Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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19 October 2017 |
Main ID: |
NCT00227266 |
Date of registration:
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23/09/2005 |
Prospective Registration:
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No |
Primary sponsor: |
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Public title:
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Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
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Scientific title:
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Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial) |
Date of first enrolment:
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September 2005 |
Target sample size:
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94 |
Recruitment status: |
Completed |
URL:
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https://clinicaltrials.gov/show/NCT00227266 |
Study type:
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Interventional |
Study design:
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Phase:
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Phase 2
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Countries of recruitment
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Canada
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United States
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Contacts
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Name:
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Kathryn J Swoboda, M.D. |
Address:
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Telephone:
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Email:
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Affiliation:
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University of Utah/Primary Children's Medical Center |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
Cohort 1
- Confirmed genetic diagnosis of 5q SMA
- SMA 2 or non-ambulatory SMA 3: all subjects must be able to sit independently for at
least 3 seconds without support
- Age 2 to 8 years at time of enrollment
Cohort 2
- Confirmed genetic diagnosis of 5q SMA
- SMA subjects (SMA types 2 or 3) who can stand independently without braces or other
support for up to 2 seconds, or walk independently
- Age 3 to 17 years at time of study enrollment
Exclusion Criteria:
Cohort 1
- Need for BiPAP support > 12 hours per day
- Spinal rod or fixation for scoliosis or anticipated need within six months of
enrollment
- Inability to meet study visit requirements or cooperate reliably with functional
testing
- Coexisting medical conditions that contraindicate travel, testing or study medications
- Use of medications or supplements which interfere with valproic acid or carnitine
metabolism within 3 months of study enrollment.
- Current use of either VPA or carnitine. If study subject is taking VPA or carnitine
then patient must go through a washout period of 12 weeks before enrollment into the
study
- Body Mass Index > 90th % for age
Cohort 2
- Spinal rod or fixation for scoliosis or anticipated need within six months of
enrollment
- Inability to meet study visit requirements or cooperate with functional testing
- Transaminases, amylase or lipase > 3.0 x normal values, WBC < 3.0 or neutropenia <
1.0, platelets < 100 K, or hematocrit < 30 persisting over a 30 day period.
- Coexisting medical conditions that contraindicate travel, testing or study medications
- Use of medications or supplements which interfere with valproic acid or carnitine
metabolism within 3 months of study enrollment.
- Current use of either VPA or carnitine. If study subject is taking VPA or carnitine
then patient must be go through a washout period of 12 weeks before enrollment in the
study.
- Body Mass Index > 90th % for age
- Pregnant women/girls, or those intending to try to become pregnant during the course
of the study.
Age minimum:
2 Years
Age maximum:
17 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Spinal Muscular Atrophy
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Intervention(s)
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Drug: Valproic Acid and Levocarnitine
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Drug: Placebo
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Primary Outcome(s)
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Modified Hammersmith Change From Baseline to 6 Months
[Time Frame: 0 months, 6 months]
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Efficacy, Measured Through Motor Function Assessments
[Time Frame: -4wks, 0, 3 mo, 6 mo, 12 mo]
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Safety Labs
[Time Frame: -4 wks, 0, 2 wks, 3 mo, 6 mo, 9 mo, 12 mo for safety labs; throughout for AEs]
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Secondary Outcome(s)
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Peds QL™ Assessment: Parental Version (All), Child Versions (> 5yrs)
[Time Frame: -4wks, 0, 3mo, 6mo, 12mo]
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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)]
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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)]
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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)]
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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)]
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DEXA
[Time Frame: 0, 6mo, 12mo]
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Growth and Vital Sign Parameters
[Time Frame: -4 wks, 0, 3mo, 6mo, 12mo]
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Nutritional Status
[Time Frame: -4 wks, 0, 3mo, 6mo, 12mo]
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Ulnar MUNE
[Time Frame: -4 wks, 0, 3 mo, 6 mo, 12 mo]
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Quantitative Assessment of SMN mRNA From Blood Samples
[Time Frame: -4wks or 0, 3 mo, 6 mo, 12 mo]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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