Study Stopped
poor enrollment (only one subject enrolled)
SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation
Smith-Lemli Opitz Syndrome: A Clinical Investigation of the Effect of Simvastatin in Patients Receiving Cholesterol Supplementation
1 other identifier
interventional
1
1 country
1
Brief Summary
The purpose of this study is to determine if simvastatin improves development and behavior in patients with Smith Lemli-Opitz syndrome (SLOS) receiving dietary cholesterol supplementation.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable
Started Sep 2011
Typical duration for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 1, 2011
CompletedFirst Submitted
Initial submission to the registry
September 7, 2011
CompletedFirst Posted
Study publicly available on registry
September 15, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2014
CompletedResults Posted
Study results publicly available
October 1, 2019
CompletedOctober 1, 2019
September 1, 2019
3.1 years
September 7, 2011
September 12, 2019
September 12, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Development Quotient (DQ)
neurocognitive assessment measured with Mullen Scales of Learning
through study completion, an average of 2 per year
Secondary Outcomes (6)
Whole Body Cholesterol Pool Size, Synthesis & Absorption Using Stable Isotope Testing
end of treatment, an average of 1 per year
Plasma Marker of Sterol Metabolism
through study completion, an average of 2 per year
ADC
end of treatment, an average of 1 per year
MVA
through study completion, an average of 2 per year
MRS Lipids
end of treatment, an average of 1 per year
- +1 more secondary outcomes
Study Arms (2)
Placebo
PLACEBO COMPARATORplacebo
Simvastatin
EXPERIMENTAL0.5 mg/kg body weight/day
Interventions
Simvastatin will be administered as a powder mixed with lactose at the dose of 0.5 mg/kg/day
Eligibility Criteria
You may qualify if:
- Male or female over 1 years old
- Subject has confirmed diagnosis of Smith-Lemli-Opitz Syndrome
- Subject is currently receiving cholesterol supplementation
You may not qualify if:
- Subjects too ill to travel to the study site
- Subjects who are unable to safely undergo study procedures
- Pregnant women
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Oregon Health and Science University
Portland, Oregon, 97239, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
Analyses were not completed and will never be completed due to insufficient funding
Results Point of Contact
- Title
- Dr. jean-Baptiste Roullet
- Organization
- Washington State University
Study Officials
- PRINCIPAL INVESTIGATOR
Jean-Baptiste Roullet, PhD
Washington State University
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- RANDOMIZED
- Masking
- SINGLE
- Who Masked
- PARTICIPANT
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Clinical Professor
Study Record Dates
First Submitted
September 7, 2011
First Posted
September 15, 2011
Study Start
September 1, 2011
Primary Completion
October 1, 2014
Study Completion
October 1, 2014
Last Updated
October 1, 2019
Results First Posted
October 1, 2019
Record last verified: 2019-09