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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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12 December 2020 |
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Main ID: |
NCT01434745 |
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Date of registration:
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07/09/2011 |
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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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SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation
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Scientific title:
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Smith-Lemli Opitz Syndrome: A Clinical Investigation of the Effect of Simvastatin in Patients Receiving Cholesterol Supplementation |
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Date of first enrolment:
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September 2011 |
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Target sample size:
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1 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT01434745 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Single (Participant).
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Phase:
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N/A
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Countries of recruitment
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United States
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Contacts
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Name:
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Jean-Baptiste Roullet, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Washington State University |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Male or female over 1 years old
- Subject has confirmed diagnosis of Smith-Lemli-Opitz Syndrome
- Subject is currently receiving cholesterol supplementation
Exclusion Criteria:
- Subjects too ill to travel to the study site
- Subjects who are unable to safely undergo study procedures
- Pregnant women
Age minimum:
1 Year
Age maximum:
89 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Smith-Lemli-Opitz Syndrome
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Intervention(s)
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Dietary Supplement: Lactose
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Drug: Simvastatin
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Primary Outcome(s)
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Development Quotient (DQ)
[Time Frame: through study completion, an average of 2 per year]
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Secondary Outcome(s)
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Whole Body Cholesterol Pool Size, Synthesis & Absorption Using Stable Isotope Testing
[Time Frame: end of treatment, an average of 1 per year]
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FA
[Time Frame: end of treatment, an average of 1 per year]
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Plasma Marker of Sterol Metabolism
[Time Frame: through study completion, an average of 2 per year]
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MRS Lipids
[Time Frame: end of treatment, an average of 1 per year]
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ADC
[Time Frame: end of treatment, an average of 1 per year]
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MVA
[Time Frame: through study completion, an average of 2 per year]
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Secondary ID(s)
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R01HL073980
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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