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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. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 October 2017 |
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Main ID: |
NCT00661453 |
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Date of registration:
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14/04/2008 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
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Scientific title:
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Phase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I) |
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Date of first enrolment:
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April 2008 |
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Target sample size:
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40 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00661453 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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Canada
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Germany
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United States
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Contacts
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Name:
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Sandra P Reyna, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Families of Spinal Muscular Atrophy |
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Name:
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Kathryn Swoboda, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Utah |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Laboratory documentation of SMN mutation/deletion consistent with a genetic diagnosis
of SMA
- Clinical diagnosis of SMA type I
- Age 2 weeks to 12 months
- Written informed consent of parents/guardian
Exclusion Criteria:
- Any clinical or laboratory evidence of hepatic or pancreatic insufficiency.
- Laboratory results drawn within 14 days prior to start of study drug demonstrating:
Liver transaminases (AST, ALT), lipase, amylase: > 1.5 x ULN White Blood Cell Count: < 3
Neutropenia: <1 Platelet: <100K Hematocrit: <30, persisting over a 30-day period
- Serious illness requiring systemic treatment and/or hospitalization within two weeks
prior to study entry.
- Use of medications or supplements within 30 days of study enrollment that interfere
with VPA or carnitine metabolism; that increase the potential risks of VPA or
carnitine; or that are hypothesized to have a beneficial effect in SMA animal models
or human neuromuscular disorders, including riluzole, valproic acid, hydroxyurea, oral
use of albuterol, sodium phenylbutyrate, butyrate derivatives, creatinine, growth
hormone, anabolic steroids, probenecid, oral or parenteral use of corticosteroids at
entry, or agents anticipated to increase or decrease muscle strength or agents with
presumed histone deacetylase (HDAC) inhibition.
- Infants who have participated in a treatment trial for SMA within 30 days of study
entry or who will become enrollees in any other treatment trial during the course of
this study.
- Unwillingness to travel for study assessments.
- Coexisting medical conditions that contradict use of VPA/carnitine or travel to and
from study site.
Age minimum:
N/A
Age maximum:
12 Months
Gender:
All
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Health Condition(s) or Problem(s) studied
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Spinal Muscular Atrophy Type I
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Intervention(s)
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Drug: Valproic Acid and Levocarnitine
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Primary Outcome(s)
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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)
[Time Frame: -2 weeks, time 0, 3 months, 6 months]
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Laboratory Safety Data
[Time Frame: -2 weeks, + 2 weeks, 3 months, 6 months]
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Secondary Outcome(s)
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Time to Death or Ventilator Dependence (Defined as >16 Hours/Day)
[Time Frame: monthly]
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Maximum Ulnar CMAP Amplitude/Area and MUNE
[Time Frame: -2 weeks, time 0, 3 months, 6 months]
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Quantitative SMN mRNA and Protein Measures
[Time Frame: -2 weeks, time 0 , 3 months, or 6 months]
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Functional Motor Assessments: TIMPSI Scores
[Time Frame: -2 weeks, time 0, 3 months, 6 months]
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Primary Caregiver Functional Rating Scale for SMA Type I Subjects (PCFRS)
[Time Frame: time 0, and monthly for 12 months]
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Whole Body DEXA Scanning for Lean Body Mass and Total Bone Mineral Density/ Content
[Time Frame: -2 weeks or time 0, 3 months, 6 months]
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Secondary ID(s)
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IND 79276
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25409
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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