120 micrograms/ml. VPA will be given in the form of 125 mg sprinkle capsules. Dosage for Carnitor will be 50 mg/kg/day with a maximum dose of 10000 mg/day divided into two doses. Carnitor elixir comes as 500 mg/5 ml. All subjects will be given Carnitor in the liquid form.
All-Cause Mortality
Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment Cohort 2 Standers and Walkers - Treatment
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment Cohort 2 Standers and Walkers - Treatment
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/31 (3.23%)   4/30 (13.33%)   4/33 (12.12%) 
Gastrointestinal disorders       
Diarrhea  0/31 (0.00%)  0/30 (0.00%)  2/33 (6.06%) 
Vomiting  0/31 (0.00%)  1/30 (3.33%)  1/33 (3.03%) 
General disorders       
General Disorder [1]  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Infections and infestations       
Pneumonitis  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Upper respiratory infection  1/31 (3.23%)  0/30 (0.00%)  1/33 (3.03%) 
Metabolism and nutrition disorders       
Dehydration  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Cough  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Pneumonia  0/31 (0.00%)  2/30 (6.67%)  1/33 (3.03%) 
Tachyponea  0/31 (0.00%)  1/30 (3.33%)  1/33 (3.03%) 
Skin and subcutaneous tissue disorders       
Skin Rash  0/31 (0.00%)  0/30 (0.00%)  1/33 (3.03%) 
[1]
Pyrexia
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cohort 1a Sitters Placebo Then Treatment Cohort 1b Sitters Treatment Cohort 2 Standers and Walkers - Treatment
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   18/31 (58.06%)   23/30 (76.67%)   28/33 (84.85%) 
Ear and labyrinth disorders       
Otorrhoea  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Gastrointestinal disorders       
Abdominal Pain Upper  2/31 (6.45%)  3/30 (10.00%)  7/33 (21.21%) 
Constipation  1/31 (3.23%)  0/30 (0.00%)  1/33 (3.03%) 
Diarrhea  0/31 (0.00%)  2/30 (6.67%)  1/33 (3.03%) 
Dry Mouth  1/31 (3.23%)  0/30 (0.00%)  0/33 (0.00%) 
Gastroesophagheal Reflux Disease  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Nausea  2/31 (6.45%)  4/30 (13.33%)  2/33 (6.06%) 
Vomiting  6/31 (19.35%)  12/30 (40.00%)  6/33 (18.18%) 
General disorders       
Fatigue  2/31 (6.45%)  0/30 (0.00%)  1/33 (3.03%) 
Pyrexia  4/31 (12.90%)  5/30 (16.67%)  7/33 (21.21%) 
Immune system disorders       
Dermatitis Allergic  3/31 (9.68%)  2/30 (6.67%)  1/33 (3.03%) 
Hypersensitivity  1/31 (3.23%)  0/30 (0.00%)  0/33 (0.00%) 
Multiple Allergies  0/31 (0.00%)  2/30 (6.67%)  5/33 (15.15%) 
Infections and infestations       
Bronchitis  0/31 (0.00%)  1/30 (3.33%)  1/33 (3.03%) 
Croup  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Ear Infection  2/31 (6.45%)  3/30 (10.00%)  4/33 (12.12%) 
Nasopharyngitis  3/31 (9.68%)  3/30 (10.00%)  0/33 (0.00%) 
Pneumonitis  1/31 (3.23%)  1/30 (3.33%)  0/33 (0.00%) 
Sinusitis  1/31 (3.23%)  1/30 (3.33%)  2/33 (6.06%) 
Tinea Infection  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Upper Respiratory Infection  2/31 (6.45%)  1/30 (3.33%)  0/33 (0.00%) 
Urinary Tract Infection  0/31 (0.00%)  3/30 (10.00%)  0/33 (0.00%) 
Injury, poisoning and procedural complications       
Join Sprain  1/31 (3.23%)  0/30 (0.00%)  0/33 (0.00%) 
Investigations       
Oxygen Saturation Decreased  0/31 (0.00%)  0/30 (0.00%)  1/33 (3.03%) 
Weight Incresed  4/31 (12.90%)  3/30 (10.00%)  0/33 (0.00%) 
Metabolism and nutrition disorders       
Decreased Appetite  1/31 (3.23%)  0/30 (0.00%)  0/33 (0.00%) 
Dehydration  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Increased Appetite  2/31 (6.45%)  0/30 (0.00%)  0/33 (0.00%) 
Musculoskeletal and connective tissue disorders       
Arthralgia  1/31 (3.23%)  0/30 (0.00%)  0/33 (0.00%) 
Femur Fracture  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Foot Fracture  0/31 (0.00%)  0/30 (0.00%)  2/33 (6.06%) 
Forearm Fracture  0/31 (0.00%)  0/30 (0.00%)  1/33 (3.03%) 
Hand Fracture  0/31 (0.00%)  0/30 (0.00%)  1/33 (3.03%) 
Joint Pain  1/31 (3.23%)  1/30 (3.33%)  1/33 (3.03%) 
Muscle Cramp  0/31 (0.00%)  0/30 (0.00%)  1/33 (3.03%) 
Pain in Extremity  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Patella Fracture  0/31 (0.00%)  0/30 (0.00%)  0/33 (0.00%) 
Nervous system disorders       
Agitation  1/31 (3.23%)  0/30 (0.00%)  0/33 (0.00%) 
Headache  1/31 (3.23%)  2/30 (6.67%)  0/33 (0.00%) 
Impulsive Behaviour  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Irritability  2/31 (6.45%)  1/30 (3.33%)  0/33 (0.00%) 
Lethargy  1/31 (3.23%)  0/30 (0.00%)  0/33 (0.00%) 
Tremor  2/31 (6.45%)  0/30 (0.00%)  0/33 (0.00%) 
Psychiatric disorders       
Emotional Disorder of Childhood  2/31 (6.45%)  0/30 (0.00%)  0/33 (0.00%) 
Renal and urinary disorders       
Incontinence  1/31 (3.23%)  0/30 (0.00%)  0/33 (0.00%) 
Increased Urination Frequency  1/31 (3.23%)  0/30 (0.00%)  0/33 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Chocking Sensation  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Cough  2/31 (6.45%)  7/30 (23.33%)  4/33 (12.12%) 
Dyspnea  2/31 (6.45%)  0/30 (0.00%)  0/33 (0.00%) 
Nasal Congestion  2/31 (6.45%)  1/30 (3.33%)  2/33 (6.06%) 
Pharyngeal Pain  1/31 (3.23%)  0/30 (0.00%)  1/33 (3.03%) 
Pharyngeal Pain  0/31 (0.00%)  0/30 (0.00%)  0/33 (0.00%) 
Pneumonia  2/31 (6.45%)  6/30 (20.00%)  2/33 (6.06%) 
Rhinorrhoea  0/31 (0.00%)  0/30 (0.00%)  1/33 (3.03%) 
Tachypnoea  0/31 (0.00%)  1/30 (3.33%)  0/33 (0.00%) 
Skin and subcutaneous tissue disorders       
Rash Papular  1/31 (3.23%)  1/30 (3.33%)  0/33 (0.00%) 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Sandra Reyna, M.D.
Organization: University of Utah
Phone: 801-581-3551
EMail: sreyna@genetics.utah.edu
Publications:
Lindstedt S, Lindstedt G. Distribution and Excretion of Carnitine in the Rat. Acta. Chem. Scand. 1961;15:701-702
Scriver C, Beautet A, Sly W, Valle D. The Metabolic Basis of Inherited Disease. New York: McGraw Hill, 1989
Schaub J, Van Hoof F, Vis H. Inborn Errors of Metabolism. New York: Raven Press, 1991
Layout table for additonal information
Responsible Party: Kathryn Swoboda, University of Utah
ClinicalTrials.gov Identifier: NCT00227266     History of Changes
Other Study ID Numbers: 13698
First Submitted: September 23, 2005
First Posted: September 27, 2005
Results First Submitted: March 19, 2010
Results First Posted: May 3, 2011
Last Update Posted: September 26, 2011