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CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

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ClinicalTrials.gov Identifier: NCT00661453
Recruitment Status : Completed
First Posted : April 18, 2008
Results First Posted : June 15, 2015
Last Update Posted : June 15, 2015
Sponsor:
Collaborators:
Families of Spinal Muscular Atrophy
Leadiant Biosciences, Inc.
Information provided by (Responsible Party):
Kathryn Swoboda, University of Utah

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Spinal Muscular Atrophy Type I
Intervention Drug: Valproic Acid and Levocarnitine
Enrollment 40
Recruitment Details  
Pre-assignment Details  
Arm/Group Title SMA Type 1
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All patients will receive VPA and carnitine.

Valproic Acid and Levocarnitine: Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight

Period Title: Overall Study
Started 38
Completed 25
Not Completed 13
Arm/Group Title SMA Type 1
Hide Arm/Group Description

All patients will receive VPA and carnitine.

Valproic Acid and Levocarnitine: Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight

Overall Number of Baseline Participants 38
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Months
Number Analyzed 38 participants
5.52  (2.72)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 38 participants
Female
18
  47.4%
Male
20
  52.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 38 participants
Canada 2
United States 34
Germany 2
1.Primary Outcome
Title Laboratory Safety Data
Hide Description [Not Specified]
Time Frame -2 weeks, + 2 weeks, 3 months, 6 months
Outcome Measure Data Not Reported
2.Primary Outcome
Title Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures)
Hide Description [Not Specified]
Time Frame -2 weeks, time 0, 3 months, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
20 participants have baseline values, only 12 have 3 and 6 month values
Arm/Group Title SMA Type 1
Hide Arm/Group Description:

All patients will receive VPA and carnitine.

Valproic Acid and Levocarnitine: Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight

Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: g
Lean Mass Baseline 4317.15  (1334.48)
Lean Mass 3 months 4993.92  (1676.13)
Lean Mass 6 months 5133.83  (2122.99)
Fat Mass Baseline 3011.37  (1467.83)
Fat Mass 3 months 3618.25  (1343.25)
Fat Mass 6 months 4316.08  (1857.16)
3.Secondary Outcome
Title Time to Death or Ventilator Dependence (Defined as >16 Hours/Day)
Hide Description [Not Specified]
Time Frame monthly
Outcome Measure Data Not Reported
4.Secondary Outcome
Title Primary Caregiver Functional Rating Scale for SMA Type I Subjects (PCFRS)
Hide Description [Not Specified]
Time Frame time 0, and monthly for 12 months
Outcome Measure Data Not Reported
5.Secondary Outcome
Title Functional Motor Assessments: TIMPSI Scores
Hide Description [Not Specified]
Time Frame -2 weeks, time 0, 3 months, 6 months
Outcome Measure Data Not Reported
6.Secondary Outcome
Title Quantitative SMN mRNA and Protein Measures
Hide Description [Not Specified]
Time Frame -2 weeks, time 0 , 3 months, or 6 months
Outcome Measure Data Not Reported
7.Secondary Outcome
Title Maximum Ulnar CMAP Amplitude/Area and MUNE
Hide Description [Not Specified]
Time Frame -2 weeks, time 0, 3 months, 6 months
Outcome Measure Data Not Reported
8.Secondary Outcome
Title Whole Body DEXA Scanning for Lean Body Mass and Total Bone Mineral Density/ Content
Hide Description [Not Specified]
Time Frame -2 weeks or time 0, 3 months, 6 months
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title SMA Type 1
Hide Arm/Group Description

All patients will receive VPA and carnitine.

Valproic Acid and Levocarnitine: Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight

All-Cause Mortality
SMA Type 1
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
SMA Type 1
Affected / at Risk (%) # Events
Total   29/38 (76.32%)    
Cardiac disorders   
Cardiac Arrest  2/38 (5.26%)  2
Congenital, familial and genetic disorders   
Congenital familial and genetic disorders - Other, specify  2/38 (5.26%)  2
Gastrointestinal disorders   
Dysphagia  1/38 (2.63%)  1
Gastroesophageal reflux disease  1/38 (2.63%)  1
Gastrointestinal disorders - Other, specify  7/38 (18.42%)  9
General disorders   
Fever  1/38 (2.63%)  1
Pain  1/38 (2.63%)  1
General disorders and administration site conditions - Other, specify  5/38 (13.16%)  6
Infections and infestations   
Bronchial Infection  1/38 (2.63%)  1
Lip Infection  1/38 (2.63%)  1
Lung Infection  1/38 (2.63%)  2
Wound Infection  1/38 (2.63%)  1
Injury, poisoning and procedural complications   
Injury, poisoning and procedural complications - Other, specify  1/38 (2.63%)  1
Investigations   
Urine output decreased  1/38 (2.63%)  1
Investigations - Other, specify  1/38 (2.63%)  1
Metabolism and nutrition disorders   
Dehydration  1/38 (2.63%)  1
Metabolism and nutrition disorders - Other, specify  1/38 (2.63%)  1
Respiratory, thoracic and mediastinal disorders   
Pneumonitis  3/38 (7.89%)  3
Aspiration  7/38 (18.42%)  9
Atelectasis  6/38 (15.79%)  7
Cough  1/38 (2.63%)  1
Hypoxia  5/38 (13.16%)  6
Nasal Congestion  1/38 (2.63%)  1
Respiratory Failure  3/38 (7.89%)  5
Respiratory, thoracic and mediastinal disorders - Other, specify  13/38 (34.21%)  17
Vascular disorders   
Hypotension  1/38 (2.63%)  1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
SMA Type 1
Affected / at Risk (%) # Events
Total   36/38 (94.74%)    
Blood and lymphatic system disorders   
Anemia  1/38 (2.63%)  1
Ear and labyrinth disorders   
Middle Ear Inflammation  1/38 (2.63%)  1
Ear and labyrinth disorders - Other, specify  1/38 (2.63%)  1
Eye disorders   
Conjunctivitis  1/38 (2.63%)  1
Gastrointestinal disorders   
Abdominal Pain  1/38 (2.63%)  1
Diarrhea  2/38 (5.26%)  2
Flatulence  1/38 (2.63%)  1
Gastroesophageal reflux disease  1/38 (2.63%)  1
Vomiting  3/38 (7.89%)  3
Gastrointestinal disorders - Other, specify  3/38 (7.89%)  3
General disorders   
Fever  12/38 (31.58%)  22
Irritability  2/38 (5.26%)  4
Pain  3/38 (7.89%)  3
General disorders and administration site conditions - Other, specify  10/38 (26.32%)  12
Immune system disorders   
Allergic Reaction  1/38 (2.63%)  1
Infections and infestations   
Bronchial Infection  3/38 (7.89%)  3
Conjunctivitis infective  1/38 (2.63%)  1
Lung infection  5/38 (13.16%)  7
Mucosal infection  1/38 (2.63%)  1
Rhinitis infective  2/38 (5.26%)  2
Sinusitis  1/38 (2.63%)  2
Skin Infection  1/38 (2.63%)  1
Urinary tract infection  1/38 (2.63%)  1
Infections and infestations - Other, specify  1/38 (2.63%)  1
Injury, poisoning and procedural complications   
Fracture  1/38 (2.63%)  1
Injury, poisoning and procedural complications - Other, specify  1/38 (2.63%)  1
Investigations   
Alanine aminotransferase increased  2/38 (5.26%)  2
Neutrophil count decreased  1/38 (2.63%)  1
Weight Loss  1/38 (2.63%)  1
Investigations - Other, specify  1/38 (2.63%)  1
Metabolism and nutrition disorders   
Glucose intolerance  1/38 (2.63%)  1
Nervous system disorders   
Nystagmus  2/38 (5.26%)  2
Vasovagal reaction  1/38 (2.63%)  1
Nervous system disorders - Other, specify  1/38 (2.63%)  1
Respiratory, thoracic and mediastinal disorders   
Aspiration  1/38 (2.63%)  1
Atelectasis  2/38 (5.26%)  2
Cough  4/38 (10.53%)  7
Hypoxia  4/38 (10.53%)  6
Nasal congestion  9/38 (23.68%)  15
Pneumonitis  3/38 (7.89%)  3
Productive cough  1/38 (2.63%)  1
Respiratory, thoracic and mediastinal disorders - Other, specify  12/38 (31.58%)  16
Skin and subcutaneous tissue disorders   
Dry Skin  1/38 (2.63%)  1
Rash maculo-papular  2/38 (5.26%)  3
Skin ulceration  1/38 (2.63%)  1
Skin and subcutaneous tissue disorders - Other, specify  1/38 (2.63%)  1
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Kathryn Swoboda
Organization: University of Utah
Phone: 801-585-9717
EMail: swoboda@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: NCT00661453     History of Changes
Other Study ID Numbers: 25409
IND 79276
First Submitted: April 14, 2008
First Posted: April 18, 2008
Results First Submitted: June 1, 2015
Results First Posted: June 15, 2015
Last Update Posted: June 15, 2015