Trial Outcomes & Findings for CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I (NCT NCT00661453)
NCT ID: NCT00661453
Last Updated: 2015-06-15
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
40 participants
Primary outcome timeframe
-2 weeks, + 2 weeks, 3 months, 6 months
Results posted on
2015-06-15
Participant Flow
Participant milestones
| Measure |
SMA Type 1
All patients will receive VPA and carnitine.
Valproic Acid and Levocarnitine: Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight
|
|---|---|
|
Overall Study
STARTED
|
38
|
|
Overall Study
COMPLETED
|
25
|
|
Overall Study
NOT COMPLETED
|
13
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
Baseline characteristics by cohort
| Measure |
SMA Type 1
n=38 Participants
All patients will receive VPA and carnitine.
Valproic Acid and Levocarnitine: Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight
|
|---|---|
|
Age, Continuous
|
5.52 months
STANDARD_DEVIATION 2.72 • n=5 Participants
|
|
Sex: Female, Male
Female
|
18 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
2 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
34 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: -2 weeks, + 2 weeks, 3 months, 6 monthsOutcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: -2 weeks, time 0, 3 months, 6 monthsPopulation: 20 participants have baseline values, only 12 have 3 and 6 month values
Outcome measures
| Measure |
SMA Type 1
n=20 Participants
All patients will receive VPA and carnitine.
Valproic Acid and Levocarnitine: Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight
|
|---|---|
|
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)
Lean Mass 3 months
|
4993.92 g
Standard Deviation 1676.13
|
|
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)
Lean Mass 6 months
|
5133.83 g
Standard Deviation 2122.99
|
|
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)
Fat Mass Baseline
|
3011.37 g
Standard Deviation 1467.83
|
|
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)
Lean Mass Baseline
|
4317.15 g
Standard Deviation 1334.48
|
|
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)
Fat Mass 3 months
|
3618.25 g
Standard Deviation 1343.25
|
|
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)
Fat Mass 6 months
|
4316.08 g
Standard Deviation 1857.16
|
SECONDARY outcome
Timeframe: monthlyOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: time 0, and monthly for 12 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: -2 weeks, time 0, 3 months, 6 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: -2 weeks, time 0 , 3 months, or 6 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: -2 weeks, time 0, 3 months, 6 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: -2 weeks or time 0, 3 months, 6 monthsOutcome measures
Outcome data not reported
Adverse Events
SMA Type 1
Serious events: 29 serious events
Other events: 36 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
SMA Type 1
n=38 participants at risk
All patients will receive VPA and carnitine.
Valproic Acid and Levocarnitine: Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
7.9%
3/38 • Number of events 3
|
|
Cardiac disorders
Cardiac Arrest
|
5.3%
2/38 • Number of events 2
|
|
Congenital, familial and genetic disorders
Congenital familial and genetic disorders - Other, specify
|
5.3%
2/38 • Number of events 2
|
|
Gastrointestinal disorders
Dysphagia
|
2.6%
1/38 • Number of events 1
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
2.6%
1/38 • Number of events 1
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
18.4%
7/38 • Number of events 9
|
|
General disorders
Fever
|
2.6%
1/38 • Number of events 1
|
|
General disorders
Pain
|
2.6%
1/38 • Number of events 1
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
13.2%
5/38 • Number of events 6
|
|
Infections and infestations
Bronchial Infection
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Lip Infection
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Lung Infection
|
2.6%
1/38 • Number of events 2
|
|
Infections and infestations
Wound Infection
|
2.6%
1/38 • Number of events 1
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications - Other, specify
|
2.6%
1/38 • Number of events 1
|
|
Investigations
Urine output decreased
|
2.6%
1/38 • Number of events 1
|
|
Investigations
Investigations - Other, specify
|
2.6%
1/38 • Number of events 1
|
|
Metabolism and nutrition disorders
Dehydration
|
2.6%
1/38 • Number of events 1
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders - Other, specify
|
2.6%
1/38 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
18.4%
7/38 • Number of events 9
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
15.8%
6/38 • Number of events 7
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.6%
1/38 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
13.2%
5/38 • Number of events 6
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
2.6%
1/38 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
7.9%
3/38 • Number of events 5
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
|
34.2%
13/38 • Number of events 17
|
|
Vascular disorders
Hypotension
|
2.6%
1/38 • Number of events 1
|
Other adverse events
| Measure |
SMA Type 1
n=38 participants at risk
All patients will receive VPA and carnitine.
Valproic Acid and Levocarnitine: Drug: Valproic Acid and Levocarnitine; syrup; dosage is by weight
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
2.6%
1/38 • Number of events 1
|
|
Ear and labyrinth disorders
Middle Ear Inflammation
|
2.6%
1/38 • Number of events 1
|
|
Ear and labyrinth disorders
Ear and labyrinth disorders - Other, specify
|
2.6%
1/38 • Number of events 1
|
|
Eye disorders
Conjunctivitis
|
2.6%
1/38 • Number of events 1
|
|
Gastrointestinal disorders
Abdominal Pain
|
2.6%
1/38 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhea
|
5.3%
2/38 • Number of events 2
|
|
Gastrointestinal disorders
Flatulence
|
2.6%
1/38 • Number of events 1
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
2.6%
1/38 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
7.9%
3/38 • Number of events 3
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
7.9%
3/38 • Number of events 3
|
|
General disorders
Fever
|
31.6%
12/38 • Number of events 22
|
|
General disorders
Irritability
|
5.3%
2/38 • Number of events 4
|
|
General disorders
Pain
|
7.9%
3/38 • Number of events 3
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
26.3%
10/38 • Number of events 12
|
|
Immune system disorders
Allergic Reaction
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Bronchial Infection
|
7.9%
3/38 • Number of events 3
|
|
Infections and infestations
Conjunctivitis infective
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Lung infection
|
13.2%
5/38 • Number of events 7
|
|
Infections and infestations
Mucosal infection
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Rhinitis infective
|
5.3%
2/38 • Number of events 2
|
|
Infections and infestations
Sinusitis
|
2.6%
1/38 • Number of events 2
|
|
Infections and infestations
Skin Infection
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Urinary tract infection
|
2.6%
1/38 • Number of events 1
|
|
Infections and infestations
Infections and infestations - Other, specify
|
2.6%
1/38 • Number of events 1
|
|
Injury, poisoning and procedural complications
Fracture
|
2.6%
1/38 • Number of events 1
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications - Other, specify
|
2.6%
1/38 • Number of events 1
|
|
Investigations
Alanine aminotransferase increased
|
5.3%
2/38 • Number of events 2
|
|
Investigations
Neutrophil count decreased
|
2.6%
1/38 • Number of events 1
|
|
Investigations
Weight Loss
|
2.6%
1/38 • Number of events 1
|
|
Investigations
Investigations - Other, specify
|
2.6%
1/38 • Number of events 1
|
|
Metabolism and nutrition disorders
Glucose intolerance
|
2.6%
1/38 • Number of events 1
|
|
Nervous system disorders
Nystagmus
|
5.3%
2/38 • Number of events 2
|
|
Nervous system disorders
Vasovagal reaction
|
2.6%
1/38 • Number of events 1
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
2.6%
1/38 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
2.6%
1/38 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
5.3%
2/38 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
10.5%
4/38 • Number of events 7
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
10.5%
4/38 • Number of events 6
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
23.7%
9/38 • Number of events 15
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
7.9%
3/38 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
2.6%
1/38 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
|
31.6%
12/38 • Number of events 16
|
|
Skin and subcutaneous tissue disorders
Dry Skin
|
2.6%
1/38 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
5.3%
2/38 • Number of events 3
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
2.6%
1/38 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
2.6%
1/38 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place