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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: |
NCT00064792 |
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Date of registration:
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11/07/2003 |
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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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Simvastatin Therapy in Smith-Lemli-Opitz Syndrome
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Scientific title:
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Investigation of Simvastatin Therapy in Smith-Lemli-Opitz Syndrome |
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Date of first enrolment:
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July 2003 |
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Target sample size:
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23 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00064792 |
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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 2
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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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Forbes D Porter, 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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Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
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Key inclusion & exclusion criteria
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- INCLUSION CRITERIA:
All patients with biochemically proven SLOS will be considered for this study.
EXCLUSION CRITERIA:
Patients will be excluded if they cannot travel to the NIH because of their medical
condition.
Age less than 4 and older than 18.
Weight less than 10 kg.
Developmental delay too severe to obtain adequate behavioral evaluation.
Severe behavioral problems that preclude proper physical and laboratory medicine
evaluation.
SLOS severity score greater than 30.
No biochemical diagnosis of SLOS.
No molecular conformation of SLOS.
Residual fibroblasts enzymatic activity less than 10% of control value (cholesterol
synthesis as a fraction of total sterol synthesis).
Dehydrocholesterol/cholesterol ratio greater than 1.0.
Renal insufficiency.
Contraindications for simvastatin use:
History of hypersensitivity to simvastatin or other "statins."
Acute liver disease.
Persistent elevations of serum transaminase levels or persistent elevations of CPK.
Concomitant therapy with tetralol-class calcium channel blockers (such as mibefradil).
Pregnancy or lactation.
History of rhabdomyolysis or myopathy.
Concomitant therapy with other drugs associated with myopathy (such as gemfibrozil or other
fibrates, niacin) or metabolism by the P450 isoform 3A4 system (such as cyclosporin,
itraconazole, ketoconazole, macrolide antibiotics, or nefazodone (Serzone)).
Warfarin-type anticoagulant therapy.
Severe cataracts.
Age minimum:
4 Years
Age maximum:
18 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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Drug: OraPlus
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Drug: Simvastatin Susp.
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Primary Outcome(s)
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Serum Cholesterol to Total Sterol Ratio
[Time Frame: 1 year after therapy.]
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Secondary Outcome(s)
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Cerebral Spinal Fluid Dehydrocholesterol to Total Sterol Ratio
[Time Frame: 12 months]
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Secondary ID(s)
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030225
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03-CH-0225
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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