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Trial record 1 of 1 for:    20808854 [PUBMED-IDS]
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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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Subject's were recruited during the periods of September 2005 to September 2006 across the United States.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Cohort 1a Sitters Placebo Then Treatment 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.
Cohort 1b Sitters Treatment 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.
Cohort 2 Standers and Walkers - Treatment 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.

Participant Flow:   Overall Study
    Cohort 1a Sitters Placebo Then Treatment   Cohort 1b Sitters Treatment   Cohort 2 Standers and Walkers - Treatment
STARTED   31   30   33 
COMPLETED   30   30   29 
NOT COMPLETED   1   0   4 
Withdrawal by Subject                0                0                1 
Protocol Violation                1                0                1 
Excessive weight gain                0                0                2 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Cohort 1a Sitters Placebo Then Treatment 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.
Cohort 1b Sitters Treatment 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.
Cohort 2 Standers and Walkers - Treatment 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 Total of all reporting groups

Baseline Measures
   Cohort 1a Sitters Placebo Then Treatment   Cohort 1b Sitters Treatment   Cohort 2 Standers and Walkers - Treatment   Total 
Overall Participants Analyzed 
[Units: Participants]
 31   30   33   94 
Age 
[Units: Participants]
       
<=18 years   31   30   33   94 
Between 18 and 65 years   0   0   0   0 
>=65 years   0   0   0   0 
Age 
[Units: Years]
Mean (Standard Deviation)
 4.4  (1.9)   4.3  (2.1)   7.3  (3.7)   5.4  (3.0) 
Gender 
[Units: Participants]
       
Female   11   17   11   39 
Male   20   13   22   55 
Ethnicity (NIH/OMB) 
[Units: Participants]
       
Hispanic or Latino   2   1   0   3 
Not Hispanic or Latino   29   27   30   86 
Unknown or Not Reported   0   2   3   5 
Race (NIH/OMB) 
[Units: Participants]
       
American Indian or Alaska Native   0   0   0   0 
Asian   1   2   1   4 
Native Hawaiian or Other Pacific Islander   0   0   0   0 
Black or African American   1   0   0   1 
White   26   25   29   80 
More than one race   0   0   0   0 
Unknown or Not Reported   3   3   3   9 
Region of Enrollment 
[Units: Participants]
       
United States   25   26   29   80 
Canada   6   4   4   14 


  Outcome Measures

1.  Primary:   Modified Hammersmith Change From Baseline to 6 Months   [ Time Frame: 0 months, 6 months ]

2.  Secondary:   Max CMAP Amplitude (Mean)   [ Time Frame: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available) ]

3.  Secondary:   Max CMAP Amplitude Median   [ Time Frame: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available) ]

4.  Secondary:   Max CMAP Area (Mean)   [ Time Frame: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available) ]

5.  Secondary:   Max CMAP Area (Median)   [ Time Frame: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available) ]

6.  Post-Hoc:   Modified Hammersmith Extend Baseline   [ Time Frame: 1 month prior to enrollment, at enrollment (0 months) ]

7.  Primary:   Safety Labs   [ Time Frame: -4 wks, 0, 2 wks, 3 mo, 6 mo, 9 mo, 12 mo for safety labs; throughout for AEs ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

8.  Primary:   Efficacy, Measured Through Motor Function Assessments   [ Time Frame: -4wks, 0, 3 mo, 6 mo, 12 mo ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

9.  Secondary:   Quantitative Assessment of SMN mRNA From Blood Samples   [ Time Frame: -4wks or 0, 3 mo, 6 mo, 12 mo ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

10.  Secondary:   Peds QL™ Assessment: Parental Version (All), Child Versions (> 5yrs)   [ Time Frame: -4wks, 0, 3mo, 6mo, 12mo ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

11.  Secondary:   Ulnar MUNE   [ Time Frame: -4 wks, 0, 3 mo, 6 mo, 12 mo ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

12.  Secondary:   Growth and Vital Sign Parameters   [ Time Frame: -4 wks, 0, 3mo, 6mo, 12mo ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

13.  Secondary:   Nutritional Status   [ Time Frame: -4 wks, 0, 3mo, 6mo, 12mo ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

14.  Secondary:   DEXA   [ Time Frame: 0, 6mo, 12mo ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

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:  
Name/Title: Sandra Reyna, M.D.
Organization: University of Utah
phone: 801-581-3551
e-mail: 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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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